Management of myelodysplastic syndromes

The treatment of myelodysplastic syndromes is reviewed, with emphasis on recently published clinical trials. Pyridoxine is rarely effective, but a trial in patients with refractory anemia with ringed sideroblasts is justifiable. Corticosteroids do not appear indicated unless in vitro data suggest response. Androgens are generally not beneficial, although danazol merits further evaluation. Both 13-cis-retinoic acid and low-dose cytosine arabinoside have considerable toxicity and yield short-lived partial responses that may not have a significant impact on survival. Combination chemotherapy may be considered in selected patients with refractory anemia with excess of blasts, refractory anemia with excess of blasts in transformation, and chronic myelomonocytic leukemia; however, in general, its toxicity outweighs potential benefit. For unusual patients under 30 years old, bone marrow transplantation should be considered as first-line therapy. Until more effective and less toxic agents are available, supportive care may still be the most appropriate therapy for many of these generally elderly patients.