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hematology

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Table of Contents

Overview

Definition and Scope

is defined as the study of blood and blood-forming organs, encompassing the , treatment, and prevention of diseases related to blood, bone marrow, and the immunologic, hemostatic, and vascular systems.[3.1] This branch of is concerned with understanding the causes, , treatment, and prevention of blood-related diseases, which include disorders affecting , hemoglobin, blood proteins, platelets, and the mechanisms of blood circulation.[4.1] Hematology is the study of blood and blood-forming organs, which includes the diagnosis, treatment, and prevention of diseases affecting the blood, bone marrow, and related systems such as the immunologic, hemostatic, and vascular systems.[3.1] Hematologists and hematopathologists are specialized healthcare providers who focus on blood disorders and their components.[5.1] As analytical methods in hematology have advanced, they have become more sensitive, allowing for the use of smaller sample volumes in diagnostic procedures.[3.1] Hematology encompasses not only the biological aspects of blood disorders but also the significant roles played by and . Research indicates that environmental conditions, such as exposure to certain chemicals, , or medications during pregnancy or throughout life, can increase the risk of developing genetic and blood disorders.[21.1] While is a critical factor in many cases, accumulating evidence underscores the profound influence of environmental factors on the onset and progression of these conditions.[22.1] Furthermore, genetic and environmental factors often interact in a non-additive manner, resulting in gene–environment (G × E) interactions that can synergistically elevate disease risk.[24.1] Understanding these interactions is essential for advancing our knowledge of blood disorders and improving diagnostic and therapeutic .

Importance in Clinical Practice

Hematology plays a crucial role in , particularly in the diagnosis and of various blood disorders. Genetic mutations significantly influence the development of these disorders, with conditions such as sickle cell anemia (SCA) and hemophilia A serving as prime examples. SCA is characterized by a mutation that alters the shape of red blood cells, which, while causing complications, also provides some protection against infection, illustrating a complex interaction between and environmental stressors.[6.1] Hemophilia A, on the other hand, results from abnormalities in the gene responsible for producing factor VIII, a protein essential for blood clotting, leading to challenges in managing bleeding episodes.[7.1] In clinical practice, understanding and managing noncancerous blood disorders is crucial for improving patient outcomes. These disorders, such as Diamond-Blackfan anemia, hinder the bone marrow's ability to produce adequate red blood cells, necessitating ongoing medical intervention.[8.1] Educating patients about their hematologic diseases is a key intervention that promotes and empowers patients to take an active role in their care. This should encompass information on the of the disease, , potential complications, and necessary .[12.1] Prior studies have shown that effective strategies can lead to improved outcomes in chronic illnesses, particularly for conditions like sickle cell disease (SCD) and thalassemia.[10.1] To facilitate this education, healthcare providers can employ various strategies, including simplifying medical jargon and personalizing treatment plans, which help make complex information more accessible to patients.[9.1] initiatives also play a significant role in raising awareness and promoting screening for hematological diseases, particularly in underserved communities. Comprehensive preventive care strategies, such as routine check-ups and blood tests, are essential for monitoring chronic conditions like hemophilia and thalassemia, thereby improving patient outcomes.[14.1] Furthermore, innovative health campaigns are necessary to address systemic barriers and improve , ensuring that all individuals have access to vital information and resources related to blood health.[16.1] Recent advancements in hematology, particularly in , have revolutionized treatment options for inherited blood disorders. For example, a groundbreaking international study demonstrated that over 80% of patients with severe genetic conditions remained transfusion-free after receiving gene addition therapy, highlighting the potential of this approach to transform patient care.[18.1] These developments underscore the importance of hematology in clinical practice, as they not only enhance treatment efficacy but also contribute to improved for patients with blood disorders.

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History

Key Milestones in Hematology Development

One of the pivotal milestones in the of hematology was the discovery of the hematopoietic function of bone marrow by Ernst Neumann and Giulio Bizzozero in 1868, which significantly advanced the understanding of blood formation and its disorders.[39.1] This period also marked the emergence of hematology as a distinct medical specialty, with its own methodologies and hospital wards, evolving from its previous classification as merely a part of during the late 19th and early 20th centuries.[42.1] The history of began in the 1600s when British scientist William Harvey discovered that blood circulated through the heart instead of the lungs.[41.1] This groundbreaking discovery led doctors to reason that could be beneficial in treating various conditions, including and even mental illness.[41.1] However, the first blood transfusions were largely unsuccessful, with many recipients dying as a result.[45.1] Despite these challenges, British and American doctors continued to experiment with blood transfusions, including attempts to transfuse blood between animals.[45.1] These early efforts laid the foundation for the development of hematology as a scientific discipline, despite the significant obstacles faced by researchers at the time. The first official meeting of the American Society of Hematology (ASH) in April 1958 marked another significant milestone, bringing together over 300 hematologists to discuss clinical and research matters related to blood and blood diseases.[40.1] Since its inception, ASH has played a crucial role in advancing the field of hematology through research, education, and , further solidifying hematology as a vital medical discipline.[55.1] The period from 1771 to 1917 also witnessed remarkable advancements in the understanding of blood types and their compatibility, which were essential for improving blood transfusion and efficacy.[43.1] These developments have had a lasting impact on and patient care, shaping the landscape of hematology as we know it today.

Recent Advancements

Innovations in Diagnostics

Recent advancements in hematology have significantly transformed diagnostic practices, particularly through the integration of (AI) . One of the primary applications of AI in this field is the of blood smears, which enhances diagnostic accuracy and efficiency. AI algorithms can analyze a wide range of data, including laboratory test results and patient , to provide healthcare providers with for and therapeutic interventions.[115.1] A notable example of AI's impact is the AI100 automated machine developed by SigTuple Technologies, which facilitates the microscopic analysis of peripheral blood smears. This has demonstrated significant improvements in diagnostic accuracy, speed, and across various hematological cases, including alpha-thalassemia trait and acute myeloid (AML).[108.1] Furthermore, AI-assisted flow cytometry has been validated in clinical settings, showing that AI models can improve diagnostic accuracy and reduce analysis time to under five minutes per case.[114.1] The integration of AI in hematological diagnostics is not limited to blood smear analysis; it also encompasses various diagnostic subfields such as and . A comprehensive review of AI applications in hematology highlights the importance of understanding concepts for hematologists, as these systems are increasingly becoming integral to routine practice.[116.1] Moreover, AI algorithms are being developed to enhance risk stratification and enable of abnormalities, which are crucial for improving patient outcomes.[117.1] However, it is essential to adopt a critical perspective regarding the implementation of AI in hematology, ensuring that its benefits are maximized while addressing potential risks.[117.1] Overall, the advancements in AI and machine learning are poised to revolutionize hematological diagnostics, leading to more strategies and improved patient care.

Breakthroughs in Treatment Modalities

Recent advancements in hematology have led to significant breakthroughs in treatment modalities, particularly in the management of acute myeloid leukemia (AML) and other blood disorders. Over the past decade, there has been a notable expansion in the understanding of AML's molecular profile, which has facilitated the development of targeted therapies aimed at specific driver mutations such as NPM1, FLT3-ITD, and IDH1-2. These advancements have resulted in several FDA-approved drugs that target these mutations, enhancing survival outcomes for patients with actionable mutations in AML.[94.1] In addition to targeted therapies, the integration of has transformed treatment paradigms for hematologic malignancies. Various categories of immunotherapies, including chimeric antigen T-cell (CAR-T) therapy, have shown substantial efficacy in treating conditions such as B-cell lymphoma and other blood cancers. The first CAR-T products were approved between 2017 and 2019, demonstrating remarkable efficacy in and real-world settings, although they often require prolonged hospitalization due to associated toxicities.[89.1] As protocols for toxicity management have improved, there is a growing interest in administering CAR-T therapies in outpatient settings.[89.1] Moreover, the application of artificial intelligence (AI) and chemotherapy-free treatments has emerged as a promising avenue in hematology, addressing longstanding challenges in blood disorders. Recent studies presented at the 65th American Society of Hematology Annual Meeting highlighted the benefits of these cutting-edge tools and treatments, indicating a shift towards more innovative and effective management strategies.[84.1]

Blood Composition And Function

Components of Blood

Blood is composed of several key components, each of which plays a vital role in maintaining physiological functions. The primary constituents of human blood include plasma, red blood cells (erythrocytes), white blood cells (leukocytes), and platelets. Plasma is the liquid component of blood in which the blood cells are suspended. Red blood cells are responsible for carrying oxygen from the lungs to the rest of the body, while white blood cells play a crucial role in fighting infections and aiding in the immune process. Additionally, platelets are involved in the clotting process, contributing to .[126.1] Red blood cells are responsible for transporting oxygen from the lungs to tissues throughout the body, while also facilitating the return of dioxide from the tissues back to the lungs for exhalation.[126.1] White blood cells are crucial for the , helping to fight infections and protect the body against foreign invaders.[126.1] Platelets play an essential role in hemostasis, contributing to blood clotting and processes.[125.1] Hematology is the study of blood, which encompasses much more than just blood tests or donations. It involves the analysis of blood components and the diagnosis and treatment of various blood diseases. Hematologists are at the forefront of advancements in the of these conditions, which can significantly impact production and function. For instance, infections often lead to an elevation in the number of white blood cells, while many forms of cancer can disrupt normal blood cell production. Specifically, an increase in immature white blood cells in a complete blood count (CBC) can be associated with leukemia. Additionally, blood diseases such as anemia and sickle cell disease are known to cause abnormally low levels of hemoglobin.[128.1] Moreover, blood can be separated into various components for therapeutic purposes, including packed red blood cell concentrates, platelet concentrates, fresh frozen plasma, and cryoprecipitate, each serving specific medical needs.[129.1] Understanding the composition and function of blood is critical for effective diagnosis and treatment in clinical practice, as variations in blood components can indicate underlying health issues.[167.1]

Roles of Blood Cells

Blood is composed of various cellular components, each playing a crucial role in maintaining physiological homeostasis. The primary components of blood include red blood cells (RBCs), white blood cells (WBCs), and platelets, all of which interact intricately to support the body's functions and respond to injuries. Red blood cells are essential for gas transport, primarily delivering oxygen from the lungs to tissues and facilitating the removal of carbon dioxide from tissues back to the lungs. This function is vital for maintaining the body's metabolic processes and overall homeostasis.[142.1] The between the production and destruction of RBCs is critical; any disruption can lead to hematological disorders such as anemia, characterized by a deficiency in red blood cell numbers or hemoglobin content, or polycythemia, which involves excessive proliferation of RBCs.[143.1] White blood cells, or leukocytes, are integral to the immune system, providing defense against infections and foreign substances. They are involved in various , including the identification and destruction of , thus playing a key role in maintaining the body's health and homeostasis. Platelets, or thrombocytes, are cell fragments that play a crucial role in hemostasis, the process of blood clotting. When a is injured, platelets adhere to the damaged tissue to form a temporary plug (primary hemostasis). This initial clot is then stabilized by a series of coagulation factors in the blood, which amplify the clotting effects and lead to the formation of a more permanent thrombus (secondary hemostasis).[141.1] This process is essential to prevent excessive blood loss and protect the body from infection. The interactions among various blood components are crucial for maintaining homeostasis. Physiological hemostasis involves complex interactions between endothelial cells, platelets, and coagulation proteins, resulting in the formation of a platelet plug and localized thrombus at the site of vascular injury.[139.1] Numerous regulatory processes are in place to prevent widespread activation of coagulation, ensuring that blood remains fluid in the absence of vascular injury.[139.1] Disruptions in these interactions can lead to various blood conditions and cancers, including anemia, such as hemophilia, blood clots, and cancers like leukemia, lymphoma, and myeloma.[164.1] Hematologic tests are essential for diagnosing these conditions, as they assess various blood components, including red blood cells, white blood cells, and platelets.[166.1] These tests help identify diseases of the blood and bone marrow, illustrating the importance of understanding blood composition and function in clinical practice.[166.1] Recent advancements in blood have led to significant breakthroughs in the treatment of hematological disorders. In November 2023, the UK regulatory agency became the first in the world to approve a revolutionary gene therapy called Casgevy, which utilizes technology to address conditions caused by defective hemoglobin production or function, including sickle cell disease and beta thalassemia.[163.1] This corrects faulty hemoglobin genes in a patient’s bone marrow , enabling them to produce functioning hemoglobin. Remarkably, a single treatment has the potential to cure some patients for life, underscoring the transformative impact of these developments on our understanding and management of blood-related diseases.[162.1]

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Common Hematological Disorders

Anemias

Anemia is defined as a decrease in the number of red blood cells (RBCs) or a reduction in the normal quantity of hemoglobin in the blood, making it the most common disorder of the blood.[169.1] It can arise from various causes, including , nutritional deficiencies, and . The major causes of anemia include iron deficiency, folate deficiency, hookworm infection, and malaria, with approximately 50% of cases related to iron deficiency.[200.1] Different types of anemia can be categorized based on their underlying mechanisms. For instance, occurs when the bone marrow fails to produce sufficient blood cells, including RBCs.[170.1] Autoimmune hemolytic anemia is characterized by an overactive immune response that destroys the body's own red blood cells.[170.1] Additionally, microcytic anemia due to iron deficiency is particularly prevalent in children and can lead to cognitive issues if not addressed.[205.1] The of anemia can vary significantly depending on its type and underlying cause. While all forms of anemia are characterized by low levels of functional red blood cells, the severity and treatment strategies differ. For example, nutritional anemia may require supplementation with iron, vitamin B12, or folate, while hereditary forms like sickle cell anemia may necessitate more complex management strategies.[203.1] for anemia typically include raising the RBC count or hemoglobin levels to improve oxygen transport, addressing the underlying causes, and preventing complications such as heart or nerve damage.[204.1] In cases of mild or moderate anemia without symptoms, treatment may not be immediately necessary.[204.1] Understanding the specific type of anemia is crucial for effective management and improving patient outcomes.

