Successful haploidentical hematopoietic stem cell transplantation for paroxysmal nocturnal hemoglobinuria with severe pancytopenia developed after long-term aplastic anemia treatment

Kazuki Sakurai; Kei Saito; Shunsuke Hatta; Yuna Katsuoka; Kuniaki Meguro; Hisayuki Yokoyama; Toru Izumi

doi:10.1002/ccr3.8832

Successful haploidentical hematopoietic stem cell transplantation for paroxysmal nocturnal hemoglobinuria with severe pancytopenia developed after long-term aplastic anemia treatment

Clin Case Rep. 2024 Apr 26;12(5):e8832. doi: 10.1002/ccr3.8832. eCollection 2024 May.

Authors

Kazuki Sakurai^{1

2}, Kei Saito¹, Shunsuke Hatta¹, Yuna Katsuoka¹, Kuniaki Meguro¹, Hisayuki Yokoyama^{1

2}, Toru Izumi¹

Affiliations

¹ Department of Hematology National Hospital Organization Sendai Medical Center Sendai Japan.
² Department of Hematology Tohoku University Hospital Sendai Japan.

Abstract

Haploidentical hematopoietic stem cell transplantation (HSCT) with posttransplant cyclophosphamide is an alternative treatment for aplastic anemia-paroxysmal nocturnal hemoglobinuria (PNH) syndrome with poor prognostic factors. Ravulizumab treatment for PNH before HSCT might have a beneficial effect.

Keywords: alternative donor; aplastic anemia–paroxysmal nocturnal hemoglobinuria syndrome; haploidentical hematopoietic stem cell transplantation; posttransplant cyclophosphamide; ravulizumab.

Publication types

Case Reports