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

Jeremy Mark Pantin, Xin Tian, Nancy Geller, Catalina Ramos, Lisa Cook, Elena Cho, Phillip Scheinberg, Sumithira Vasu, Hahn Khuu, David Stroncek, John Barrett, Neal S. Young, Theresa Donohue, Richard W. Childs

Research output: Contribution to journalArticle

12 Scopus citations

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 languageEnglish (US)
Pages (from-to)1435-1439
Number of pages5
JournalBiology of Blood and Marrow Transplantation
Volume20
Issue number9
DOIs
StatePublished - Jan 1 2014

Keywords

  • Fludarabine
  • Hematopoietic cell transplantation
  • Paroxysmal nocturnal hemoglobinuria
  • Reduced-intensity
  • Survival

ASJC Scopus subject areas

  • Hematology
  • Transplantation

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    Pantin, J. M., Tian, X., Geller, N., Ramos, C., Cook, L., Cho, E., Scheinberg, P., Vasu, S., Khuu, H., Stroncek, D., Barrett, J., Young, N. S., Donohue, T., & Childs, R. W. (2014). Long-term outcome of fludarabine-based reduced-intensity allogeneic hematopoietic cell transplantation for debilitating paroxysmal nocturnal hemoglobinuria. Biology of Blood and Marrow Transplantation, 20(9), 1435-1439. https://doi.org/10.1016/j.bbmt.2014.05.012