Blood Cancers

Blood cancers, also known as hematologic malignancies, encompass a range of disorders that the blood, bone marrow, and . These include leukemia, lymphoma, and multiple myeloma, each characterized by the uncontrolled proliferation of blood cells. Recent advancements in the diagnosis and treatment of these conditions have significantly improved patient outcomes. One notable development in the treatment of blood cancers is the introduction of bispecific antibodies, which target two different antigens simultaneously. This innovative approach has shown promise in treating patients with refractory leukemia and lymphoma, providing new therapeutic options for those who have not responded to traditional treatments.[179.1] Additionally, the last decade has seen the approval of new therapies for thrombotic thrombocytopenic purpura (TTP), such as caplacizumab and recombinant ADAMTS13 (rADAMTS13). These therapies have been shown to enhance acute TTP outcomes, reduce the need for plasma therapy, and decrease the time to clinical response, thereby alleviating the treatment burden on patients.[180.1] The integration of advanced technologies, including artificial intelligence (AI), has also transformed the landscape of hematology. AI-assisted systems have been applied in various aspects of clinical diagnosis and treatment, focusing on , , cytogenetics, and . These systems aid in comprehensive typing diagnosis and have the potential to improve disease prediction, , and prognosis tracking.[181.1] Furthermore, advancements in genetic and profiling have provided deeper insights into the of hematologic disorders, facilitating more personalized treatment approaches.[177.1]

Diagnosis And Management

Diagnostic Tools and Techniques

Diagnosis of hematologic disorders relies on a variety of tools and techniques that are essential for accurate assessment and management. Blood tests, including a complete blood count (CBC), blood smears, and specific tests for clotting factors, are fundamental in diagnosing conditions such as anemia, infections, and blood-clotting disorders.[219.1] Additionally, bone marrow examinations, which may involve aspiration or biopsy, are critical for evaluating bone marrow function and diagnosing malignancies like leukemia and lymphoma.[220.1] The integration of advanced technologies has significantly enhanced the diagnostic landscape in hematology. in hematology laboratories has improved efficiency and accuracy, allowing for streamlined blood analysis processes that can handle increased workloads while maintaining high standards of care.[235.1] Furthermore, artificial intelligence (AI) has emerged as a transformative force in hematological diagnostics. AI applications, such as automated analysis of blood smears and flow cytometry, have demonstrated substantial improvements in diagnostic accuracy and speed, enabling quicker and more reliable assessments of hematologic conditions.[238.1] Emerging diagnostic techniques, particularly in genetic sequencing, are expected to further refine the diagnosis and assessment of hematological disorders. As genetic syndromes are identified, these tests will enhance the precision of diagnoses, allowing for more tailored treatment strategies.[224.1] The use of technology also represents a significant advancement, as it miniaturizes and integrates laboratory processes into a single chip, reducing the amount of blood required for testing and facilitating rapid analysis at the point of care.[223.1]

Treatment Strategies

Effective treatment strategies in hematology are increasingly reliant on multidisciplinary approaches that integrate the expertise of various healthcare professionals. The integration of teams (MDTs) has been shown to significantly enhance patient outcomes by combining diverse expertise, which is essential for managing complex hematologic conditions. These teams typically include hematologists, oncologists, nurses, pharmacists, and other specialists who collaborate to provide comprehensive care tailored to individual patient needs.[244.1] The adoption of MDTs has been linked to improved decision-making, care coordination, and adherence to clinical guidelines, which collectively contribute to better patient outcomes, including reduced complications and enhanced overall survival rates.[245.1] Furthermore, the structured nature of MDT meetings facilitates among specialists, ensuring that treatment plans are comprehensive and consider all aspects of a patient's health.[247.1] This collaborative approach is particularly beneficial in the management of hematologic malignancies, where treatment often requires input from multiple disciplines to navigate the complexities of patient care.[233.1] In addition to the collaborative framework provided by MDTs, effective are crucial in the treatment of hematologic disorders. Research indicates that effective doctor-patient directly enhances patient outcomes, adherence to treatment, and overall satisfaction, while also reducing the risk of .[228.1] This includes avoiding technical jargon and ensuring that patients understand their diagnosis and treatment options.[228.1] Moreover, as telemedicine becomes more prevalent, developing effective communication methods for delivering sensitive information in a digital context is becoming increasingly important.[227.1] Educational interventions are crucial in the management of patients with hematologic disorders, as they significantly enhance patient knowledge regarding their conditions, treatment options, and potential .[229.1] Such have been recognized as vital contributors to successful self-management and improved outcomes in various chronic diseases, including those related to hematology.[230.1] Furthermore, cognitive and emotional factors play a significant role in influencing decision-making processes and the evaluation of risks and benefits associated with new treatment approaches.[239.1] By implementing adaptive strategies that promote patient participation and adherence to treatment, healthcare providers can foster a shared decision-making (SDM) approach, which is essential for effective in hematological conditions.[239.1]

Blood Transfusion And Component Therapy

Types of Blood Transfusions

Blood transfusions can be categorized into several types based on the components being transfused and the clinical indications for their use. The most common types of blood transfusions include red blood cell (RBC) transfusions, platelet transfusions, plasma transfusions, and cryoprecipitate transfusions. Red blood cell (RBC) transfusions are critical in the management of critically ill children, particularly those with acquired and congenital heart disease. To date, there are no published guidelines specifically directing RBC transfusion decision-making for this population.[256.1] The Transfusion and Anemia Expertise Initiative (TAXI) has developed recommendations and supporting for RBC transfusions in critically ill children, which were established through a consensus conference series of multidisciplinary, international experts in RBC transfusion management.[257.1] These recommendations aim to address the unique physiological differences between pediatric patients and adults, which are essential considerations in the decision-making process for blood transfusions and component therapy in critical care settings.[256.1] Blood component therapy is crucial in the management of and patients, as well as those experiencing critical illnesses. This therapy includes various types of blood transfusions, which are tailored to meet the specific needs of patients based on a thorough understanding of the associated risks and benefits of transfusion.[256.1] It is essential for healthcare providers to consider these factors when determining the appropriate blood components to administer, ensuring optimal patient outcomes. Blood transfusions, which involve the transfer of blood and its components such as red blood cells (RBCs), platelets, plasma, and cryoprecipitate, are among the most frequently performed medical procedures in developed countries. The decision to administer a transfusion is complex and requires careful consideration by medical practitioners, as it involves evaluating the specific needs of the patient and the potential risks associated with the procedure.[259.1] Blood transfusion involves the perioperative administration of blood and its components, which is crucial for various medical conditions. It is essential to adhere to proper indications for blood component therapy due to the potential adverse effects and costs associated with transfusions. Over the years, the importance of blood components in treating specific diseases and conditions has been increasingly recognized.[261.1] The practice of whole blood transfusion has largely been supplanted by component therapy, which allows for more targeted treatment. Whole blood or modified whole blood may still be used in specific scenarios, such as large-volume blood loss, where it can save time and reduce the number of donors exposed to the recipient.[260.1]

Risks and Benefits

Blood transfusions and component therapy play a crucial role in the management of patients with hematological conditions, particularly those undergoing such as chemotherapy. The primary purpose of blood transfusions is to replace lost blood components, which is essential for maintaining acceptable hemoglobin levels in patients experiencing anemia due to their treatment.[266.1] However, while transfusions can alleviate symptoms and improve quality of life, they also carry inherent risks, including adverse reactions and the potential for immune complications.[265.1] Anemia is highly prevalent among patients with advanced cancer and significantly impacts their quality of life.[264.1] Despite the common use of red blood cell (RBC) transfusions to manage this condition, there is limited data on their clinical utility and effectiveness, with no or clinical practice guidelines available to guide their use.[264.1] Approximately 15.1% of blood resources in the United States are allocated to hematology/oncology patients, highlighting the importance of transfusion therapy in this population.[266.1] The risks associated with blood transfusions necessitate comprehensive patient education and . It is crucial for healthcare providers to communicate effectively regarding the benefits, risks, and alternatives of transfusing blood products, including red blood cells, plasma, and platelets, ensuring that patients fully understand the implications of receiving these products.[271.1] A significant concern in this context is the potential for transfusion-transmitted infections (TTIs), which pose considerable risks due to factors such as the asymptomatic infectious phase in donors and the persistence of pathogens despite processing and storage.[277.1] Recent advancements in donor testing technologies, particularly testing (NAT), have been implemented to screen for TTIs in donated blood samples, thereby enhancing blood safety.[276.1] Furthermore, the introduction of Reduction Technology (PRT) represents a notable innovation in blood transfusion safety, as it actively targets and dismantles the DNA or RNA of pathogens that may be present in blood products.[279.1] Bacterial contamination of blood products, particularly platelets, has long been a significant concern in transfusion safety. In response to this issue, various advancements have been made over the past decade to avoid, detect, and eliminate this complication. These improvements include enhanced donor selection processes and specialized preparation techniques for the arm prior to needle insertion.[289.1] Additionally, a review of studies over the last 30 years has identified several interventions aimed at improving transfusion safety. These interventions include platelet pathogen inactivation, restrictive transfusion practices, the Peri-operative Transfusion Trigger Score of Emergency (Potts-E), and the use of hemoglobin-based oxygen carrier-201 (HBOC-201) as an alternative to reduce or eliminate perioperative transfusions.[290.1] Collectively, these measures have contributed to enhancing the overall efficacy and safety of blood component therapy.

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Bone Marrow Transplantation

Indications for Transplant

(BMT) is indicated for a variety of hematological conditions, primarily those involving malignancies and certain non-. The eligibility for transplantation is influenced by multiple factors, including the type of disease, the patient's overall health, and the compatibility between donor and recipient. The eligibility criteria for hematopoietic (HCT) in malignant diseases have evolved over time, influenced by the outcomes of both frontline and relapse chemotherapy protocols as well as transplantation outcomes. These criteria are shaped by the balance between the effectiveness of these treatment protocols and the degree of compatibility within each donor-recipient pair, which is crucial for successful transplantation.[314.1] As a result, the determination of eligibility for HCT is a complex process that considers various factors, including the patient's disease status and the specific characteristics of the donor-recipient match.[314.1] In non-malignant diseases, such as aplastic anemia, studies have shown that hematopoietic (HPSCT) can yield improved outcomes compared to conventional .[315.1] Specifically, a study involving 1,886 patients indicated that cells collected from the bone marrow provided superior outcomes compared to those collected from peripheral blood.[318.1] Furthermore, haploidentical stem cell transplantation has emerged as a viable option for patients lacking a matched unrelated donor, offering advantages such as lower costs and quicker access to hematopoietic cell products.[315.1] The success rates of bone marrow transplants (BMT) are influenced by several factors, including the patient's age, overall health, and the specific type of disease being treated.[319.1] Additionally, the matching of the donor's stem cells to the recipient's genetic markers, known as HLA matching, is essential to prevent complications during the transplant process.[319.1] Research indicates that patients with severe aplastic anemia (SAA) who receive matched sibling donor grafts experience some of the highest survival rates, often exceeding 80% at five years.[320.1] Understanding these factors is crucial for improving outcomes in BMT and addressing the complexities associated with transplant rates. Recent advancements in biology are promising for enhancing the efficacy of BMT. Innovative methods for activating and expanding from human bone marrow have been developed, which may facilitate bone marrow regeneration and the construction of new blood and immune systems.[336.1] Moreover, breakthroughs in creating lab-grown blood stem cells that closely resemble those in the human body could transform the landscape of bone marrow transplantation.[335.1] These developments highlight the ongoing evolution of BMT indications and the potential for improved patient outcomes through tailored .

