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Leukemia & Lymphoma Volume 57, 2016 - Issue 4
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Original Articles: Clinical

Haploidentical hematopoietic stem cell transplant in paroxysmal nocturnal hemoglobinuria

Hong TianJiangsu Institute of Hematology, The First Affiliated Hospital of Soochow University, Suzhou, China; ;Collaborative Innovation Center of Hematology, Soochow University, Suzhou, China; ;Suzhou Institute of Blood and Marrow Transplantation, Suzhou, China
,
Liming LiuJiangsu Institute of Hematology, The First Affiliated Hospital of Soochow University, Suzhou, China; ;Collaborative Innovation Center of Hematology, Soochow University, Suzhou, China; ;Suzhou Institute of Blood and Marrow Transplantation, Suzhou, China
,
Jia ChenJiangsu Institute of Hematology, The First Affiliated Hospital of Soochow University, Suzhou, China; ;Collaborative Innovation Center of Hematology, Soochow University, Suzhou, China; ;Suzhou Institute of Blood and Marrow Transplantation, Suzhou, China
,
Yang XuJiangsu Institute of Hematology, The First Affiliated Hospital of Soochow University, Suzhou, China; ;Collaborative Innovation Center of Hematology, Soochow University, Suzhou, China; ;Suzhou Institute of Blood and Marrow Transplantation, Suzhou, China
,
Zhengming JinJiangsu Institute of Hematology, The First Affiliated Hospital of Soochow University, Suzhou, China; ;Collaborative Innovation Center of Hematology, Soochow University, Suzhou, China; ;Suzhou Institute of Blood and Marrow Transplantation, Suzhou, China
,
Miao MiaoJiangsu Institute of Hematology, The First Affiliated Hospital of Soochow University, Suzhou, China; ;Collaborative Innovation Center of Hematology, Soochow University, Suzhou, China; ;Suzhou Institute of Blood and Marrow Transplantation, Suzhou, China
,
Zhengzheng FuJiangsu Institute of Hematology, The First Affiliated Hospital of Soochow University, Suzhou, China; ;Collaborative Innovation Center of Hematology, Soochow University, Suzhou, China; ;Suzhou Institute of Blood and Marrow Transplantation, Suzhou, China
,
Huiying QiuJiangsu Institute of Hematology, The First Affiliated Hospital of Soochow University, Suzhou, China; ;Collaborative Innovation Center of Hematology, Soochow University, Suzhou, China; ;Suzhou Institute of Blood and Marrow Transplantation, Suzhou, China
,
Aining SunJiangsu Institute of Hematology, The First Affiliated Hospital of Soochow University, Suzhou, China; ;Collaborative Innovation Center of Hematology, Soochow University, Suzhou, China; ;Suzhou Institute of Blood and Marrow Transplantation, Suzhou, China
&
Depei WuJiangsu Institute of Hematology, The First Affiliated Hospital of Soochow University, Suzhou, China; ;Collaborative Innovation Center of Hematology, Soochow University, Suzhou, China; ;Suzhou Institute of Blood and Marrow Transplantation, Suzhou, ChinaCorrespondence[email protected]
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Pages 835-841 | Received 30 Mar 2015, Accepted 26 Jun 2015, Published online: 24 Feb 2016
 
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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.

Acknowledgements

This work was supported by the Jiangsu Provincial Special Program of Medical Science (BL2012005), the Jiangsu Province’s Key Medical Center (ZX201102), the Priority Academic Program Development of Jiangsu Higher Education Institutions (PAPD), and the National Public Health Grand Research Foundation (No. 201202017).

Potential conflict of interest

Disclosure forms provided by the authors are available with the full text of this article at http://dx.doi.org/10.3109/10428194.2015.1068309.

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