Abstract
Paroxysmal nocturnal hemoglobinuria (PNH) is characterized by intravascular hemolysis, venous thrombosis, and bone marrow failure. Seventeen patients with debilitating PNH, including 8 who were HLA-alloimmunized, underwent a reduced-intensity allogeneic hematopoietic cell transplantation (HCT). All received cyclophosphamide/fludarabine+/- antithymocyte globulin followed by a granulocyte colony-stimulating factor-mobilized HCT from an HLA-matched relative. Glycosylphosphatidylinositol-negative neutrophils were detectable after engraftment but disappeared completely at a median 100days after transplantation. With a median follow-up of nearly 6years, 15 patients (87.8%) survived, all without any evidence of PNH, transfusion independent, and off anticoagulation. Allogeneic reduced-intensity HCT remains a curative therapeutic option for PNH patients who are not candidates for eculizumab treatment.
Original language | English (US) |
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Pages (from-to) | 1435-1439 |
Number of pages | 5 |
Journal | Biology of Blood and Marrow Transplantation |
Volume | 20 |
Issue number | 9 |
DOIs | |
State | Published - Sep 2014 |
Externally published | Yes |
Keywords
- Fludarabine
- Hematopoietic cell transplantation
- Paroxysmal nocturnal hemoglobinuria
- Reduced-intensity
- Survival
ASJC Scopus subject areas
- Hematology
- Transplantation