Procedure and Recovery

Bone marrow transplantation (BMT) is a complex and multifaceted procedure that serves as a critical for various hematologic disorders, including leukemia, lymphoma, and multiple myeloma. The process involves replacing damaged or destroyed bone marrow with healthy stem cells, which can be sourced from the patient’s own body (autologous transplant) or from a compatible donor (allogeneic transplant).[300.1] Bone marrow transplantation (BMT) is a widely accepted treatment for various hematologic disorders, including malignant diseases such as leukemia, lymphoma, and multiple myeloma, as well as for conditions like aplastic anemia and genetically determined diseases.[299.1] The procedure is often preceded by a preparatory phase that may involve intensive chemoradiotherapy aimed at eliminating diseased cells and preparing the patient for transplantation.[298.1] Following this conditioning regimen, the actual transplantation involves the infusion of stem cells into the patient's bloodstream, which allows these cells to migrate to the bone marrow and initiate the production of new blood cells.[298.1] However, many patients encounter difficulties in proceeding with allogeneic transplantation due to the unavailability of suitable bone marrow donors, particularly when HLA-matched unrelated donors are not accessible.[299.1] after a bone marrow transplantation can be a highly stressful experience for patients, as the treatment is aggressive and often leads to significant physical, social, psychological, and emotional stress.[326.1] Patients frequently express concerns regarding the success of the transplant, potential complications, and their long-term prognosis, which can contribute to feelings of sadness, hopelessness, or .[309.1] Furthermore, research indicates that patients who report inadequate social support prior to the transplant are associated with poorer survival outcomes, highlighting the critical importance of emotional and psychological support during this challenging period.[326.1] Therefore, addressing the emotional and psychological needs of patients through effective support systems is essential for improving their overall and recovery outcomes.[309.1] Undergoing a bone marrow transplantation can be an anxiety-provoking experience, as patients often worry about the success of the transplant, potential complications, and their long-term prognosis. These concerns can lead to feelings of sadness, hopelessness, or depression, highlighting the importance of addressing the psychological effects of the procedure alongside its physical aspects.[309.1] Support systems are crucial in helping patients navigate these emotional challenges. The National Marrow Donor Program (NMDP) offers a Center that connects bone marrow and blood stem cell transplant recipients and their families to free emotional, financial, and educational resources, assisting them in coping with illness, treatment, transplant, and recovery.[321.1] Additionally, the National Bone Marrow Transplant Link (nbmtLINK) provides psychosocial and educational support to patients, , and healthcare professionals throughout the bone marrow, stem cell, and CAR-T journey.[322.1] By acknowledging these psychological challenges and seeking help, patients can better manage the emotional terrain that accompanies this life-changing experience.[327.1] Patients undergoing bone marrow transplantation face significant physical and emotional challenges that can impact their treatment outcomes. Research indicates that patient psychological distress is associated with various aspects of the bone marrow transplantation process and has been linked to poor treatment outcomes.[328.1] Addressing these emotional and psychological needs through support networks and educational resources is crucial. Specifically, pre-transplant emotional support has been associated with longer survival after allogeneic hematopoietic stem cell transplantation, underscoring the importance of providing comprehensive emotional care throughout the transplant journey.[311.1] Therefore, effective management of both physical and emotional health is essential to optimize recovery and outcomes for patients following bone marrow transplantation.

Hematology-Oncology Intersection

Blood Cancers and Treatment Approaches

Blood cancers, including , lymphomas, and myelomas, represent a diverse group of malignancies that arise from blood-forming tissues and cells of the immune system. The biological heterogeneity of these cancers is significant, as they can originate from various stem cells at different hierarchical levels of hematopoietic and lymphoid cell development, which is associated with a variety of etiological mechanisms.[343.1] Among these, leukemia and lymphoma exhibit distinct biological behaviors; leukemia primarily the bone marrow, leading to the overproduction of abnormal white blood cells, while lymphoma originates in the lymphatic system, resulting in abnormal cell clusters primarily within lymph nodes and other lymphatic tissues.[344.1] Hematologic malignancies are the most frequent cancers in the pediatric population, with leukemia for approximately 28% of cancer diagnoses, particularly acute lymphoblastic leukemia, while lymphomas represent about 9% of cases, ranking third after brain tumors.[346.1] The treatment approaches for these malignancies differ significantly from those used in adults, primarily due to fundamental biological differences between the two age groups. Pediatric patients often tolerate chemotherapy better than adults because their organs are healthier, which contributes to a higher chance of recovery; indeed, most children with cancer can be cured.[349.1] Additionally, at a genetic level, pediatric cancers typically exhibit fewer mutations compared to adult cancers, with childhood cancer genomes often containing as few as ten genetic mutations, many of which are rare or absent in adult cancers.[350.1] These differences in biology and genetics significantly influence treatment strategies for hematologic malignancies in children. The treatment and management of hematologic malignancies, such as leukemia and lymphoma, are significantly influenced by genetic mutations. Specifically, mutations like RUNX1 and IKZF1 are associated with the progression to accelerated or blast phases in chronic myeloid leukemia (CML), although they are rare in certain patient populations.[371.1] Additionally, germline mutations present a clinical challenge in the treatment of these malignancies, and ongoing research into their contribution to leukemogenesis is essential for advancing preemptive testing and treatment options for individuals with such mutations linked to leukemia and lymphoma.[372.1] The drive toward is based on the premise that the most effective treatment can be reliably selected for an individual using precise diagnostic and prognostic information. However, current practices often involve "group medicine," where patients are assigned to prognostic groups that guide treatment decisions for conditions like acute leukemia, lymphoma, and myeloma.[373.1] Continued research into the underlying mechanisms of these malignancies is essential for enhancing patient care and developing more effective treatment strategies.[342.1] As the field evolves, the integration of hematology and oncology remains critical in addressing the complexities of blood cancers and improving outcomes for affected individuals.

Emerging Therapies in Hematology-Oncology

Recent advancements in hematology-oncology have led to significant breakthroughs in nonmalignant hematology care. Notably, the 2023 FDA approvals of Casgevy and Lyfgenia for the treatment of patients with sickle cell disease represent a major milestone in this field.[351.1] Furthermore, during the 65th American Society of Hematology (ASH) Annual Meeting and Exposition, results from the phase 2/3 CLIMB SCD-121 trial (NCT03745287) were presented, showcasing the use of exagamglogene autotemcel (exa-cel).[351.1] These developments underscore the ongoing evolution of treatment protocols in hematology-oncology. In the realm of malignant hematology, the importance of minimal residual disease (MRD) has been underscored, particularly in multiple myeloma, Hodgkin disease, and acute leukemias. New agents introduced into standard regimens have improved the likelihood of patients achieving MRD-negative status.[352.1] Furthermore, the randomized Phase 2 study of Axatilimab showed that over 75% of subjects who had failed prior FDA-approved therapies responded positively, with a median failure-free survival of 17 months, offering new hope for patients suffering from chronic graft-versus-host disease (GVHD).[352.1] is rapidly advancing, with several categories being developed and evaluated in clinical trials for the treatment of blood cancers. These categories include stem cell transplantation, , antigen-targeted antibodies, antibody-drug conjugates, tumor , and adoptive (ACT).[353.1] Among these, CAR T- has emerged as a particularly promising approach for treating . This therapy involves genetically modifying T cells to recognize specific tumor-associated antigens, allowing them to exert cytotoxic effects on .[356.1] The development of CD19-targeted CAR T-cell therapy has been particularly notable, as it is currently approved for relapsed/refractory diffuse large B-cell lymphoma and acute lymphoblastic leukemia, marking a significant therapeutic advance in recent years.[354.1] The complete remission rate for CAR T-cell therapy in diffuse large B-cell lymphoma is reported to be 43%, highlighting its effectiveness in this patient population.[355.1] Recent advancements in hematology-oncology have introduced several promising therapies that have the potential to significantly alter treatment protocols. Notably, marstacimab, a novel monoclonal antibody that neutralizes tissue factor pathway inhibitors, has been investigated for patients with severe hemophilia A and moderate to severe hemophilia B, enhancing thrombin generation through the extrinsic coagulation pathway.[352.1] Additionally, the Phase 2 study of Axatilimab demonstrated that over 75% of subjects who had failed prior FDA-approved therapies responded positively, with a median failure-free survival of 17 months, offering new hope for patients suffering from chronic graft-versus-host disease (GVHD).[352.1] The role of minimal residual disease (MRD) has also been underscored in the management of malignant hematology, particularly in multiple myeloma, Hodgkin disease, and acute leukemias, with the introduction of new agents improving the likelihood of achieving MRD-negative status.[352.1] Furthermore, chimeric antigen receptor T-cell (CAR-T) therapy has transformed the management of patients with relapsed/refractory hematologic malignancies, including B-cell lymphomas and multiple myeloma, although there remains limited data on long-term complications associated with this therapy.[358.1] These emerging therapies highlight the evolving landscape of hematologic treatment and the need for continued research to address existing challenges in . In pediatric hematology-oncology, precision treatment has evolved beyond traditional approaches such as cytotoxic chemotherapy, , and , becoming a reality for some pediatric cancers. Regulatory changes have incentivized pharmaceutical companies to include children in research, thereby facilitating access to innovative therapies.[359.1] Among these advancements, gene replacement therapy has emerged as a central strategy in gene therapy, showing great promise in treating certain pediatric cancers. This approach focuses on identifying specific mutated genes in tumors and utilizing viral vectors to deliver a normal, "wild type" version of those genes.[362.1] Furthermore, recent discussions in pediatric oncology highlight five major domains, including reducing toxicity, , , detection and monitoring, and access to care, which collectively aim to improve treatment outcomes for young patients.[361.1]

In this section:

Sources:

Faqs

Common Questions about Hematology

Hematology, as a specialized branch of medicine, addresses various questions and misconceptions related to blood and its disorders. One common inquiry pertains to the nature of hematology itself. Hematology is defined as the study of blood and blood-forming organs, encompassing the diagnosis, treatment, and prevention of diseases affecting blood, bone marrow, and related systems, including immunologic, hemostatic, and vascular systems.[381.1] The field has evolved significantly, with its distinct methodologies and hospital wards emerging between the nineteenth and twentieth centuries, transitioning from being a subset of internal medicine to a specialized discipline.[379.1] Misconceptions about blood disorders are prevalent. For instance, a widespread myth is that only men can have bleeding disorders. While hemophilia is more common in men, women can also be affected, with approximately one-third of female carriers experiencing bleeding symptoms.[385.1] Another misconception is that individuals with bleeding disorders cannot lead normal lives; however, modern treatment options allow for effective management, enabling individuals to live full and healthy lives.[386.1] Public health initiatives play a crucial role in addressing blood-related diseases and enhancing community awareness. Campaigns focused on not only save lives but also promote and raise awareness about blood-related conditions.[390.1] Programs such as the Blood Diseases & Disorders Education Program aim to educate patients and the public about various blood disorders, their management, and the importance of blood donation.[389.1] Additionally, community engagement is vital for promoting health and well-being, as it fosters collaboration among stakeholders to address health-related issues effectively.[392.1] Recent advancements in hematology research have also generated significant interest. Breakthroughs such as the 2023 FDA approvals of new treatments for sickle cell disease highlight the ongoing progress in understanding and managing blood disorders at a molecular level.[393.1] These developments not only enhance treatment options but also contribute to a broader understanding of hematologic conditions.

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Sources:

References

sciencedirect.com favicon

sciencedirect

https://www.sciencedirect.com/topics/medicine-and-dentistry/hematology

[3] Hematology - an overview | ScienceDirect Topics Hematology Introduction. Hematology is the study of blood and blood-forming organs, including the diagnosis, treatment, and prevention of diseases of the blood, bone marrow, and immunologic, hemostatic, and vascular systems. Hematologic analysis is often used for the diagnosis and treatment of animal diseases. As analytical methods have become more sensitive, requiring smaller sample volumes

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wikipedia

https://en.wikipedia.org/wiki/Hematology

[4] Hematology - Wikipedia Hematology (spelled haematology in British English) is the branch of medicine concerned with the study of the cause, prognosis, treatment, and prevention of diseases related to blood. It involves treating diseases that affect the production of blood and its components, such as blood cells, hemoglobin, blood proteins, bone marrow, platelets, blood vessels, spleen, and the mechanism of

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hopkinsmedicine

https://www.hopkinsmedicine.org/health/treatment-tests-and-therapies/hematology

[5] Hematology - Johns Hopkins Medicine Hematology is the study of blood and blood disorders. Hematologists and hematopathologists are highly trained healthcare providers who specialize in diseases of the blood and blood components. Measles Cases on the Rise . Several states are reporting cases of measles, a highly contagious viral disease. Both children and adults can get measles.

