Haploidentical hematopoietic stem cell transplant in paroxysmal nocturnal hemoglobinuria

Leukemia & Lymphoma
Hong TianDe-Pei Wu

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.

References

Jan 1, 1989·Baillière's Clinical Haematology·B Rotoli, L Luzzatto
Mar 2, 1999·British Journal of Haematology·R SasoE C Gordon-Smith
Aug 2, 2003·European Journal of Haematology·Jae-Lyun LeeKyoo-Hyung Lee
Dec 4, 2003·Biology of Blood and Marrow Transplantation : Journal of the American Society for Blood and Marrow Transplantation·U HegenbartR Storb
Apr 7, 2005·JAMA : the Journal of the American Medical Association·Russell P RotherMark T Gladwin
Mar 24, 2007·Experimental Hematology·Charles J Parker
Mar 28, 2008·Transfusion·Minoko TakanashiKazunori Nakajima
Jan 16, 2009·Lancet·Charles Parker
Jul 16, 2010·Blood·Minoko TakanashiShunichi Kato
Jun 13, 2012·Haematologica·Régis Peffault de LatourGerard Socié
Jan 29, 2013·Biology of Blood and Marrow Transplantation : Journal of the American Society for Blood and Marrow Transplantation·Douglas E GladstoneMary S Leffell
Feb 14, 2014·The New England Journal of Medicine·Jun-ichi NishimuraYuzuru Kanakura
May 23, 2014·Biology of Blood and Marrow Transplantation : Journal of the American Society for Blood and Marrow Transplantation·Jeremy PantinRichard W Childs
May 20, 2015·Biology of Blood and Marrow Transplantation : Journal of the American Society for Blood and Marrow Transplantation·Stefan O CiureaKai Cao

❮ Previous
Next ❯

Related Concepts

Related Feeds

CNS Lymphoma

In CNS lymphoma, cancerous cells from lymph tissues or other parts of the body form tumors in the brain and/or spinal cord. Here is the latest research on this rare non-Hodgkin lymphoma.

Allogenic & Autologous Therapies

Allogenic therapies are generated in large batches from unrelated donor tissues such as bone marrow. In contrast, autologous therapies are manufactures as a single lot from the patient being treated. Here is the latest research on allogenic and autologous therapies.

Blood And Marrow Transplantation

The use of hematopoietic stem cell transplantation or blood and marrow transplantation (bmt) is on the increase worldwide. BMT is used to replace damaged or destroyed bone marrow with healthy bone marrow stem cells. Here is the latest research on bone and marrow transplantation.

Astrocytes

Astrocytes are glial cells that support the blood-brain barrier, facilitate neurotransmission, provide nutrients to neurons, and help repair damaged nervous tissues. Here is the latest research.