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Haploidentical hematopoietic stem cell transplant in paroxysmal nocturnal hemoglobinuria.

Post date: 2016-06-26


Leuk Lymphoma. 2016;57(4):835-41. doi: 10.3109/10428194.2015.1068309. Epub 2016 Feb 24.

 

Tian H1,2,3Liu L1,2,3Chen J1,2,3Xu Y1,2,3Jin Z1,2,3Miao M1,2,3Fu Z1,2,3Qiu H1,2,3Sun A1,2,3Wu D1,2,3.

 

Author information

1 Jiangsu Institute of Hematology, The First Affiliated Hospital of Soochow University , Suzhou , China ;

2 Collaborative Innovation Center of Hematology, Soochow University , Suzhou , China ;

3 Suzhou Institute of Blood and Marrow Transplantation , Suzhou , China.

 

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.