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steadyhealth

https://www.steadyhealth.com/articles/the-16-most-common-genetic-diseases

[6] The 16 Most Common Genetic Diseases - SteadyHealth We also know that some genetic disorders, sickle cell anemia (SCA), for example, are an attempt by the body to combat stressors in the environment the person encounters. In sickle cell anemia , the mutation that causes the red blood cells to change shape also helps minimize infection with the malarial parasite, making the condition a remarkable

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scienceofbiogenetics

https://scienceofbiogenetics.com/articles/a-comprehensive-list-of-genetic-disorders-causes-symptoms-and-treatments

[7] List of Genetic Disorders Hemophilia A is an inherited genetic disorder that affects the blood's ability to clot properly. It is caused by abnormalities in the gene responsible for producing a protein called factor VIII, which is crucial for clotting. ... depending on the number of genes affected and the specific mutation involved. Symptoms of thalassemia can include

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clevelandclinic

https://my.clevelandclinic.org/health/diseases/21545-blood-disorders

[8] Blood Disorders: Types, Symptoms & Treatments - Cleveland Clinic Blood Disorders: Types, Symptoms & Treatments Blood Disorders Blood Disorders What are blood disorders? In general, noncancerous blood disorders are conditions that affect your blood cells and platelets and cause issues that may: Paroxysmal nocturnal hemoglobinuria: This rare blood disorder happens when your immune system attacks your red blood cells, increasing your risk of blood clots. Diamond-Blackfan anemia: This inherited disorder keeps your bone marrow from making enough red blood cells. For example, many people with blood clotting disorders may have normal lifespans, but may require medication and treatment for the rest of their lives. Treat any bleeding right away: If you have a bleeding disorder, your healthcare provider may prescribe medication (factor) to help your blood clot.

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doctorsmagazine

https://doctorsmagazine.co/qa/how-to-educate-patients-about-blood-disorders-and-treatment-options/

[9] How to Educate Patients About Blood Disorders and Treatment Options Navigating the complexities of blood disorders and their treatments can be daunting for patients. This article distills expert knowledge into practical tips for clear, accessible patient education. Learn strategies for effective communication, from simplifying medical jargon to personalizing treatment plans. Use Visual Aids for Clear Communication

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ashpublications

https://ashpublications.org/blood/article/134/Supplement_1/5783/425805/Patient-Education-Interventions-for-Improving-Self

[10] Patient Education Interventions for Improving Self-Management in Adults ... Prior studies have demonstrated improved outcomes in chronic illness as a result of empowering patients through various education strategies. This systematic review investigates the current state of patient education for SCD and thalassemia with the goal of elucidating which strategies have been effective in improving patient knowledge and/or

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needle

https://www.needle.tube/resources-14/nursing-interventions-for-hematologic-diseases-vital-signs-medications-education-support-and-collaboration

[12] Nursing Interventions for Hematologic Diseases: Vital Signs ... 3. Educating Patients. Educating patients about their hematologic disease is a key nursing intervention to promote self-management and improve outcomes. Nurses must provide information on the nature of the disease, treatment options, potential complications, and lifestyle modifications to empower patients to take an active role in their care.

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advancementsinhematology

https://advancementsinhematology.com/healthcare-strategy/preventive-strategies-in-hematology-reduce-risks-and-improve-outcomes/

[14] Preventive Strategies in Hematology - Hematology Advancements Comprehensive Preventive Care Strategies. Integrating comprehensive preventive care strategies, including routine check-ups and monitoring, is essential for managing chronic hematologic conditions. For example, regular blood tests and imaging studies can help monitor disease progression in patients with conditions like hemophilia and thalassemia.

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marymount

https://marymount.edu/blog/designing-effective-health-campaigns-strategies-for-public-health-educators/

[16] Designing Effective Health Campaigns: Strategies for Public Health ... Designing effective health campaigns is essential to improving health equity and reducing the systemic barriers in today's communities. Those interested in a career in public health education and promotion will not only need to learn the basics of health communication, but also the advanced strategies required to create compelling and effective public health education campaigns.

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hematology

https://www.hematology.org/newsroom/press-releases/2022/research-highlights-cutting-edge-new-treatments-for-blood-disorders

[18] Research Highlights Cutting-Edge New Treatments for Blood Disorders In an international study – the largest to date of gene addition therapy for an inherited blood disorder – more than 80% of evaluable patients with a severe genetic condition, who had been dependent on regular blood transfusions to stay alive, remained transfusion-free three years after receiving a single infusion of their own blood-forming stem cells that had been altered to correct the genetic mutation that caused their disease. Gene therapy can be an equitable and potentially cost-effective therapeutic option for people in the United States with SCD despite a projected per-patient cost of over $2 million for one-time treatment, according to researchers, who conducted the first study to try to answer this question using a novel approach that factors health disparities into SCD patient care.

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scienceofbiogenetics

https://scienceofbiogenetics.com/articles/genetic-disorders-caused-by-environmental-conditions-unraveling-the-complex-connection

[21] Genetic Disorders: The Impact of Environmental Conditions In addition to genetic factors, environmental conditions can also play a role in the development of genetic and blood disorders. Exposure to certain chemicals, toxins, or medications during pregnancy or throughout life can increase the risk of these conditions.

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winsomepublishing

https://winsomepublishing.org/en/article/the-influence-of-environmental-factors-on-the-development-of-blood-disorders

[22] The Influence of Environmental Factors on The Development of Blood ... Blood disorders represent a diverse group of conditions affecting the components and functions of blood. While genetic predisposition plays a significant role in many cases, accumulating evidence highlights the profound influence of environmental factors in their development and progression.

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nature

https://www.nature.com/articles/s41435-022-00192-6

[24] Gene-environment interactions and their impact on human health Often, genetic and environmental factors contribute to a given disease or phenotype in a non-additive manner, yielding a gene–environment (G × E) interaction. In this work, we broadly review current knowledge on the impact of gene–environment interactions on human health. We next detail well-established G × E interactions that impact human health involving environmental toxicants, pollution, viruses, and sex chromosome composition. Such “Gene by Environment” (G × E) interactions are the focus of this review.

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haematologica

https://haematologica.org/article/view/9472

[39] One hundred years of Haematologica An important scientific step in the history of hematology was the discovery of the hematopoietic function of bone marrow by Ernst Neumann (1834-1918) and Giulio Bizzozero (1846-1901) in 1868.3 Another important development in this field of medicine was the description by Paul Ehrlich (who used the aniline staining technique in his degree

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hematology

https://www.hematology.org/about/history

[40] History of ASH and Hematology Explore the Rich History of ASH and Hematology In April 1958, the first official meeting of the American Society of Hematology (ASH) was held in Atlantic City, New Jersey, where more than 300 hematologists gathered to discuss clinical and research matters related to blood and blood diseases. Since that initial meeting, ASH has played an active

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scienceinfo

https://scienceinfo.net/history-of-hematology.html

[41] History of hematology - Science The history of blood transfusion began in the 1600s when British scientist William Harvey discovered that blood circulated through the heart instead of the lungs. With this knowledge, doctors reason that blood transfusions can help treat many conditions, such as postpartum haemorrhage, even mental illness. The first blood transfusions in

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sciencedirect

https://www.sciencedirect.com/org/science/article/pii/S1592872120003950

[42] One hundred years of Haematologica - ScienceDirect The historical-scientific background Hematology as a separate specialty, with its own methodology and hospital wards, only began to emerge between the nineteenth and the twentieth centuries. Before then, the pathophysiology and the clinical practice of hematologic diseases were mainly considered to be simply a part of internal medicine.

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healthdor

https://healthdor.com/article/the-history-of-blood-in-medical-science-from-1771-to-1917-part-1

[43] The History of Blood in Medical Science from 1771 to 1917. Part 1 In conclusion, the period from 1771 to 1917 witnessed remarkable advancements in the understanding and application of blood in medical science, shaping the landscape of transfusion medicine. The understanding of blood types and their compatibility has evolved significantly during this period, leading to groundbreaking developments in transfusion medicine and patient care. In conclusion, the challenges in preserving and storing blood from 1771 to 1917 were significant, but the historical developments in the understanding and application of blood in medical science have led to remarkable improvements in blood transfusion safety. History of blood circulation discovery Advancements in blood transfusions Impact of bloodletting practices Blood typing and compatibility Understanding blood coagulation Challenges in preserving and storing blood Impact of blood-related discoveries on medicine

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scienceinfo

https://scienceinfo.net/history-of-hematology.html

[45] History of hematology - Science The first blood transfusions in history were also performed around this time, but most of them were unsuccessful, the blood recipient died. British and American doctors also experimented with blood transfusion between animals.

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hematology

https://www.hematology.org/newsroom/press-releases/2024/researchers-report-new-opportunities-to-improve-quality-of-life

[55] Researchers Report New Opportunities to Improve Quality of Life ... The American Society of Hematology (ASH) (hematology.org) is the world's largest professional society of hematologists dedicated to furthering the understanding, diagnosis, treatment, and prevention of disorders affecting the blood.Since 1958, the Society has led the development of hematology as a discipline by promoting research, patient care, education, training, and advocacy in hematology.

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hematology

https://www.hematology.org/newsroom/press-releases/2023/studies-highlight-impacts-of-applying-new-technologies-in-everyday-care

[84] Studies Highlight Impacts of Applying New Technologies in Everyday Care Findings show benefits of AI-driven techniques, chemotherapy-free treatments, and cell therapy (SAN DIEGO, Dec. 9, 2023) - Researchers report striking progress in the application of cutting-edge tools and treatments to address longstanding challenges in blood disorders and related health conditions in four studies presented during the 65th American Society of Hematology (ASH) Annual Meeting

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astctjournal

https://www.astctjournal.org/article/S2666-6367(23

[89] Chimeric Antigen Receptor T-Cell Therapy in the Outpatient Setting: An ... The first series of chimeric antigen receptor T (CAR-T) cell therapy products were approved in 2017 to 2019 and have shown remarkable efficacy in both clinical trials and the real-world setting, but at the cost of prolonged patient hospitalization. As the toxicity management protocols were refined, the concept of cellular therapy administered in the outpatient setting gained steam, and single

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ashpublications

https://ashpublications.org/blood/article/144/Supplement+1/4238/532282/Association-between-Targeted-Therapy-and-Survival

[94] Association between Targeted Therapy and Survival in Patients with AML Conclusion Targeted therapies have an impact on survival in patients exhibiting targetable mutations. Our findings support the notion that incorporating targeted therapies for actionable mutations into treatment regimens can enhance survival outcomes for patients with AML.

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nih

https://pmc.ncbi.nlm.nih.gov/articles/PMC11625413/

[108] Transformative Role of Artificial Intelligence in Reporting Haematology ... This study explores the use of the AI100 (SigTuple Technologies Private Limited, Bangalore, India) automated machine, a smart robotic microscope designed to automate the microscopic analysis of peripheral blood smears. Through the analysis of four haematology cases, this study demonstrates how AI technology facilitates efficient cell identification, enhances risk stratification, enables early detection of abnormalities, and accelerates diagnostic turnaround times. The present study, using the AI100 automated machine, demonstrated significant improvements in diagnostic accuracy, speed, and standardisation across four haematological cases (alpha-thalassaemia trait, AML, eosinophilia, chronic lymphoproliferative disorder). Compared to previous studies, such as El Alaoui et al., which highlighted AI’s potential but emphasised the need for extensive training data , and Acharya et al., which noted the effectiveness of AI in AML detection but faced workflow integration challenges, the present study offered more practical case examples .

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advancementsinhematology

https://advancementsinhematology.com/technology/ai-in-hematology/ai-revolutionizing-hematological-diagnostics-case-studies-and-outcomes/

[114] AI Revolutionizing Hematological Diagnostics - Hematology Advancements AI Revolutionizing Hematological Diagnostics - Hematology Advancements AI Revolutionizing Hematological Diagnostics: Case Studies and Outcomes Artificial intelligence (AI) has made significant inroads into hematology, particularly in diagnostics. This article explores specific case studies demonstrating the transformative impact of AI in hematological diagnostics. One prominent example is the use of AI in flow cytometry, a critical diagnostic tool for hematologic and immunologic disorders. A study validated an AI-assisted flow cytometry workflow using 379 clinical cases, showing that the AI model significantly improved diagnostic accuracy and reduced analysis time to under five minutes per case. AI’s role in enhancing the efficiency of hematological diagnostics cannot be overstated. From improving accuracy and speed in flow cytometry to revolutionizing leukemia diagnostics and enhancing near-patient testing, AI has proven its potential to transform hematology.

