Hematopoietic Stem Cell Transplantation in PNH

The only curative treatment for PNH is hematopoietic stem cell transplantation (HSCT). However, the risk of treatment-related mortality after HSCT is relatively high, with graft-versus-host disease (GvHD) accounting for most of the transplant-related deaths. In recent years, the introduction of eculizumab, a humanized monoclonal antibody directed against the terminal complement protein C5, has had a major impact on the management of PNH. Eculizumab is highly effective in reducing intravascular hemolysis and seems to reduce the risk of thrombosis markedly. The decision to perform an HSCT in PNH has usually been deferred until disease progression to recurrent, life-threatening thromboembolism, refractory or transfusion-dependent hemolytic anemia, or development of severe aplastic anemia. Indications for HSCT should weight the natural history of the disease, the prognostic risk factors, as well as specific complications related to transplantation. Here we reviewed both single-center studies and registry studies to identify the best candidates eligible for an HSCT in patients with PNH in the complement inhibitors era.