Long-term outcome of fludarabine-based reduced-intensity allogeneic hematopoietic cell transplantation for debilitating paroxysmal nocturnal hemoglobinuria

Biol Blood Marrow Transplant. 2014 Sep;20(9):1435-9. doi: 10.1016/j.bbmt.2014.05.012. Epub 2014 May 17.

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 100 days after transplantation. With a median follow-up of nearly 6 years, 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.

Keywords: Fludarabine; Hematopoietic cell transplantation; Paroxysmal nocturnal hemoglobinuria; Reduced-intensity; Survival.

Publication types

  • Research Support, N.I.H., Intramural

MeSH terms

  • Adult
  • Female
  • Hematopoietic Stem Cell Transplantation / methods*
  • Hemoglobinuria, Paroxysmal / mortality
  • Hemoglobinuria, Paroxysmal / therapy*
  • Humans
  • Male
  • Survival Analysis
  • Transplantation Conditioning / methods*
  • Treatment Outcome
  • Vidarabine / administration & dosage
  • Vidarabine / analogs & derivatives*
  • Vidarabine / therapeutic use
  • Young Adult

Substances

  • Vidarabine
  • fludarabine