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askhematologist

https://askhematologist.com/artificial-intelligence-in-hematology/

[115] Artificial Intelligence in Hematology - Ask Hematologist | Understand ... One of the primary applications of AI in hematology is the automated analysis of blood smears. Among the most promising applications of AI in hematology is the automated analysis of blood smears and other hematological images. AI-based predictive models can analyze a patient’s genetic and clinical data to determine the risk of developing hematological disorders, such as leukemia or anemia. AI algorithms can integrate and analyze a wide range of data, including laboratory test results, patient histories, and treatment outcomes, to provide healthcare providers with evidence-based recommendations for diagnostic tests, therapeutic interventions, and disease management strategies. AI is poised to revolutionize hematology by enhancing diagnostic accuracy, personalizing treatments, and improving workflow efficiency. Artificial Intelligence, Hematology, Blood Smear Analysis, Flow Cytometry, Predictive Analytics, Drug Discovery, Laboratory Automation, Telemedicine, Ethics in AI, Image Analysis, Predictive Modeling, Personalized Treatment, Clinical Decision-Making.

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nih

https://pmc.ncbi.nlm.nih.gov/articles/PMC11578717/

[116] Optimization of diagnosis and treatment of hematological diseases via ... This study summarizes the research progress of various AI-assisted systems applied in the clinical diagnosis and treatment of hematological diseases, with a focus on their application in morphology, immunology, cytogenetics, and molecular biology diagnosis, as well as prognosis prediction and treatment. Using PubMed, Web of Science, and other network search engines, we conducted a literature search on studies from the past 5 years using the main keywords “artificial intelligence” and “hematological diseases.” We classified the clinical applications of AI systems according to the diagnosis and treatment. Here, we review the application of AI in hematology diagnosis and treatment over the past 5 years, with a particular focus on comprehensive typing diagnosis (including morphology, immunology, cytogenetics, and molecular biology) (Figure 1) to clarify the role and limitations of AI in areas such as disease prediction, drug development, risk stratification, and prognosis tracking.

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probiologists

https://probiologists.com/Uploads/Articles/37_638663567116042591.pdf

[117] PDF AI algorithms can also potentially improve the accuracy and eficiency of diagnosis and prognosis, leading to better patient outcomes. However, it is crucial to adopt a critical perspective and carefully evaluate the risks and benefits of AI in hematology to ensure its responsible and ethical implementation.

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myhematology

https://myhematology.com/commentaries/hematology-an-exploration-of-blood/

[125] Hematology: An Exploration of the Blood | My Hematology Blood composition: Hematology delves into the different components of blood, including plasma, red blood cells, white blood cells, and platelets, and their respective functions. Blood cell function : It explores the diverse roles of various white blood cells in immunity, red blood cells in oxygen transport, and platelets in clotting.

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inova

https://healthlibrary.inova.org/BreatheEasy/90,P02316

[126] Inova - Overview of Blood and Blood Components What are the components of blood? The components of human blood are: Plasma. The liquid component of the blood in which the following blood cells are suspended: Red blood cells (erythrocytes). These carry oxygen from the lungs to the rest of the body . White blood cells (leukocytes). These help fight infections and aid in the immune process.

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rochester

https://www.urmc.rochester.edu/encyclopedia/content?ContentTypeID=90&ContentID=P02316

[128] Overview of Blood and Blood Components Often with an infection, the number of white blood cells will be elevated. Many forms of cancer can affect the production of blood cells. For instance, an increase in the immature white blood cells in a CBC can be associated with leukemia. Blood diseases, such as anemia and sickle cell disease, will cause abnormally low hemoglobin. Common blood

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nih

https://pmc.ncbi.nlm.nih.gov/articles/PMC4260297/

[129] Overview of blood components and their preparation - PMC The whole blood which is a mixture of cells, colloids and crystalloids can be separated into different blood components namely packed red blood cell (PRBC) concentrate, platelet concentrate, fresh frozen plasma and cryoprecipitate. Each blood

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nih

https://www.ncbi.nlm.nih.gov/books/NBK534253/

[139] Physiological Haemostasis - Mechanisms of Vascular Disease - NCBI Bookshelf Physiological haemostasis involves complex interactions between endothelial cells, platelets and coagulation proteins, that result in a prompt platelet plug and then localised thrombus formation at the site of a break in vascular integrity. Numerous regulatory processes prevent widespread activation of coagulation, ensuring that blood remains fluid in the absence of vascular injury or other

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clevelandclinic

https://my.clevelandclinic.org/health/symptoms/21999-hemostasis

[141] Hemostasis: What It Is & Stages - Cleveland Clinic Platelets circulating in your blood stick to the damaged tissue to form a temporary plug (primary hemostasis). That clot works like a bottle cork, keeping blood in and debris or germs out. The "coagulation cascade" sequence starts (secondary hemostasis). Coagulation factors in your blood amplify the clotting effects to stabilize the plug.

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healthbenefitstimes

https://www.healthbenefitstimes.com/health-wiki/red-blood-cells/

[142] What is Red Blood Cells (RBC): Their Structure and Function and normal ... Function of Red Blood Cells. Red blood cells (RBCs) play a vital role in maintaining physiological homeostasis by transporting gases throughout the body. They are primarily responsible for delivering oxygen from the lungs to tissues and removing carbon dioxide from tissues to the lungs. Key functions of RBCs include: 1. Oxygen Transport

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biologyinsights

https://biologyinsights.com/red-blood-cells-functions-structure-and-life-cycle/

[143] Red Blood Cells: Functions, Structure, and Life Cycle The balance between red blood cell production and destruction is vital for maintaining homeostasis. Any disruption in this balance can lead to hematological disorders, such as anemia or polycythemia. Anemia results from a deficiency in red blood cell numbers or hemoglobin content, while polycythemia arises from an excessive proliferation of

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nationalgeographic

https://www.nationalgeographic.com/premium/article/medical-breakthroughs-2023-gene-therapy-crispr-cancer-fertility

[162] 7 medical breakthroughs that gave us hope in 2023 - National Geographic Aside from COVID-19 vaccines, there were many other interesting and groundbreaking discoveries made this year, some of which are especially notable for their potential impact on health and medicine. It treats sickle cell disease and beta thalassemia, genetic disorders that affect the red blood cells. The newly approved gene therapy, named CASGEVY, corrects faulty hemoglobin genes in a patient’s bone marrow stem cells so they can produce functioning hemoglobin. A single treatment can potentially cure some patients for life.

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pbs

https://www.pbs.org/newshour/science/a-look-at-the-top-science-stories-and-breakthroughs-of-2023

[163] A look at the top science stories and breakthroughs of 2023 Yes Not now Full Episodes Podcasts Newsletters Live By —Nicola Jones, Knowable Magazine Nicola Jones, Knowable Magazine Leave your feedback Share Copy URL https://www.pbs.org/newshour/science/a-look-at-the-top-science-stories-and-breakthroughs-of-2023 Email Facebook Twitter LinkedIn Pinterest Tumblr Share on Facebook Share on Twitter A look at the top science stories and breakthroughs of 2023 Science Updated on Jan 5, 2024 4:31 PM EST — Published on Dec 31, 2023 9:00 AM EST As 2023 rolls to a close, Knowable Magazine has looked back over its articles and canvassed editorial committee members from the 51 academic journals — covering analytical chemistry to vision science — published by Knowable’s parent company, Annual Reviews. Here we present 12 newsworthy developments from 2023. But 2023 really was a standout: The planet had its hottest year on record. Gene editing In November, the UK medicines regulatory agency became the first in the world to approve a therapy that uses CRISPR gene editing — a revolutionary biotechnology that snips at DNA like a molecular scalpel. The treatment, called Casgevy, helps people with conditions caused by defective hemoglobin production or function, including sickle cell disease.

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hematology

https://www.hematology.org/education/patients/blood-disorders

[164] Blood Disorders - Hematology.org People may be affected by many different types of blood conditions and blood cancers. Common blood disorders include anemia, bleeding disorders such as hemophilia, blood clots, and blood cancers such as leukemia, lymphoma, and myeloma. Talking to your doctor is the first step to take if you believe you may have a blood condition.

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nih

https://www.ncbi.nlm.nih.gov/books/NBK593683/

[166] Techniques for Hematological Disorders - Advances in the Diagnosis and ... Hematologic tests help diagnose diseases of the blood and bone marrow cells, such as anemia, infection, hemophilia, blood-clotting disorders, leukemia, lymphoma, and myeloma. Common hematology tests include the complete blood count (including red blood cells, white blood cells, platelet count, hemoglobin, hematocrit, red blood cell volume, differential white blood count, and other red blood

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bns

https://bns.institute/applied-sciences/blood-composition-in-disease-conditions/

[167] How Blood Composition Varies in Disease Conditions Understanding the variations in blood composition is vital for diagnosing diseases and monitoring health. Blood tests are a cornerstone of medical diagnostics, offering insights into a patient's physiological state.

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wikipedia

https://en.wikipedia.org/wiki/List_of_hematologic_conditions

[169] List of hematologic conditions - Wikipedia An anemia is a decrease in number of red blood cells (RBCs) or less than the normal quantity of hemoglobin in the blood. However, it can include decreased oxygen-binding ability of each hemoglobin molecule due to deformity or lack in numerical development as in some other types of hemoglobin deficiency.[citation needed]Anemia is the most common disorder of the blood.

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webmd

https://www.webmd.com/cancer/lymphoma/blood-disorder-types-and-treatment

[170] Blood Disorder Types, Symptoms, and Treatments - WebMD Blood Disorders Affecting Red Blood Cells Blood Disorders Affecting Red Blood Cells Blood disorders that affect red blood cells include: Anemia: People with anemia have a low number of red blood cells. Aplastic anemia: In people with aplastic anemia, the bone marrow does not produce enough blood cells, including red blood cells. Autoimmune hemolytic anemia: In people with this condition, an overactive immune system destroys the body's own red blood cells, causing anemia. The excess red blood cells usually create no problems but may cause blood clots in some people. Blood disorders that affect white blood cells include: Leukemia: A form of blood cancer in which a white blood cell becomes malignant and multiplies inside bone marrow.

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ashpublications

https://ashpublications.org/hematology/article/2003/1/279/18648/Molecular-Diagnostics

[177] Molecular Diagnostics - American Society of Hematology A huge body of new information on the genetic, genomic and proteomic profiles of different hematopoietic diseases is accumulating. This chapter focuses on new technologies and advancements in understanding the molecular basis of hematologic disorders, providing an overview of new information and its significance to patient care.

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linkedin

https://www.linkedin.com/pulse/breaking-barriers-hematology-10-groundbreaking-advances-reshaping-kvxcc/

[179] Breaking Barriers in Hematology: 10 Groundbreaking Advances ... - LinkedIn Bispecific antibodies have gained traction in blood cancer treatment by simultaneously targeting two different antigens, offering new hope for patients with refractory leukemia and lymphoma.

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ashpublications

https://ashpublications.org/blood/article/145/13/1353/535463/Impact-of-new-medications-on-the-treatment-of

[180] Impact of new medications on the treatment of immune TTP The last decade has seen the introduction of 2 new licensed therapies for thrombotic thrombocytopenic purpura (TTP), caplacizumab and recombinant ADAMTS13 (rADAMTS13), for immune and congenital TTP (cTTP), respectively. They improve acute TTP outcomes, and reduce the need for plasma therapy, time to clinical response, and treatment burden.

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nih

https://pmc.ncbi.nlm.nih.gov/articles/PMC11578717/

[181] Optimization of diagnosis and treatment of hematological diseases via ... This study summarizes the research progress of various AI-assisted systems applied in the clinical diagnosis and treatment of hematological diseases, with a focus on their application in morphology, immunology, cytogenetics, and molecular biology diagnosis, as well as prognosis prediction and treatment. Using PubMed, Web of Science, and other network search engines, we conducted a literature search on studies from the past 5 years using the main keywords “artificial intelligence” and “hematological diseases.” We classified the clinical applications of AI systems according to the diagnosis and treatment. Here, we review the application of AI in hematology diagnosis and treatment over the past 5 years, with a particular focus on comprehensive typing diagnosis (including morphology, immunology, cytogenetics, and molecular biology) (Figure 1) to clarify the role and limitations of AI in areas such as disease prediction, drug development, risk stratification, and prognosis tracking.

