异基因造血干细胞移植治疗18例阵发性睡眠性血红蛋白尿症疗效分析

OBJECTIVE: To evaluate the outcomes of allogeneic hematopoietic stem cell transplantation (allo-HSCT) for paroxysmal nocturnal haemoglobinuria (PNH) and aplastic anemia (AA)-PNH syndrome. METHODS: The clinical data of 18 PNH or AA-PNH patients, including 4 classic PNH and 14 AA-PNH, received allo-HSCT from Dec 2007 to Feb 2015 were analyzed retrospectively. Nine patients received HLA-haploidentical donor HSCT (1 patient received salvage HLA-haploidentical donor HSCT after the graft failure of double cord blood transplantation), 7 patients received HLA-identical sibling donor HSCT, and 2 HLA-identical unrelated donor HSCT. The conditioning regimens were as follow: 13 patients received modified BU/CY-based regimens, 5 non-myeloablative regimens [fludarabine (Flu) + anti-thymocyte globulin (ATG) + cyclophosphamide (CY) or busulfan (BU) ]. Prophylaxis for graft-versus-host disease (GVHD) : the patients with HLA-identical sibling donor received cyclosporine (CsA) plus short-term methotrexate (MTX), the patients with HLA-haploidentical donor or HLA-identical unrelated donor received CsA or tacrolimus (FK506) + mycophenolate mofetil (MMF) + short-term methotrexate (MTX). RESULTS: All patients were engrafted successfully (1 patient engrafted by haploidentical donor after the graft failure of double cord blood transplantation). The median days of neutrophils (ANC) above 0.5×10(9)/L and platelets (PLT) more than 20×10(9)/L were 11 (10–26) days and 15 (11–120) days, respectively. Three patients (17.6%) developed acute GVHD (aGVHD), 2 for grade Ⅱ aGVHD, 1 for grade Ⅳ aGVHD. Of 16 patients, 2 occurred limited chronic GVHD (cGVHD). After a median follow-up of 14.6 (2.0–86.7) months, 3 patients (17.6%) died, out of which one died of severe aGVHD, one died of severe pulmonary infection, one pulmonary infection with transplant-associated thrombotic microangiopathy. The 5-year estimated disease free survival was (80.5±10.2) %. No patient relapsed. CONCLUSION: Allo-HSCT is an effective and curable therapy for PNH or AA-PNH with improved prognosis, and offers a valid therapeutic option for these patients before humanized monoclonal antibody against C5 are widely used clinically.