Immunosuppressive therapy for hypoplastic myelodysplastic syndrome

Abstract

BACKGROUND: Hypoplastic myelodysplastic syndrome (MDS) is characterized by dysplasia and hypocellularity. The treatment of choice for young patients is bone marrow transplantation. METHODS: This report describes the effect of immunosuppressive therapy in two patients with hypoplastic MDS for whom no suitable bone marrow donors were available. RESULTS: Both patients had no human lymphocyte antigen-identical siblings and no suitable matched unrelated donor could be found. They received cyclosporin A (CsA), antithymocyte globulin (ATG), or a combination of the two. Treatment with CsA, ATG, or the combination led to clinical improvement (resolution of transfusion requirement), increase of bone marrow cellularity, and the disappearance of dysplastic characteristics in the two patients with hypoplastic MDS. At the time of recurrence, this disease was still responsive to immunosuppressive therapy. CONCLUSIONS: Treatment with ATG and CsA can be an attractive alternative for patients with hypoplastic MDS for whom there is no possibility of bone marrow transplantation.