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.
- paroxysmal nocturnal hemoglobinuria (PNH)