Objective: To evaluate the efficacy of cyclosporin A (CsA) in treatment of myelodysplastic syndromes (MDS).
Methods: Thirty-three patients with MDS, including refractory anemia (RA, n = 24), refractory anemia with ringed sideroblasts (RAS, n = 2), and refractory anemia with excess blasts (RAEB, n = 7), 23 males and 10 females, aged 46 (6 approximately 71), hospitalized in 4 CsA, grade 3 hospitals in Beijing who failed to respond to folic acid and vitamin B12, received CsA 3 approximately 5 mg x kg(-1)x d(-1), 2 times per days, taken orally in 2 separate doses for at least 3 months (2 approximately 27 months). During the course of treatment the dosage was adjusted according to the CsA blood concentration and curative effect. Follow-up was conducted for 14 months on average. The curative effect of those who took CSA for more than 2 months underwent statistical analysis, and for those who took CSA for less than 2 months only the side effects underwent statistical analysis.
Results: After 3 months of the treatment, the hematological improvement (HI) was observed in 18 of the 32 evaluative patients (56.3%) by the criteria of the International Working Group (IWG). At the end of the follow-up 4 patients showed alteration of disease progression, including 1 case of complete remission (CR) and 3 cases of partial remission (PR), and 16 patients showed homological improvement, responders numbering 20 totally, with a response rate of 62.5% (20/32). One of the 20 patients respondent to CsA died and 7 of the 12 patients who failed to respond to CsA died.
Conclusion: Improving the clinical manifestations and lengthening the survival time, CsA therapy is effective in the patients with RA or RAEB, and both hypoplastic bone marrow and hyperplastic bone marrows respond to the therapy. CsA therapy is potentially the most effective therapy for MDS.