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researchgate

https://www.researchgate.net/publication/373434922_REVIEW_OF_ANEMIA_TYPES_AND_CAUSES

[200] REVIEW OF ANEMIA: TYPES AND CAUSES - ResearchGate The major causes of anemia are iron deficiency, foliate deficiency, hookworm infection and malaria. Out of two billion people suffering from anemia, about 50% cases are related to iron deficiency.

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nih

https://www.ncbi.nlm.nih.gov/books/NBK499994/

[203] Anemia - StatPearls - NCBI Bookshelf Anemia - StatPearls - NCBI Bookshelf Anemia Iron deficiency anemia  In the elderly, approximately one-third of patients have a nutritional deficiency as the cause of anemia, such as iron, folate, and vitamin B12 deficiency. 2) Anemia due to nutritional deficiencies: Oral/IV iron, B12, and folate. Macrocytic anemia with B12/folate deficiency: consider in a patient with paresthesias, vegans/vegetarians or in patients with recent gastric bypass surgeries Patients with nutritional anemia due to iron deficiency should be educated on food which is rich in iron. Consider screening for sickle cell and thalassemia in patients with unexplained anemia or with a family history of these diseases. Anemia. Anemia - StatPearls Anemia - StatPearls

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nih

https://www.nhlbi.nih.gov/files/docs/public/blood/anemia-inbrief_yg.pdf

[204] PDF The main goals of treatment are to: n Raise your RBC count or hemoglobin level to im­ prove your blood’s ability to carry oxygen n Treat the underlying condition causing your anemia n Prevent complications of the anemia, such as heart or nerve damage n Relieve symptoms and improve your quality of life If you have a mild or moderate anemia with no symptoms—or if your anemia isn’t getting worse—you may not need treatment. You also can visit the following Web sites to learn more about clinical re­ search and to search for clinical trials: n www.clinicaltrials.gov n clinicalresearch.nih.gov n www.nhlbi.nih.gov/studies/index.htm To Learn More The National Heart, Lung, and Blood Institute (NHLBI) provides information about the causes, pre­ vention, diagnosis, and treatment of anemia and other blood diseases.

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wjoanemia

https://www.wjoanemia.com/doi/WJOA/pdf/10.5005/jp-journals-10065-0024

[205] Clinical Evaluation of Different Types of Anemia Microcytic anemia due to iron deficiency is the most common type of anemia in children. Iron deficiency anemia, which can be associated with cognitive issues, is prevented and treated with iron supplements or increased intake of dietary iron.

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nih

https://www.ncbi.nlm.nih.gov/books/NBK593683/

[219] Techniques for Hematological Disorders Hematologic tests help diagnose diseases of the blood and bone marrow cells, such as anemia, infection, hemophilia, blood-clotting disorders, leukemia, lymphoma, and myeloma. Common hematology tests include the complete blood count (including red blood cells, white blood cells, platelet count, hemoglobin, hematocrit, red blood cell volume, differential white blood count, and other red blood

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missouri

https://prep.missouri.edu/Download_PDFS/browse/4020074/ClinicalHematologyAndFundamentalsOfHemostasis.pdf

[220] PDF The diagnosis and management of hematological malignancies, such as leukemia, lymphoma, and myeloma, require a multidisciplinary approach and expertise in various techniques. Accurate diagnosis relies on morphologic evaluation of bone marrow aspirates and biopsies, immunophenotyping by flow cytometry, cytogenetics, and molecular diagnostics.

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nyweekly

https://nyweekly.com/tech/the-future-of-blood-testing-how-new-technologies-are-revolutionizing-diagnostics/

[223] The Future of Blood Testing: How New Technologies Are Revolutionizing ... Lab-on-a-chip technology miniaturized and integrates laboratory processes into a single chip, significantly reducing the amount of blood required for testing. These devices streamline blood analysis, making it possible to conduct complex tests quickly and efficiently at the point of care, rather than in a distant laboratory.

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nationalacademies

https://nap.nationalacademies.org/read/26941/chapter/9

[224] 7 Techniques for Hematological Disorders | Advances in the Diagnosis ... EMERGING DIAGNOSTIC TECHNIQUES. In the upcoming years breakthroughs in genetic sequencing tests are expected to continue to improve the diagnosis and assessment of hematological disorders. As more genetic syndromes are identified, these tests will continue to become more accurate in their diagnoses.

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physiciansweekly

https://www.physiciansweekly.com/strategies-for-communicating-cancer-diagnoses-effectively-in-hematology/

[227] Strategies for Communicating Cancer Diagnoses Effectively in Hematology Additionally, as telemedicine expands, figuring out how to deliver sensitive news effectively in a digital setting is another pressing area to explore. While delivering bad news will never be easy, having a structured approach helps both the patient and the clinician. Compassionate communication supports the patient and reduces clinician burnout.

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deltapsychology

https://www.deltapsychology.com/articles-for-doctors/effective-doctor-patient-communication-research-backed-techniques-to-improve-outcomes

[228] Effective Doctor-Patient Communication: Research-Backed Techniques to ... Effective doctor‐patient communication is vital to quality care, directly enhancing patient outcomes, adherence, and satisfaction while reducing malpractice risks. ... Avoid unnecessary technical jargon - for example, say "high blood pressure" instead of "hypertension," or "an x-ray picture" instead of "radiograph." If you

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iosrjournals

https://www.iosrjournals.org/iosr-jnhs/papers/vol13-issue5/Ser-2/A1305020108.pdf

[229] PDF Key-Word: Educational interventions, Hematologic diseases, Patient education. ----- Date of submission: 07-09-2024 Date of acceptance: 17-09-2024 ... patients with malignant hematologic disorders, as well as enhancing patient knowledge about medications and their side effects. The study by Cioce et al evaluated the impact of an educational

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nih

https://pmc.ncbi.nlm.nih.gov/articles/PMC6058256/

[230] Principles and theory guiding development and delivery of patient ... Patient education has long been recognized as an important contributor to successful self‐management of a variety of chronic diseases, as well as improved patient outcomes.6, 9, 11, 46, 51 Recent literature in disorders of thrombosis and hemostasis focuses on describing and evaluating teaching strategies and content rather than exploring the

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nih

https://pubmed.ncbi.nlm.nih.gov/35028826/

[233] Multidisciplinary Approach to Older Adults with Hematologic ... Multidisciplinary oncology care has been utilized in solid tumor oncology for decades, initially driven by the need for multi-modality treatment. In this review, we make the case for multidisciplinary oncogeriatric care for older adults with hematologic malignancies in order to best navigate the intersection of aging and blood cancer.

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advancementsinhematology

https://advancementsinhematology.com/healthcare-strategy/operational-efficiency/enhancing-efficiency-and-accuracy-the-role-of-automation-in-hematology-laboratories/

[235] Enhancing Efficiency and Accuracy: The Role of Automation in Hematology ... Enhancing Efficiency and Accuracy: The Role of Automation in Hematology Laboratories - Hematology Advancements Enhancing Efficiency and Accuracy: The Role of Automation in Hematology Laboratories Automation technologies are transforming hematology laboratories, enhancing efficiency and accuracy in diagnostic processes. 1. Improved Efficiency and Speed: Automated hematology analyzers significantly enhance laboratory efficiency by streamlining the entire blood analysis process. The adoption of this automated system has improved efficiency and enabled the laboratory to handle an increasing workload while maintaining high standards of care​ (Abbott MediaRoom)​. Automation is revolutionizing hematology laboratories by enhancing efficiency, accuracy, and data management. As technology continues to advance, automated systems will further elevate the standards of diagnostic medicine, ultimately leading to better patient outcomes and more efficient laboratory operations.

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nih

https://pmc.ncbi.nlm.nih.gov/articles/PMC11625413/

[238] Transformative Role of Artificial Intelligence in Reporting Haematology ... This study explores the use of the AI100 (SigTuple Technologies Private Limited, Bangalore, India) automated machine, a smart robotic microscope designed to automate the microscopic analysis of peripheral blood smears. Through the analysis of four haematology cases, this study demonstrates how AI technology facilitates efficient cell identification, enhances risk stratification, enables early detection of abnormalities, and accelerates diagnostic turnaround times. The present study, using the AI100 automated machine, demonstrated significant improvements in diagnostic accuracy, speed, and standardisation across four haematological cases (alpha-thalassaemia trait, AML, eosinophilia, chronic lymphoproliferative disorder). Compared to previous studies, such as El Alaoui et al., which highlighted AI’s potential but emphasised the need for extensive training data , and Acharya et al., which noted the effectiveness of AI in AML detection but faced workflow integration challenges, the present study offered more practical case examples .

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sciencedirect

https://www.sciencedirect.com/science/article/pii/S1040842815300998

[239] The choice dilemma in chronic hematological conditions: Why choosing is ... Cognitive and emotional factors may influence decision making and risk-and-benefits evaluation of new treatment approaches. ... Possible adaptive strategies and their implications to improve patient participation and adherence to treatment are proposed. ... We argue that adopting a SDM approach to disease management for these hematological

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advancementsinhematology

https://advancementsinhematology.com/healthcare-strategy/practice-development/multidisciplinary-care-teams-in-hematology-a-collaborative-approach/

[244] Multidisciplinary Care Teams in Hematology: A Collaborative Approach This approach ensures that treatment plans are comprehensive and consider all possible aspects of the patient's health. ... The adoption of multidisciplinary care teams in hematology represents a significant advancement in patient care. By combining the expertise of various healthcare professionals, MDTs improve decision-making, enhance

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advancementsinhematology

https://advancementsinhematology.com/clinical-practice/implementing-comprehensive-care-plans-for-hematological-patients/

[245] Comprehensive Hematology Care Plans - Hematology Advancements This multidisciplinary approach resulted in improved patient outcomes, including reduced complications and better overall survival rates (SpringerLink) (SpringerLink) . A Path to Better Outcomes Implementing comprehensive care plans in hematology is essential for providing high-quality, patient-centered care.

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jpmsonline

https://jpmsonline.com/article/490/

[247] Challenges and Benefits of a Multidisciplinary Approach to Treatment in ... In a multidisciplinary approach, a patient-centered care paradigm, healthcare professionals from various specialties get together, form a team, and collaborate to offer their expertise and contribute in real-time, coordinated ways to patient care [].Each member of the multidisciplinary team plays a part in patient care and includes hospitalists, care partners, nurses, pharmacists, case

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sciencedirect

https://www.sciencedirect.com/topics/medicine-and-dentistry/blood-component-therapy

[256] Blood Component Therapy - an overview | ScienceDirect Topics Summary Blood component therapy plays a critical role in the treatment of pediatric hematology and oncology patients and those with critical illness. Product modifications should be based on a firm understanding of the risks and benefits of transfusion.

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oup

https://academic.oup.com/labmed/article/43/6/237/2657653

[257] Blood Components for Hemostasis - Oxford Academic Abstract We present an overview of revised indications and doses for routinely transfused blood components. Targeted blood component therapy is one of the earliest models of personalized medicine, striving to achieve the tenets of the right dose at the right time for the right reason to the right patient. Despite rigorous quality assessment and federally mandated regulatory requirements, blood

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ashpublications

https://ashpublications.org/hematology/article/2013/1/638/20793/Transfusion-guidelines-when-to-transfuse

[259] Transfusion guidelines: when to transfuse | Hematology, ASH Education ... Transfusion of blood and blood components (ie, RBCs, platelets, plasma, and cryoprecipitate) is one of the most common medical procedures performed in the developed world. However, the decision to transfuse or not to transfuse is one of the more complex decisions made by medical practitioners.

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mhmedical

https://hemonc.mhmedical.com/content.aspx?bookid=1802&sectionid=124980639

[260] BLOOD COMPONENT THERAPY | Hematology in Clinical Practice, 5e ... The practice of whole blood transfusion has largely been replaced by component therapy. However, some blood centers are able to offer whole blood or "modified" whole blood (whole blood minus the platelet component) for the treatment of large-volume blood loss. Its use can save time, cost less, and expose the recipient to fewer donors.

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nih

https://pmc.ncbi.nlm.nih.gov/articles/PMC3127320/

[261] Blood component therapy: Which, when and how much - PMC Blood transfusion refers to the perioperative administration of blood and blood components. Adherence to proper indications for blood component therapy is essential because of its potential adverse effects and costs of transfusion. Over the years, the significance of blood components in treating certain diseases or conditions has been recognized.

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ascopubs

https://ascopubs.org/doi/10.1200/JCO.2021.39.15_suppl.e24090

[264] Benefits of blood transfusions in palliative care patients with ... Background: Anemia is highly prevalent in patients with advanced cancer and adversely affects quality of life. There is limited data on the frequency, clinical utility and effectiveness of red blood cell transfusions (RBC), and no randomized controlled trials or clinical practice guidelines on this subject are available.

