Peripheral blood stem cell transplant (PBSCT) in a large series of patients with poems syndrome

Abstract

Background: The POEMS syndrome is characterized by peripheral neuropathy (PN), clonal plasma cell disorder (PCD), organomegaly, endocrinopathy, skin changes, edema, sclerotic bone lesions, and thrombocytosis. Based on the improved response rates observed with PBSCT in patients with other PCD, autologous PBSCT may be an attractive treatment option for patients with POEMS syndrome. Methods: Between March 1999 and February 2003, 11 patients with POEMS syndrome underwent PBSCT at Mayo Clinic Rochester and Jacksonville. Conditioning regimens were single agent melphalan (n = 9) and BEAM (n = 1). All received a minimum of 4.4 × 106 CD34 autologous PBSC/kg. Standard supportive care with prophylactic antibiotics and growth factor support was provided. Results: All but one had a severe rapidly progressive sensorimotor PN involving both upper and lower extremities, 7 wheelchair confined. One patient had sensory neuropathy without significant motor involvement. All but one was male. A monoclonal lambda PCD was documented in all patients, and two had biopsy proven Castleman Disease. Median age was 50 years (range 19–62). Seven had organomegaly (5 splenomegaly; 2 hepatomegaly; and 3 lymphadenopathy). Both endocrinopathy and skin changes were present in 9 patients. Seven had thrombocytosis and/or erythrocytosis, and 8 had sclerotic bone lesions (diffuse in 5, solitary lesion in 3). Using the Bardwick Criteria, the median number of POEMS features was 5 (range 2–5). The median number of therapeutic regimens prior to PBSCT was 3 (range 0–6). From first symptoms and from diagnosis of POEMS the median times to transplant were 27 and 10 months (ranges 4–180 and 2–180), respectively. All but one patient had significantly abnormal pre-transplant pulmonary function tests. Of the 8 evaluable patients, all have had a hematologic response and 7 have had neurologic improvement at a median follow-up of 6 months. Other symptoms including fatigue, organomegaly, pulmonary compromise, hyperpigmentation and extravascular volume overload have improved substantially in affected patients. Since February 2003, an additional 4 patients have been transplanted, but are not yet evaluable. Conclusions: PBSCT for POEMS syndrome may result in a high hematologic response rate and improvement in peripheral neuropathy and systemic symptoms. The outcome of these patients will be updated at the meeting.