单倍型造血干细胞移植治疗阵发性睡眠性血红蛋白尿症17例疗效和安全性研究

OBJECTIVE: To explore the efficacy and safety of haploidentical hematopoietic stem cell transplantation (Haplo-HSCT) for paroxysmal nocturnal hemoglobinuria (PNH). METHODS: A total of 17 patients with PNH who received Haplo-HSCT from January 2013 to September 2017 were analyzed retrospectively. RESULTS: Of them, 4 patients had de novo PNH, 13 patients had aplastic anemia-PNH syndrome (AA-PNH). All patients received modified busulfan and Cytoxan (BuCy)-based regimens combined with anti-thymocyte globulin (ATG). Granulocyte colony-stimulating factor-mobilized bone marrow and peripheral blood stem cells were transplanted as graft. Prophylaxis for graft-versus-host disease (GVHD) was ciclosporin A+ mycophenolate mofetil (MMF)+short-term methotrexate (MTX). All patients were engrafted successfully. The median time of neutrophils to 0.5×10(9)/L and platelets to 20×10(9)/L was 12(10–15) days and 14(11–45) days, respectively. All of the 17 patients achieved full donor chimerism at 30 d after Haplo-HSCT. Seven patients developed grade Ⅱ–Ⅳ acute GVHD, and 4 chronic GVHD. Median follow-up time was 27.1 (8.6–60.4) months. Of the 17 patients, 15 survived and 2 died of severe pulmonary infection and transplant associated thrombotic microangiopathy. Three-year overall survival was (77.8±15.2)%. CONCLUSION: Haplo-HSCT may be effective and safe for PNH patients who did not have matched donor.