Outcomes of allogeneic hematopoietic stem cell transplantation in patients with paroxysmal nocturnal hemoglobinuria compared to paroxysmal nocturnal hemoglobinuria-aplastic anemia syndrome

• Published in: Zhōnghuá xuèyèxué zázhì Vol. 40; no. 6; p. 472
• Main Authors: Liu, L M, Zhou, H F, Wang, Q Y, Qiu, H Y, Tang, X W, Han, Y, Fu, C C, Jin, Z M, Chen, S N, Sun, A N, Miao, M, Wu, D P
• Format: Journal Article
• Language: Chinese
• Published: China 14.06.2019
• Subjects: Anemia, Aplastic - therapy; Hematopoietic Stem Cell Transplantation; Hemoglobinuria, Paroxysmal - therapy; Humans; Retrospective Studies; Transplantation, Homologous; Treatment Outcome; Paroxysmal nocturnal hemoglobinuria; Allogeneic stem cell transplantation; Outcomes; Aplastic anemia
• ISSN: 0253-2727
• DOI: 10.3760/cma.j.issn.0253-2727.2019.06.005

To compare the outcomes of allogeneic hematopoietic stem cell transplantation (allo-HSCT) for paroxysmal nocturnal hemoglobinuria (PNH) with paroxysmal nocturnal hemoglobinuria-aplastic anemia (PNH-AA) syndrome. The outcomes of 46 patients who received allo-HSCT (16 PNH patients, 30 PNH-AA patients) from July 10, 2007 to June 2, 2018 were analyzed retrospectively. The conditioning regimen was busulfan, cyclophosphoramide, and ATG in haploidentical donors and unrelated donors. Patients with matched sibling donors were treated with the fludarabine, cyclophosphamide, and ATG regimen. There were no differences of baseline data between the 2 groups except gender distribution and the numbers of haploidentical donor transplantation. The median values of absolute nucleated cell counts were 10.58 (3.83-13.83) ×10(8)/kg in the PNH group and 10.81 (3.96-33.40) ×10(8)/kg in the PNH-AA group ( =0.668) . The median doses of CD34(+) cells infused were 5.00 (3.14-8.42) ×10(6)/kg and 3.57 (1.97-6.17) ×10(6)/kg ( =0.002) , res