Long-Term Outcome of Fludarabine-Based Reduced-Intensity Allogeneic Hematopoietic Cell Transplantation for Debilitating Paroxysmal Nocturnal Hemoglobinuria |
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| Affiliation: | 1. Hematology Branch, National Heart, Lung and Blood Institute, National Institutes of Health, Bethesda, Maryland;2. Division of Hematology and Oncology, Department of Medicine, Georgia Regents University, Augusta, Georgia;3. Office of Biostatistics Research, National Heart, Lung and Blood Institute, National Institutes of Health, Bethesda, Maryland;4. Hospital São José - Hospital Beneficência Portuguesa de São Paulo, São Paulo, Brazil;5. Division of Hematology, Department of Medicine, Wexner Medical Center, Ohio State University, Columbus, Ohio;6. Department of Transfusion Medicine, Clinical Research Center, National Institutes of Health, Bethesda, Maryland |
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| Abstract: | Paroxysmal nocturnal hemoglobinuria (PNH) is characterized by intravascular hemolysis, venous thrombosis, and bone marrow failure. Seventeen patients with debilitating PNH, including 8 who were HLA-alloimmunized, underwent a reduced-intensity allogeneic hematopoietic cell transplantation (HCT). All received cyclophosphamide/fludarabine +/− antithymocyte globulin followed by a granulocyte colony-stimulating factor–mobilized HCT from an HLA-matched relative. Glycosylphosphatidylinositol-negative neutrophils were detectable after engraftment but disappeared completely at a median 100 days after transplantation. With a median follow-up of nearly 6 years, 15 patients (87.8%) survived, all without any evidence of PNH, transfusion independent, and off anticoagulation. Allogeneic reduced-intensity HCT remains a curative therapeutic option for PNH patients who are not candidates for eculizumab treatment. |
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| Keywords: | Paroxysmal nocturnal hemoglobinuria Reduced-intensity Hematopoietic cell transplantation Survival Fludarabine |
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