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

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

Journal Title: 
Biol Blood Marrow Transplant
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
Primary Author: 
Pantin J
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.