Cyclosporin A in myelodysplastic syndrome: a preliminary report.

Therapeutic approaches are not well established in patients with myelodysplastic syndrome (MDS). We evaluated response to cyclosporin A (CyA) in 19 cases with MDS who were enrolled for the study [13 refractory anemia (RA), 5 refractory anemia with excess of blasts (RAEB), and 1 refractory anemia with ringed sideroblasts (RARS)]. Bone marrow was normocellular in ten, hypercellular in five, and hypocellular in four cases. Fifteen patients were transfusion dependent and the rest were not transfusion dependent but with a hemoglobin range of 6.4-8.8 g% with a mean of 7.4 g%. CyA was given at a dose of 3-5 mg/kg per day. A major response was observed in seven patients with RA, which was sustained on follow-up. Four cases of RA showed minor response and two cases of RA did not respond to CyA therapy. A minor response was also seen in one RAEB and one RARS case, while one RAEB case that initially showed a major response relapsed on therapy. The first effect of therapy was evident after a mean period of 2.5 months. A rise in platelets and leukocyte count was seen in three and two cases, respectively. One case developed renal failure on therapy and later died of septicemia. Response to CyA was independent of bone marrow cellularity. CyA could be an effective mode of therapy in patients with MDS especially those having RA.