Long-term outcome of fludarabine-based reduced intensity allogeneic hematopoietic cell transplantation for debilitating paroxysmal nocturnal hemoglobinuria | Aplastic Anemia & MDS International Foundation Return to top.

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

Journal Title: 
Biol Blood Marrow Transplant
Primary Author: 
Pantin J
Author(s): 
Pantin J, 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
Original Publication Date: 
Saturday, May 17, 2014

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 transplant (HCT). All received cyclophosphamide/fludarabine +/- ATG followed by a G-CSF mobilized HCT from an HLA-matched relative. GPI-negative neutrophils were detectable following engraftment, but disappeared completely at a median 100 days after transplant. With a median follow-up of nearly 6 years, 15patients (87.8%) survive, all without any evidence of PNH, transfusion-independent, off anticoagulation. Allogeneic reduced-intensity (RI) HCT remains a curative therapeutic option for PNH patients who are not candidates for eculizumab treatment.

Bone Marrow Disease(s): 
  • paroxysmal nocturnal hemoglobinuria (PNH)
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