ABSTRACT
Allogeneic bone marrow transplantation (BMT) is the only treatment modality that has consistently been demonstrated to cure patients with the myelodysplastic syndromes (MDS). Since the early 1980s, numerous publications have reported the results with BMT for over 700 patients with MDS and the lead patients are now disease-free for more than 16 years. Overall, these studies show that approximately 40% of patients are likely to be cured with allogeneic BMT. The best results have been reported for patients with refractory anemia who receive marrow from fully matched related donors, with 75% long-term disease-free survival rates. Factors that are associated with an increased risk of relapse and, thereby, shorter disease-free survival include increased blast percentage and poor-risk karyotype. Factors that are associated with an increased risk of nonrelapse mortality and, in some studies, shorter disease-free survival include longer disease duration, advanced patient age, therapy-related MDS, male patients, and use of mismatched or unrelated donors. However, favorable results have been seen in small studies of patients 55-66 years of age and of patients with refractory anemia undergoing matched unrelated donor BMT. In the opinion of this author, allogeneic BMT is appropriate therapy for patients with high-or intermediate-risk dis-
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ease (risk category based on the International Prognostic Scoring System). The use of allogeneic BMT for patients with low-risk disease is not well defined, but may be appropriate for particularly young individuals or those with a lifethreatening single cytopenia. The use of nonmyeloablative preparative regimens for allogeneic BMT is currently undergoing investigation, but results are too preliminary to make definitive recommendation for use outside of clinical trials.