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physicscore

https://physicscore.com/articles/blood-transfusion-cancer-implications-insights/

[265] Blood Transfusion and Cancer: Risks and Benefits Definition and Purpose: Blood transfusions are used primarily to replace lost components of the blood, which is essential for patients undergoing cancer treatments like chemotherapy. Risks and Benefits : While transfusions can help alleviate symptoms such as anemia, they also carry risks, including adverse reactions and the potential for immune

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nih

https://pmc.ncbi.nlm.nih.gov/articles/PMC4790595/

[266] Impact of Transfusion on Cancer Growth and Outcome - PMC Cancer patients are also often transfused with red blood cell (RBC) concentrates in the context of chemotherapy to keep an acceptable hemoglobin level, and ~15.1% of the blood resources in the USA are allocated to hematology/oncology patients.7 Therefore, it was noted that transfusion of blood components can possibly be related to worse long

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ca

https://www.cdph.ca.gov/Programs/OSPHLD/LFS/CDPH+Document+Library/BloodTransfusionGuideEnglish.pdf

[271] PDF regarding the benefits, risks, and alternatives of transfusion of blood products, including red blood cells, plasma, platelets, or other products, collected from a patient or someone who is not the patient. This material serves as a supplement to the discussions you have with your physician. It is important that you fully understand and

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nih

https://pmc.ncbi.nlm.nih.gov/articles/PMC10247654/

[276] Blood Donation Screening of Transfusion-Transmissible Viral Infection ... Nucleic acid testing (NAT) is used to screen transfusiontransmittable infections (TTIs) in donated blood samples and provide an additional layer of blood safety. In this study, we describe our experience in screening viral TTIs using two formats of

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ashpublications

https://ashpublications.org/blood/article/133/17/1854/275902/Prevention-of-transfusion-transmitted-infections

[277] Prevention of transfusion-transmitted infections | Blood | American ... Blood-transfusion therapy is integral to management of diverse hematological and other diseases. Prevention of transfusion-transmitted (TT) infectious agents (TTIs) remains a key element of blood-transfusion safety. Attributes of TTIs that pose greatest risk to blood safety include an asymptomatic infectious phase in the donor and the ability to persist despite processing and storage 1-3

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fastercapital

https://fastercapital.com/content/Blood-transfusion--Advancements-in-Blood-Transfusion-Technology--What-s-New.html

[279] Blood transfusion: Advancements in Blood Transfusion Technology: What s ... 4. Pathogen Reduction Technology. In the realm of blood transfusion, the advent of Pathogen Reduction Technology (PRT) stands as a sentinel of safety, guarding against the insidious threat of transfusion-transmitted infections. This innovative approach is not merely a filter but a proactive warrior, wielding a spectrum of light to dismantle the very DNA or RNA of pathogens that may lurk within

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hematology

https://www.hematology.org/about/history/50-years/transfusion-medicine-advances

[289] Advances in Transfusion Medicine - Hematology.org Another of the most significant issues complicating transfusion safety has been bacterial contamination of blood products, particularly of platelets. However, steps have been taken in the last decade to avoid, detect, and eliminate this complication through improved donor selection, specialized preparation of the arm before needle insertion

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nih

https://pmc.ncbi.nlm.nih.gov/articles/PMC9623141/

[290] Ways To Enhance Blood Transfusion Safety: A Systematic Review Study data in the electronic databases, including PubMed/MEDLINE, EMBASE, Web of Science, Cochrane CENTRAL, Scopus, and Google Scholar, from the last 30 years were systematically searched using the following keywords: patient safety, blood transfusion, risk management, safety management, and transfusion reaction. The remaining studies (n \= 6) implemented different interventions for improving transfusion safety, such as the platelet pathogen inactivation (Amato et al., 2017), restrictive transfusion (de Gast-Bakker et al., 2013; Deleon et al., 2016), the Peri-operative Transfusion Trigger Score of Emergency (Potts-E) (Liu et al., 2015), and the hemoglobin-based oxygen carrier-201 (HBOC-201) as an alternative method to safely reduce and/or eliminate perioperative transfusion and ultimately the early use of fresh, whole blood in a resource-limited setting (Auten et al., 2015).

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nih

https://pmc.ncbi.nlm.nih.gov/articles/PMC1306495/

[298] Overview of Marrow Transplantation - PMC Abstract. Bone marrow transplantation is now an accepted form of therapy for many hematologic disorders including aplastic anemia, genetically determined diseases and malignant diseases, particularly leukemia, and for rescue of patients given intensive chemoradiotherapy for malignant disease.

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https://www.sciencedirect.com/topics/immunology-and-microbiology/bone-marrow-transplantation

[299] Bone Marrow Transplantation - an overview - ScienceDirect Introduction. Bone marrow transplantation (BMT) is a widely accepted treatment for many hematologic malignancies, including leukemia, lymphoma, and multiple myeloma. Current bone marrow (BM) from HLA-matched unrelated donors has been a major alternative graft source .However, many patients are unable to proceed to an allogeneic transplantation because of the lack of a suitable BM donor.

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mayoclinic

https://connect.mayoclinic.org/blog/hematology/tab/bmt/

[300] Hematology: BMT - Mayo Clinic Connect A bone marrow transplant is also called a stem cell transplant. A bone marrow transplant may be necessary if your bone marrow stops working and doesn't produce enough healthy blood cells. Bone marrow transplants may use cells from your own body (autologous transplant) or from a donor (allogeneic transplant). The Bone Marrow Transplant Group at

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lifecancercenter

https://www.lifecancercenter.com/psychological-effects-after-bone-marrow-transplantation/

[309] Psychological Effects after Bone Marrow Transplantation Undergoing a bone marrow transplantation could be an anxiety-provoking experience. Patients may worry about the success of the transplant, potential complications, or their long-term prognosis. ... The physical and emotional challenges associated with bone marrow transplantation can contribute to feelings of sadness, hopelessness, or depression

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nih

https://pmc.ncbi.nlm.nih.gov/articles/PMC6482095/

[311] Distress and Quality of Life in Patient and Caregiver Dyads Facing Stem ... Patients undergoing transplant face significant physical and emotional challenges ... Scheide T, Baveja S, Weiland R, Galvin J, Mehta J, and Penedo FJ, "Pre-transplant emotional support is associated with longer survival after allogeneic hematopoietic stem cell transplantation," Bone Marrow Transplant, vol. 51, no. 12, pp. 1594-1598

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nih

https://www.ncbi.nlm.nih.gov/books/NBK608270/

[314] Donor Selection for Adults and Pediatrics - The EBMT Handbook - NCBI ... The eligibility criteria for HCT in malignant diseases vary overtime, resulting from the balance between the outcome of frontline and relapse chemotherapy protocols and the outcome of transplantation, which partially depends on the degree of compatibility within each donor-recipient pair.

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nih

https://www.ncbi.nlm.nih.gov/books/NBK536951/

[315] Hematopoietic Stem Cell Transplantation - StatPearls - NCBI Bookshelf Systematic and retrospective studies have suggested an improved outcome with HPSCT in acquired aplastic anemia compared to conventional immunosuppressive therapy. In a study of 1886 patients with acquired aplastic anemia, transplanted cells collected from the bone marrow produced superior outcomes compared to those collected from the peripheral blood. Patients with aplastic anemia need a preparative regimen, as they still can develop immune rejection to the graft. Haploidentical stem cell transplantation involves administering bone marrow products from a first-degree related haplotype-mismatched donor. This helps underserved patients without broad access to resources as they have fewer chances of having a matched unrelated donor. The advantages of this method include lower cost and rapid availability of hematopoietic cell products.

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nih

https://www.ncbi.nlm.nih.gov/books/NBK536951/

[318] Hematopoietic Stem Cell Transplantation - StatPearls - NCBI Bookshelf Systematic and retrospective studies have suggested an improved outcome with HPSCT in acquired aplastic anemia compared to conventional immunosuppressive therapy. In a study of 1886 patients with acquired aplastic anemia, transplanted cells collected from the bone marrow produced superior outcomes compared to those collected from the peripheral blood. Patients with aplastic anemia need a preparative regimen, as they still can develop immune rejection to the graft. Haploidentical stem cell transplantation involves administering bone marrow products from a first-degree related haplotype-mismatched donor. This helps underserved patients without broad access to resources as they have fewer chances of having a matched unrelated donor. The advantages of this method include lower cost and rapid availability of hematopoietic cell products.

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arogyajivan

https://arogyajivan.com/Transplants/Blog/success-rates-outcomes-bone-marrow-transplants

[319] Success Rates and Outcomes of Bone Marrow Transplants Several factors influence the success rates of bone marrow transplants, including the patient's age, overall health, and the type of disease being treated. The matching of the donor's stem cells to the recipient's genetic markers ( HLA matching ) is crucial to prevent complications.

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biologyinsights

https://biologyinsights.com/stem-cell-transplant-death-rate-factors-and-outcomes/

[320] Stem Cell Transplant Death Rate: Factors and Outcomes Explore the factors influencing stem cell transplant mortality rates, including treatment approaches, complications, and patient-specific considerations. ... SAA patients receiving matched sibling donor grafts experience some of the highest survival rates, often exceeding 80% at five years, according to EBMT. ... Studies in Bone Marrow

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nmdp

https://www.nmdp.org/patients/transplant-support/patient-support-center

[321] Patient Support Center for Transplant Recipients | NMDP NMDP's Patient Support Center connects bone marrow/blood stem cell transplant recipients & families to free emotional, financial and educational resources.

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nbmtlink

https://www.nbmtlink.org/

[322] National Bone Marrow Transplant LINK (nbmtLINK) | Bone Marrow Transplant National Bone Marrow Transplant Link (nbmtLINK) has supported patients, caregivers and healthcare professionals, providing psychosocial and educational support as it relates to the bone marrow/stem cell/CAR-T Cellular Therapy journey.

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rn-journal

https://rn-journal.com/journal-of-nursing/distress-and-depression-among-bone-and-marrow-transplant-patients

[326] Distress and Depression Among Bone and Marrow Transplant Patients Bone and marrow transplant treatment is very aggressive that creates significant physical, social, psychological, and emotional stress. ... Patients scoring a problematic social support prior to stem cell transplant showed a correspondence with poorer survival (Frick et al., 2005) Patients with a high level of perceived social support had

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lifecancercenter

https://www.lifecancercenter.com/psychological-effects-after-bone-marrow-transplantation/

[327] Psychological Effects after Bone Marrow Transplantation The psychological effects of bone marrow transplantation are as essential to address as the physical aspects of recovery. By acknowledging these challenges and seeking help, patients can better navigate the emotional terrain accompanying this life-changing experience.

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nih

https://pubmed.ncbi.nlm.nih.gov/12080358/

[328] Assessment of psychological distress in prospective bone marrow ... Patient psychological distress is associated with many aspects of the bone marrow transplantation (BMT) process and has been linked with poor treatment outcomes. We assessed psychological distress in potential BMT candidates, and compared patient and nurse coordinator ratings of emotional distress at the time of initial BMT consultation.

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sciencedaily

https://www.sciencedaily.com/releases/2024/09/240902111756.htm

[335] Blood stem cell breakthrough could transform bone marrow transplants Blood stem cell breakthrough could transform bone marrow transplants | ScienceDaily Melbourne researchers have made a world first breakthrough into creating blood stem cells that closely resemble those in the human body. MCRI Associate Professor Elizabeth Ng said the team had made a significant discovery in human blood stem cell development, paving the way for these lab grown cells to be used in blood stem cell and bone marrow transplants. "Prior to this study, developing human blood stem cells in the lab that were capable of being transplanted into an animal model of bone marrow failure to make healthy blood cells had not been achievable. "Blood stem cell breakthrough could transform bone marrow transplants." ScienceDaily. "Blood stem cell breakthrough could transform bone marrow transplants." ScienceDaily.

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tau

https://english.tau.ac.il/research/stem-cell-boost

[336] Can Stem Cells Restore Bone Marrow? | Tel Aviv University | Tel Aviv ... An international research team, led by scientists from Tel Aviv University and Sheba Medical Center, has unveiled an innovative method for activating adult stem cells from human bone marrow, enabling their expansion outside the body for use in bone marrow regeneration and the construction of a new blood and immune system.

