Successful unrelated donor bone marrow transplantation for paroxysmal nocturnal hemoglobinuria

Bone Marrow Transplantation
P WoodardL Bowman

Abstract

Paroxysmal nocturnal hemoglobinuria (PNH) is an acquired clonal disease of hematopoiesis due to a mutation in the PIG-A gene. Affected patients may demonstrate hemolysis or venous thrombosis, and may develop MDS or aplastic anemia. Successful results may be obtained after conditioning and transplantation from syngeneic or genotypically matched sibling donors. Experience with transplantation from matched unrelated donors (MUD) is limited to eight patients, with only one survivor. We report three patients who underwent successful MUD BMT for PNH. All three patients had severe aplastic anemia (SAA) and PNH at the time of BMT. Unrelated donors were six-antigen HLA-matched (n = 2) or HLA-A mismatched (n = 1). Conditioning consisted of cytarabine, cyclophosphamide, TBI, and ATG. Grafts were T cell-depleted by anti-CD6/CD8 antibodies + complement. Further GVHD prophylaxis consisted of cyclosporine. Patients received 0.7-1.1 x 10(8) nucleated cells/kg and 1.1-2.1 x 10(6) CD34(+) cells/kg. Neutrophil engraftment occurred at 16-21 days. One patient developed grade 1 acute GVHD. Although all three patients experienced significant transplant-related complications, they ultimately resolved and all patients are alive and well 30-62 months af...Continue Reading

References

Apr 1, 1992·American Journal of Hematology·K KawaharaR Storb
Oct 1, 1988·Journal of Clinical Oncology : Official Journal of the American Society of Clinical Oncology·S I BearmanE D Thomas
Feb 1, 1997·American Journal of Hematology·W J HarringtonY S Ahn
Feb 27, 1999·Leukemia & Lymphoma·J W HarrisM E Medof
Mar 2, 1999·British Journal of Haematology·R SasoE C Gordon-Smith
Apr 29, 1999·Proceedings of the National Academy of Sciences of the United States of America·D J AratenL Luzzatto
May 8, 1999·British Journal of Haematology·M BembaG Socié
Sep 25, 1999·Annals of Internal Medicine·D E DunnN S Young

❮ Previous
Next ❯

Citations

Jul 9, 2002·British Journal of Haematology·Christian FlothoCharlotte M Niemeyer
Jul 30, 2005·Blood·Charles ParkerUNKNOWN International PNH Interest Group
Nov 5, 2016·Indian Journal of Hematology & Blood Transfusion : an Official Journal of Indian Society of Hematology and Blood Transfusion·Amrallah A MohammedAyman A Rasmy
Mar 31, 2009·Hematology/oncology Clinics of North America·Charles J Parker
Aug 2, 2003·European Journal of Haematology·Jae-Lyun LeeKyoo-Hyung Lee
Apr 27, 2011·British Journal of Haematology·Lucio LuzzattoRosario Notaro
Dec 4, 2003·Biology of Blood and Marrow Transplantation : Journal of the American Society for Blood and Marrow Transplantation·U HegenbartR Storb
May 20, 2009·Biology of Blood and Marrow Transplantation : Journal of the American Society for Blood and Marrow Transplantation·Nelson A Matos-FernandezMohamed A Kharfan-Dabaja
Jan 29, 2009·Biology of Blood and Marrow Transplantation : Journal of the American Society for Blood and Marrow Transplantation·Charles J ParkerJohn E Levine
Oct 31, 2014·The Cochrane Database of Systematic Reviews·Arturo J Martí-CarvajalIvan Solà

❮ Previous
Next ❯

Related Concepts

Related Feeds

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.

© 2021 Meta ULC. All rights reserved