Survival and complications in patients with thalassemia major treated with transfusion and deferoxamine.

TLDR

Data from seven Italian centers on 1,697 thalassemia major patients were analyzed to assess survival, causes of death, and complication incidence, examining the effects of gender, birth cohort, ferritin levels, and time period on outcomes. Patients born more recently and females had significantly better survival, with 68 % alive at 35 years, while 67 % of deaths were cardiac; lower ferritin (<1,000 ng/mL) correlated with reduced heart‑failure risk and longer survival, and overall complication rates included heart failure (6.8 %), arrhythmia (5.7 %), hypogonadism (54.7 %), hypothyroidism (10.8 %), diabetes (6.4 %), HIV (1.7 %), and thrombosis (1.1 %).

Abstract

Seven Italian centers reported data on survival, causes of death and appearance of complications in patients with thalassemia major. The interactions between gender, birth cohort, complications, and ferritin on survival and complications were analyzed.Survival after the first decade was studied for 977 patients born since 1960 whereas survival since birth and complication appearance was studied for the 720 patients born after 1970. Better survival was demonstrated for patients born in more recent years (p<0.00005) and for females (p=0.0003); 68% of the patients are alive at the age of 35 years. In the entire population 67% of the deaths were due to heart disease.There was a significant association between birth cohort and complication-free survival (p<0.0005). The prevalence of complications was: heart failure 6.8%, arrhythmia 5.7%, hypogonadism 54.7%, hypothyroidism 10.8%, diabetes 6.4%, HIV infection 1.7%, and thrombosis 1.1%. Lower ferritin levels were associated with a lower probability of heart failure (hazard ratio =3.35, p<0.005) and with prolonged survival (hazard ratio = 2.45, p<0.005), using a cut-off as low as 1,000 ng/mL.Survival and complication-free survival of patients with thalassemia major continue to improve, especially for female patients born shortly before or after the availability of iron chelation.