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nwiot

https://nwiot.org/types-of-blood-cancer-what-are-the-differences-between-leukemia-and-lymphoma/

[342] Blood Cancer 101: Leukemia vs Lymphoma - Know the Difference Continued research efforts are necessary to further elucidate the underlying mechanisms of this complex disease and to enhance patient care. ... The biological behavior of leukemia and lymphoma also differs; Leukemia tends to disseminate rapidly throughout the bloodstream, whereas lymphoma often presents with localized disease, which may spread

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nih

https://pubmed.ncbi.nlm.nih.gov/15055306/

[343] Biological models for leukaemia and lymphoma - PubMed Blood-cell cancers (leukaemias, lymphomas and myeloma) are a very diverse group of neoplasms derived from a variety of stem cells at different hierarchical levels of haemopoietic and lymphoid cell development. This biological heterogeneity is likely to be associated with a variety of different etiological mechanisms.

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hcgoncology

https://www.hcgoncology.com/blog/leukemia-vs-lymphoma/

[344] Leukemia Vs Lymphoma - What Is the Difference? | HCG The difference between leukemia and lymphoma is that leukemia primarily impacts the bone marrow and causes the overproduction of abnormal white cells. Lymphoma, on the other hand, starts in the lymphatic system, causing abnormal cell clusters, forming primarily within lymph nodes and other lymphatic tissues.

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frontiersin

https://www.frontiersin.org/journals/pediatrics/articles/10.3389/fped.2023.1303561/full

[346] Editorial: Diagnosis and therapy pediatric hematological malignancies ... Hematological malignancies are the most frequent cancer in the pediatric population. Leukemia represents approximately 28% of cancer diagnosis (acute lymphoblastic leukemia being the most common type of cancer), while lymphomas account for 9% (third after brain tumors) . In recent decades, the overall survival (OS) rate for childhood

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https://medlineplus.gov/ency/patientinstructions/000845.htm

[349] How childhood cancers are different from adult cancers - MedlinePlus Childhood cancers are not the same as adult cancers. The type of cancer, how far it spreads, and how it is treated is often different from adult cancers. ... One big difference is that the chance of recovery is high in children. Most children with cancer can be cured. ... Rachel Offenbacher, MD, Pediatric Hematology & Oncology, The Children's

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dana-farber

https://blog.dana-farber.org/insight/2018/09/difference-cancer-adults-cancer-children/

[350] What is the Difference Between Cancer in Adults and Cancer in Children ... At a basic, genetic level as well, pediatric cancers display differences from adult cancers. Childhood cancer genomes (the entire collection of genes within a cell) often have few genetic mutations—as few as 10, in some cases, many of which are rare or absent in the cells of adult cancers—studies have found.

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cancernetwork

https://www.cancernetwork.com/view/key-takeaways-from-ash-2023-emerging-trends-in-hematologic-oncology

[351] Key Takeaways From ASH 2023: Emerging Trends in Hematologic Oncology There have been several breakthroughs in nonmalignant hematology care recently, including the 2023 FDA approvals of Casgevy and Lyfgenia for the treatment of patients with sickle cell disease. 1 During the 65th American Society of Hematology (ASH) Annual Meeting and Exposition, results from the phase 2/3 CLIMB SCD-121 trial (NCT03745287) using exagamglogene autotemcel (exa-cel) were presented

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medpace

https://www.medpace.com/blog/emerging-trends-in-hematology-ash-2023-highlights/

[352] Emerging Trends in Hematology - ASH 2023 Highlights - Medpace Corporate Over 75% of subjects who had failed prior FDA approved therapy demonstrated response in the randomized Phase 2 study of Axatilimab  with a median failure free survival of 17 months, offering new hope to patients with debilitating chronic GVHD.6 Finally, the role of the microbiome in graft versus host disease (GVHD) is a new avenue of clinical and translational research in patients post transplant with new explorations of the role of the microbiota composition in the alloreactive T cell response in GVHD.7

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nature

https://www.nature.com/articles/s41392-023-01521-5

[353] Immunotherapy in hematologic malignancies: achievements, challenges and ... Several categories of immunotherapies have been developed and are being further evaluated in clinical trials for the treatment of blood cancers, including stem cell transplantation, immune checkpoint inhibitors, antigen-targeted antibodies, antibody-drug conjugates, tumor vaccines, and adoptive cell therapies. ACT is the most popular immunotherapy for patients with R/R hematologic malignancies, such as TCR-T (T cell receptor-engineered T) cell, γ/δ-T (gamma/delta T) cell, NK (nature killer) cell and CAR-NK (chimeric antigen receptor nature killer) cell and especially CAR-T (chimeric antigen receptor T) cell therapy.33,34,35 Fred Hutchison Cancer Institute used CAR-T cells for the first time to treat B-cell lymphoma and proved its safety in the year 2008.

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nih

https://pubmed.ncbi.nlm.nih.gov/31622496/

[354] CAR T-Cell Therapy in Hematologic Malignancies: A Voyage in Progress The development of chimeric antigen receptor (CAR) T-cell therapy for select hematological malignancies represents one of the most remarkable therapeutic advances in the past decade. Currently, CD19-targeted CAR T-cell therapy is approved for relapsed/refractory diffuse large B-cell lymphoma and acute lymphoblastic leukemia.

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biomedcentral

https://ehoonline.biomedcentral.com/articles/10.1186/s40164-024-00536-0

[355] CAR-T cell combination therapies in hematologic malignancies The principle of chimeric antigen receptor (CAR) T-cell therapy is to genetically modify T cells to recognize specific unique targets on tumor surfaces and exert cytotoxic effects .CAR-T cell therapy has been highly successful in treating various hematologic malignancies and solid tumors .The complete remission rate (CRR) in diffuse large B-cell lymphoma (DLBCL) is 43%, while

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https://pubmed.ncbi.nlm.nih.gov/34445701/

[356] CAR T-Cell Therapy in Hematological Malignancies - PubMed Chimeric antigen receptor (CAR) T-cells (CAR T-cells) are a promising therapeutic approach in treating hematological malignancies. CAR T-cells represent engineered autologous T-cells, expressing a synthetic CAR, targeting tumor-associated antigens (TAAs) independent of major histocompatibility compl …

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biomedcentral

https://jhoonline.biomedcentral.com/articles/10.1186/s13045-025-01676-4

[358] Second primary malignancies following CAR T-cell therapy in patients ... Chimeric antigen receptor T-cell (CAR-T) therapy has transformed the management of patients with relapsed/refractory (R/R) hematologic malignancies, including B-cell lymphomas and multiple myeloma (MM). While data pertaining to the efficacy and toxicity associated with CAR-T have been widely reported, there are limited data on long-term complications. We retrospectively analyzed 246 patients

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nih

https://pmc.ncbi.nlm.nih.gov/articles/PMC10529891/

[359] Children's Oncology Group's 2023 Blueprint For Research Precision treatment, beyond traditional cytotoxic chemotherapy, radiation, and surgery, has become a reality for some pediatric cancers. Regulatory changes have provided significant incentives to pharmaceutical companies to include children in clinical trial research with innovative therapies.

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nih

https://pubmed.ncbi.nlm.nih.gov/37920939/

[361] Innovations in Cancer Treatment of Children - PubMed 2023 Dec 1;152(6):e2023061539. doi: 10.1542/peds.2023-061539. Authors ... 3 Dana-Farber/Boston Children's Cancer and Blood Disorders Center ... cancer survivors. In this State-of-The-Art review, we discuss 5 major domains in pediatric oncology: reducing toxicity, cancer biology, novel therapies, detection and monitoring, and access to care, to

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nih

https://pmc.ncbi.nlm.nih.gov/articles/PMC11149429/

[362] Advances and challenges in gene therapy strategies for pediatric cancer ... Gene replacement therapy has emerged as a central strategy in the field of gene therapy and has shown great promise in the treatment of certain pediatric cancers (Pearl et al., 2023). The thrust of this approach is to identify a particular mutated gene in a tumor and use viral vectors to deliver a normal, "wild type" version of that gene to

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ashpublications

https://ashpublications.org/blood/article/142/Supplement+1/449/501959/Impact-of-Mutations-in-Blood-Cancer-Related-Genes

[371] Impact of Mutations in Blood Cancer-Related Genes on Clinical Outcomes ... RUNX1 and IKZF1 mutations, which are associated with progression to accelerated or blast phase in CML, were very rare in this pt population, supporting their role in CML disease progression. The high frequency of co-occurrence of BCR:: ABL1 mutations and blood cancer gene mutations in CML-CP is an important finding since cancer gene mutations

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nih

https://pmc.ncbi.nlm.nih.gov/articles/PMC7362903/

[372] Germline Mutations: Many Roles in Leukemogenesis - PMC As such, germline mutations represent a clinical challenge in the treatment and management of hematological malignancies. Continued research in the realm of germline mutations' contribution to leukemogenesis will advance preemptive testing and treatment options for those with germline mutations associated with leukemia and lymphoma.

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ashpublications

https://ashpublications.org/thehematologist/article/doi/10.1182/hem.V15.1.8121/463022/Lighting-the-Path-to-Personalized-Medicine-for

[373] Lighting the Path to Personalized Medicine for Hematologic Malignancies ... The drive toward personalized medicine is predicated on the belief that the most effective treatment strategy can be reliably selected for an individual based on precise diagnostic and prognostic information. Currently though, for diseases such as acute leukemia, lymphoma, and myeloma, we typically practice "group medicine," where we assign individuals to prognostic groups that dictate

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nih

https://pmc.ncbi.nlm.nih.gov/articles/PMC6939507/

[379] One hundred years of Haematologica - PMC - PubMed Central (PMC) The historical-scientific background. Hematology as a separate specialty, with its own methodology and hospital wards, only began to emerge between the nineteenth and the twentieth centuries. Before then, the pathophysiology and the clinical practice of hematologic diseases were mainly considered to be simply a part of internal medicine.

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sciencedirect

https://www.sciencedirect.com/topics/medicine-and-dentistry/hematology

[381] Hematology - an overview | ScienceDirect Topics Hematology Introduction. Hematology is the study of blood and blood-forming organs, including the diagnosis, treatment, and prevention of diseases of the blood, bone marrow, and immunologic, hemostatic, and vascular systems. Hematologic analysis is often used for the diagnosis and treatment of animal diseases. As analytical methods have become more sensitive, requiring smaller sample volumes

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hemaware

https://hemaware.org/mind-body/what-10-myths-and-facts-about-hemophilia

[385] Hemophilia Facts and Myths - HemAware Myth: Only men have bleeding disorders. Fact: Although hemophilia is much more common in men, women can have it. About 1/3 of women who are carriers of hemophilia experience bleeding symptoms. In addition, as many women as men have von Willebrand disease, another type of bleeding disorder. Myth: It's possible to outgrow hemophilia.

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thechc

https://www.thechc.com/chc-in-the-new/bleeding-disorder-myth-busters-w-dr-wong/

[386] Bleeding Disorder Myth Busters w/ Dr. Wong - CHC - The Cancer ... However, modern treatment options allow for effective management, reducing the risk of severe bleeding episodes. A bleeding disorder is not a life-limiting condition, and with proper care, individuals can live full and healthy lives. Can you donate blood if you have a bleeding disorder? No, people with bleeding disorders cannot donate blood.

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nih

https://www.nhlbi.nih.gov/education/blood

[389] Blood Diseases and Disorders Education Program - NHLBI, NIH The Blood Diseases & Disorders Education Program offers resources for health and community professionals to educate patients, their caregivers, and the public about blood diseases, blood disorders, blood donation and safety.

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gauthmath

https://www.gauthmath.com/knowledge/How-can-blood-donation-campaigns-improve-public-well-being--7408204021180825613

[390] How can blood donation campaigns improve public well-being? Blood donation campaigns are essential initiatives that contribute significantly to improving public well-being. By ensuring a readily available supply of blood for patients in need, these campaigns not only save lives but also promote community engagement and raise awareness about blood-related conditions.

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who

https://www.who.int/news-room/feature-stories/detail/donors-making-a-difference--community-engagement-to-promote--provide-and-protect-the-health-and-well-being-of-all

[392] Donors making a difference: community engagement to promote, provide ... A process of developing relationships that enable stakeholders to work together to address health-related issues and promote well-being to achieve positive health impact and outcomes. WHO's partners and donors support the Organization to work in this area as there are undeniable benefits to engaging communities in promoting health and well-being. At its core, community engagement enables

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cancernetwork

https://www.cancernetwork.com/view/key-takeaways-from-ash-2023-emerging-trends-in-hematologic-oncology

[393] Key Takeaways From ASH 2023: Emerging Trends in Hematologic Oncology There have been several breakthroughs in nonmalignant hematology care recently, including the 2023 FDA approvals of Casgevy and Lyfgenia for the treatment of patients with sickle cell disease. 1 During the 65th American Society of Hematology (ASH) Annual Meeting and Exposition, results from the phase 2/3 CLIMB SCD-121 trial (NCT03745287) using exagamglogene autotemcel (exa-cel) were presented