Immunosuppressive therapy for patients with refractory anemia

Trials of immunosuppressive therapy have been reported in some case reports of hypoplastic myelodysplastic syndrome (MDS). In this study, we gave immu nosuppressive therapies to eight patients with normo- or hyperplastic MDS o f refractory anemia subtype without karyotypic abnormalities and analyzed t he HLA-DRB1 type or the presence of paroxysmal nocturnal hemoglobinuria (PN H) neutrophils in these patients. Cyclosporin A (CyA) therapy was effective for improving cytopenia in four of the eight MDS patients. While the side effects of CyA were mostly mild and transient, one patient demonstrated kar yotypic abnormality following CyA therapy and accelerated to refractory ane mia with an excess of blasts. Additional antithymocyte globulin (ATG) thera py was 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 therapy and the HLA-DRB1 type, the re were more responders with DRB1*1501 (three of four patients) than withou t (one of four patients), but a statistically significant difference was no t evident between the two groups. In addition, the presence of PNH neutroph ils was not correlated with the response to CyA and/or ATG therapy. These r esults indicate the usefulness of immunosuppressive therapies even for norm o- or hyperplastic MDS patients. Further trials using more patients with a long follow-up period would be worthwhile in order to clarify the possibili ty of disease progression and in order to predict the response of patients.