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 Citations (Scopus)

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

Fingerprint

Paroxysmal Hemoglobinuria
Cell Transplantation
Glycosylphosphatidylinositols
Antilymphocyte Serum
Granulocyte Colony-Stimulating Factor
Hemolysis
Venous Thrombosis
Cyclophosphamide
Neutrophils
Transplantation
Bone Marrow
fludarabine
Therapeutics

Keywords

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

ASJC Scopus subject areas

  • Hematology
  • Transplantation

Cite this

Long-term outcome of fludarabine-based reduced-intensity allogeneic hematopoietic cell transplantation for debilitating paroxysmal nocturnal hemoglobinuria. / Pantin, Jeremy Mark; Tian, Xin; Geller, Nancy; Ramos, Catalina; Cook, Lisa; Cho, Elena; Scheinberg, Phillip; Vasu, Sumithira; Khuu, Hahn; Stroncek, David; Barrett, John; Young, Neal S.; Donohue, Theresa; Childs, Richard W.

In: Biology of Blood and Marrow Transplantation, Vol. 20, No. 9, 01.01.2014, p. 1435-1439.

Research output: Contribution to journalArticle

Pantin, JM, Tian, X, Geller, N, Ramos, C, Cook, L, Cho, E, Scheinberg, P, Vasu, S, Khuu, H, Stroncek, D, Barrett, J, Young, NS, Donohue, T & Childs, RW 2014, 'Long-term outcome of fludarabine-based reduced-intensity allogeneic hematopoietic cell transplantation for debilitating paroxysmal nocturnal hemoglobinuria', Biology of Blood and Marrow Transplantation, vol. 20, no. 9, pp. 1435-1439. https://doi.org/10.1016/j.bbmt.2014.05.012
Pantin, Jeremy Mark ; Tian, Xin ; Geller, Nancy ; Ramos, Catalina ; Cook, Lisa ; Cho, Elena ; Scheinberg, Phillip ; Vasu, Sumithira ; Khuu, Hahn ; Stroncek, David ; Barrett, John ; Young, Neal S. ; Donohue, Theresa ; Childs, Richard W. / Long-term outcome of fludarabine-based reduced-intensity allogeneic hematopoietic cell transplantation for debilitating paroxysmal nocturnal hemoglobinuria. In: Biology of Blood and Marrow Transplantation. 2014 ; Vol. 20, No. 9. pp. 1435-1439.
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