Immunosuppressive therapy for patients with refractory anemia.

Trials of immunosuppressive therapyhave been reported in some case reports of hypoplastic myelodysplastic syndrome(MDS). In this study, we gave immunosuppressive therapiesto eight patients with normo- or hyperplastic MDS of refractory anemia subtype without karyotypic abnormalities and analyzed the HLA-DRB1type or the presence of paroxysmal nocturnal hemoglobinuria(PNH) neutrophils in these patients. Cyclosporin A (CyA) therapywas effective for improving cytopeniain four of the eight MDS patients. While the side effects of CyA were mostly mild and transient, one patient demonstrated karyotypic abnormality following CyA therapyand accelerated to refractory anemia with an excess of blasts. Additional antithymocyte globulin (ATG) therapywas effective in one of three nonresponders to CyA therapy. One patient died due to leukemic transformation after ATG therapy. When we analyzed the correlation between the response to CyA therapyand the HLA-DRB1type, there were more responders with DRB1*1501 (three of four patients) than without (one of four patients), but a statistically significant difference was not evident between the two groups. In addition, the presence of PNH neutrophils was not correlated with the response to CyA and/or ATG therapy. These results indicate the usefulness of immunosuppressive therapieseven for normo- or hyperplastic MDS patients. Further trials using more patients with a long follow-up period would be worthwhile in order to clarify the possibility of disease progression and in order to predict the response of patients.