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Allogeneic peripheral-blood stem cell transplantation (alloSCT) is curative for many patients with relapsed or high-risk hematologic malignancies. However, chronic graft-versus-host disease (GVHD) can lead to persistent complications, poor quality of life, and increased mortality — a “Pyrrhic victory” for cured patients. In a phase 3, multicenter study, patients with acute leukemia or myelodysplastic syndrome undergoing matched related donor alloSCT were randomized to experimental GVHD prophylaxis with high-dose cyclophosphamide on days 3 and 4 posttransplant plus cyclosporine (n=61) or standard prophylaxis with methotrexate plus cyclosporine (n=68).
During a median follow-up of 36 months, the experimental-prophylaxis group had:
A significant…