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Paroxysmal Nocturnal Haemoglobinuria

Successful unrelated donor bone marrow transplantation for paroxysmal nocturnal hemoglobinuria

Bone Marrow Transplantation volume 27, pages 589–592 (2001)Cite this article

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 × 108 nucleated cells/kg and 1.1–2.1 × 106 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 after BMT. T cell-depleted MUD BMT is an effective treatment option for PNH-related MDS and SAA. Bone Marrow Transplantation (2001) 27, 589–592.

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Authors and Affiliations

  1. Division of Bone Marrow Transplantation, Department of Hematology/Oncology, St Jude Children's Research Hospital, Memphis, TN, USA

    P Woodard, N Pitts, E Benaim, E Horwitz, J Cunningham & L Bowman

  2. Division of Hematology, Department of Hematology/Oncology, St Jude Children's Research Hospital, Memphis, TN, USA

    W Wang

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  1. P Woodard
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  2. W Wang
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  3. N Pitts
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  4. E Benaim
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  5. E Horwitz
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  6. J Cunningham
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  7. L Bowman
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Woodard, P., Wang, W., Pitts, N. et al. Successful unrelated donor bone marrow transplantation for paroxysmal nocturnal hemoglobinuria. Bone Marrow Transplant 27, 589–592 (2001). https://doi.org/10.1038/sj.bmt.1702827

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