Hegenbart, Ute and Niederwieser, D. and Forman, S. and Holler, E. and Leiblein, S. and Johnston, L. and Poenisch, W. and Epner, E. and Witherspoon, R. and Blume, K. and Storb, R. (2003) Hematopoietic cell transplantation from related and unrelated donors after minimal conditioning as a curative treatment modality for severe paroxysmal nocturnal hemoglobinuria. BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION, 9 (11). pp. 689-697. ISSN 1083-8791
Full text not available from this repository. (Request a copy)Abstract
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare clonal. disorder caused by a somatic mutation of the X-linked phosphatidylinositol glycan class A gene. Allogeneic hematopoietic cell transplantation (HCT) after high-dose conditioning is the only curative treatment; however, it is associated with high treatment-related mortality. Here, we report on allogeneic HCT for PNH after minimal conditioning. Seven adult patients with high-risk PNH underwent peripheral blood HCT from HIA-A-, -B-, -C-, -DRB1-, and -DQB1-matched related (n = 2) and unrelated (n = 5) donors. Conditioning included fludarabine 30 mg/m(2)/d on days -4 to -2 and 2 Gy of total body irradiation on day 0. After HCT, patients were given immunosuppressive therapy with oral cyclosporine starting on day -3 and mycophenolate mofetil starting on day 0. All 7 patients attained durable engraftment. After 28 days, a median of 77% (range, 53%-96%) T-cell donor chimerism. was found in bone marrow and peripheral blood. T-cell chimerism increased to 91% (range, 76%-100%) on day +180 and to 100% in all surviving patients after 12 months. All 7 patients attained complete remissions of their disease. Four patients are alive 13 to 38 months after HCT. Three patients died of treatment-related mortality, 1 because of complications after acute pancreatitis and multiorgan failure, 1 because of infection related to chronic graft-versus-host disease (GVHD), and 1 because of bleeding after liver biopsy for late subacute/chronic GVHD. Allogeneic HCT from related and unrelated donors after minimal conditioning is a new and potentially curative option for patients with advanced PNH. (C) 2003 American Society for Blood and Marrow Transplantation.
| Item Type: | Article |
|---|---|
| Uncontrolled Keywords: | BONE-MARROW-TRANSPLANTATION; TOTAL-BODY IRRADIATION; VERSUS-HOST DISEASE; THERAPY; IMMUNOSUPPRESSION; MALIGNANCIES; CHIMERISM; ANCHOR; ANEMIA; paroxysmal nocturnal hemoglobinuria; allogeneic HCT |
| Subjects: | 600 Technology > 610 Medical sciences Medicine |
| Divisions: | Medicine > Lehrstuhl für Innere Medizin III (Hämatologie und Internistische Onkologie) |
| Depositing User: | Dr. Gernot Deinzer |
| Date Deposited: | 30 Aug 2021 05:18 |
| Last Modified: | 30 Aug 2021 08:27 |
| URI: | https://pred.uni-regensburg.de/id/eprint/38428 |
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