Exploration on the treatment of paroxysmal nocturnal hemoglobinuria by allogeneic hematopoietic stem cell transplantation

Abstract

Objective To explore the efficacy and safety of allogeneic hematopoietic stem cell transplantation (allo-HSCT) in the treatment of refractory paroxysmal nocturnal hemoglobinuria (PNH).Methods The clinical data of four cases including three patients with refractory PNH and one patient with PNH-aplastic anemia syndrome who underwent allo-HSCT with HLA 6/6 family donor either were analyzed retrospectively.Conditioning regimen was BU/CY + ATG scheme,combining cyclosporin and methotrexate to prevent acute graft versus host disease (aGVHD).The treatment of the cases was discussed with literatures review.Results All patients' deoxyribonucleic acid (DNA) were totally donors' by DNA polymorphism analysis of short tandem repeat sequence.The number of neutrophil was more than 0.5×109/L in 15 days (11-18 days) averagely after transplantation,and platelet was more than 20×109/L in 19 days (14-28 days).The expression of CD55 and CD59 on the membrane of erythrocyte and neutrophil returned to normal on 15-30 days after transplantation.In addition,aGVHD at grade 2 occurred in two cases,and chronic GVHD in one case,excitingly,no graft rejection happened.All recipients were followed up for 14 months (7-42 months)with disease-free survival.Conclusion allo-HSCT is an effective technique to cure refractory PNH. Key words: Paroxysmal nocturnal hemoglobinuria; Hematopoietic stem cell transplantation,allogeneic