title: Haploidentical hematopoietic stem cell transplant in paroxysmal nocturnal hemoglobinuria.
abstract: Eighteen patients with paroxysmal nocturnal hemoglobinuria (PNH) receiving allogeneic hematopoietic stem cell transplant (allo-HSCT), either from HLA-haploidentical donors (HRD; n = 10) or HLA-matched donors (n = 5 from siblings and n = 3 from unrelated donors), were retrospectively evaluated. One showed primary graft failure following unrelated-donor HSCT. He was given a second HRD-HSCT, but died from cytomegalovirus pneumonia after achieving hematopoietic recovery. The other 17 patients achieved sustained engraftment and full-donor chimerism. Four in the HRD-HSCT group experienced grade II/III acute graft-versus-host disease (aGVHD), and five in the HLA-matched HSCT group developed grade II aGVHD. Among all 18 patients, 10 developed chronic GVHD (cGVHD), only one patient receiving HRD-HSCT developed extensive cGVHD. Nine in the HRD-HSCT group and all those in the HLA-matched HSCT group were alive and transfusion-independent at last follow-up. Our findings suggest that allo-HSCT is a promising treatment for PNH, and HRD-HSCT is a viable option for patients with PNH who lack HLA-matched donors.
authors.affiliation: a Jiangsu Institute of Hematology, The First Affiliated Hospital of Soochow University , Suzhou , China ;|b Collaborative Innovation Center of Hematology, Soochow University , Suzhou , China ;|c Suzhou Institute of Blood and Marrow Transplantation , Suzhou , China.
authors.forename: Hong
authors.initials: H
authors.lastname: Tian
authors.affiliation: a Jiangsu Institute of Hematology, The First Affiliated Hospital of Soochow University , Suzhou , China ;|b Collaborative Innovation Center of Hematology, Soochow University , Suzhou , China ;|c Suzhou Institute of Blood and Marrow Transplantation , Suzhou , China.
authors.forename: Liming
authors.initials: L
authors.lastname: Liu
authors.affiliation: a Jiangsu Institute of Hematology, The First Affiliated Hospital of Soochow University , Suzhou , China ;|b Collaborative Innovation Center of Hematology, Soochow University , Suzhou , China ;|c Suzhou Institute of Blood and Marrow Transplantation , Suzhou , China.
authors.forename: Jia
authors.initials: J
authors.lastname: Chen
authors.affiliation: a Jiangsu Institute of Hematology, The First Affiliated Hospital of Soochow University , Suzhou , China ;|b Collaborative Innovation Center of Hematology, Soochow University , Suzhou , China ;|c Suzhou Institute of Blood and Marrow Transplantation , Suzhou , China.
authors.forename: Yang
authors.initials: Y
authors.lastname: Xu
authors.affiliation: a Jiangsu Institute of Hematology, The First Affiliated Hospital of Soochow University , Suzhou , China ;|b Collaborative Innovation Center of Hematology, Soochow University , Suzhou , China ;|c Suzhou Institute of Blood and Marrow Transplantation , Suzhou , China.
authors.forename: Zhengming
authors.initials: Z
authors.lastname: Jin
authors.affiliation: a Jiangsu Institute of Hematology, The First Affiliated Hospital of Soochow University , Suzhou , China ;|b Collaborative Innovation Center of Hematology, Soochow University , Suzhou , China ;|c Suzhou Institute of Blood and Marrow Transplantation , Suzhou , China.
authors.forename: Miao
authors.initials: M
authors.lastname: Miao
authors.affiliation: a Jiangsu Institute of Hematology, The First Affiliated Hospital of Soochow University , Suzhou , China ;|b Collaborative Innovation Center of Hematology, Soochow University , Suzhou , China ;|c Suzhou Institute of Blood and Marrow Transplantation , Suzhou , China.
authors.forename: Zhengzheng
authors.initials: Z
authors.lastname: Fu
authors.affiliation: a Jiangsu Institute of Hematology, The First Affiliated Hospital of Soochow University , Suzhou , China ;|b Collaborative Innovation Center of Hematology, Soochow University , Suzhou , China ;|c Suzhou Institute of Blood and Marrow Transplantation , Suzhou , China.
authors.forename: Huiying
authors.initials: H
authors.lastname: Qiu
authors.affiliation: a Jiangsu Institute of Hematology, The First Affiliated Hospital of Soochow University , Suzhou , China ;|b Collaborative Innovation Center of Hematology, Soochow University , Suzhou , China ;|c Suzhou Institute of Blood and Marrow Transplantation , Suzhou , China.
authors.forename: Aining
authors.initials: A
authors.lastname: Sun
authors.affiliation: a Jiangsu Institute of Hematology, The First Affiliated Hospital of Soochow University , Suzhou , China ;|b Collaborative Innovation Center of Hematology, Soochow University , Suzhou , China ;|c Suzhou Institute of Blood and Marrow Transplantation , Suzhou , China.
authors.forename: Depei
authors.initials: D
authors.lastname: Wu
mesh_terms: D000293:Adolescent; D000328:Adult; D005190:Family; D005260:Female; D006085:Graft Survival; D006086:Graft vs Host Disease / Q000209:etiology; D006239:Haplotypes; D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects / Q000379:methods; D006457:Hemoglobinuria, Paroxysmal / Q000175:diagnosis / Q000401:mortality / Q000628:therapy*; D006649:Histocompatibility Antigens / Q000235:genetics / Q000276:immunology; D006801:Humans; D008297:Male; D008875:Middle Aged; D019172:Transplantation Conditioning; D014184:Transplantation, Homologous; D016896:Treatment Outcome; D055815:Young Adult
chemical_list: D006649:Histocompatibility Antigens
keywords: Haploidentical hematopoietic stem cell transplant; myeloablative conditioning; paroxysmal nocturnal hemoglobinuria