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Excellent outcomes of allogeneic haematopoietic stem cell transplantation in 44 patients with paroxysmal nocturnal haemoglobinuria: a single-centre study
Liu, L., Liu, S., Zhang, Y., Zhou, H., Wang, Q., Tian, H., Chen, F., Qiu, H., Tang, X., Han, Y., et al
Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation. 2019
Abstract
We analysed the outcomes of 44 paroxysmal nocturnal haemoglobinuria (PNH) patients who received allogeneic haematopoietic stem cell transplantation (allo-HSCT) (haplo-donors, n=25; matched sibling donors [MSD], n=15; and matched unrelated donors, n=4) between July 2007 and May 2018. All cases achieved successful donor engraftment. The median time was 12 days (range 7-26) for myeloid engraftment and 13 days (range 11-75) for platelets. The cumulative incidences were 15.91% and 2.27% for grades II-IV and grades III-IV acute graft-versus-host disease (aGVHD), respectively, with a median follow-up time of 36 months (range 4-132). The cumulative incidences were 26.73% for chronic GVHD (cGVHD) and 9.70% for moderate-severe cGVHD. 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 OSs of the haplo-donor and MSD groups were 86.5 +/- 7.3% versus 93.3 +/- 6.4% (P=0.520). The 3-year GFFSs of the haplo-donor and MSD groups were 78.3 +/- 8.6% versus 92.9 +/- 6.9% (P=0.250). 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.