Efficacy and safety of haploidentical hematopoietic stem cell transplantation for 17 patients with paroxysmal nocturnal hemamoglobinuria
10.3760/cma.j.issn.0253-2727.2018.11.006
- VernacularTitle: 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
- Author:
Jing XIA
1
;
Suning CHEN
;
Jia CHEN
;
Yi FAN
;
Feng CHEN
;
Xiao MA
;
Miao MIAO
;
Depei WU
Author Information
1. Jiangsu Institute of Hematology, Department of Hematology, the First Affiliated Hospital of Soochow University, Key Lab of Thrombosis and Hemostasis of Ministry of Health, Suzhou 215006, China
- Publication Type:Journal Article
- Keywords:
Paroxysmal nocturnal hemoglobinuria;
Haplotypes;
Hematopoietic stem cell transplantation
- From:
Chinese Journal of Hematology
2018;39(11):904-907
- CountryChina
- Language:Chinese
-
Abstract:
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 0.5×109/L and platelets to 20×109/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.
