Advances in Hematopoietic Stem Cell Transplantation for Patients with Paroxysmal Nocturnal Hemoglobinuria.

In the era of eculizumab, the number of patients with paroxysmal nocturnal hemoglobinuria (PNH) who undergo hematopoietic stem cell transplantation (HSCT) has decreased significantly. However, owing to the possibility of severe aplastic anemia (AA) or a suboptimal response to eculizumab, HSCT still plays an important role in the treatment of patients with PNH combined with AA or recurrent hemolysis-related symptoms despite its high level of risk. Here we review studies involving patients with PNH who underwent HSCT over the past 15 years and conclude that patients with refractory AA/PNH and patients with severe classical PNH are candidates for HSCT in countries where eculizumab is unavailable. The major causes of death from transplantation include graft-versus-host disease (GVHD), infection, and thrombotic microangiopathy. A haploidentical donor is a potential choice for patients without an HLA-matched donor. In addition, the use of eculizumab in combination with HSCT may help prevent GVHD.