Transplant strategies for myelodysplastic syndrome

Abstract

Myelodysplastic syndrome (MDS) is an acquired bone marrow disorder characterized by ineffective hematopoiesis and cellular dysfunction and has an increased risk of transforming into acute myeloid leukemia. Most patients are of advanced age with attendant comorbidities, making treatment difficult. Current treatment options have included supportive care and, in difficult cases, chemotherapy regimens designed for acute leukemia patients. A major effort has been made to determine the role of stem cell transplantation in adult MDS patients, currently the only curative option available for them. Based on relapse rates, studies indicate that allogeneic and autologous transplants provide better antileukemic activity than intensive chemotherapy schedules. Use of DNA methyltransferase inhibitors may assist in managing MDS patients while awaiting a transplant match, but the procedural mortality for transplant remains high. Reduced conditioning or nonmyeloablative conditioning, particularly in the elderly, has been attempted with some success. Reduced conditioning also increases the graft-versus-leukemia effect, allowing for a higher percentage of disease-free survival. Current use of peripheral blood as a source of stem cells for autotransplant is associated with an extremely low procedural mortality. Improvement in such transplant procedures as myeloablation, preparation of the autograft, and posttransplant prophylaxis are improving recovery rates for these patients. In addition, as the biology of this disease is being revealed, newer options will become available in the near future.