Excellent Outcomes of Allogeneic Hematopoietic Stem Cell Transplantation in Patients with Paroxysmal Nocturnal Hemoglobinuria: A Single-Center Study.

Abstract

We analyzed the outcomes of 44 patients with paroxysmal nocturnal hemoglobinuria (PNH) who received allogeneic hematopoietic stem cell transplantation (allo-HSCT) (haploidentical [haplo]-donors, 25; matched sibling donors [MSDs], 15; and matched unrelated donors, 4) between July 2007 and May 2018. All patients achieved successful donor engraftment. The median time was 12 days (range, 7 to 26) for myeloid engraftment and 13 days (range, 11 to 75) for platelets. The cumulative incidences were 15.91% and 2.27% for grades II to IV and grades III to IV acute graft-versus-host disease (GVHD), respectively, with a median follow-up time of 36 months (range, 4 to 132). The cumulative incidences were 26.73% for chronic GVHD and 9.70% for moderate to severe chronic GVHD. No patients relapsed. The probabilities of 3-year overall survival (OS) and GVHD-free, failure-free survival (GFFS) were 90.4% ± 4.6% and 85.6% ± 5.4%, respectively. The 3-year OS rates of the haplo-donor and MSD groups were 86.5% ± 7.3% versus 93.3% ± 6.4% (P = .520). The 3-year GFFS rates of the haplo-donor and MSD groups were 78.3% ± 8.6% versus 92.9% ± 6.9% (P = .250). The preliminary results indicated that allo-HSCT is a feasible option for patients with PNH; however, this should not be considered as a first-choice therapy, because the results seemed to only suggest rather than confirm that haplo-HSCT and MSD-HSCT exerted similar therapeutic efficacies.

• Authors: Limin Liu, Shan Liu, Yanming Zhang, Huifen Zhou, Qingyuan Wang, Hong Tian, Feng Chen, Huiying Qiu, Xiaowen Tang, Yue Han, Chengcheng Fu, Zhengming Jin, Suning Chen, Aining Sun, Miao Miao, Depei Wu
• Journal: Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation (Biol Blood Marrow Transplant) Vol. 25 Issue 8 Pg. 1544-1549 (08 2019) ISSN: 1523-6536 [Electronic] United States
• PMID: 30853571 (Publication Type: Clinical Trial, Journal Article, Research Support, Non-U.S. Gov't)
• Copyright: Copyright © 2019 American Society for Blood and Marrow Transplantation. Published by Elsevier Inc. All rights reserved.

Topics

• Acute Disease
• Adolescent
• Adult
• Allografts
• Child
• Chronic Disease
• Disease-Free Survival
• Female
• Follow-Up Studies
• Graft vs Host Disease (mortality, therapy)
• Hematopoietic Stem Cell Transplantation
• Hemoglobinuria, Paroxysmal (therapy)
• Humans
• Incidence
• Male
• Middle Aged
• Siblings
• Survival Rate
• Tissue and Organ Procurement