Paroxysmal nocturnal hemoglobinuria (PNH) is a clonal proliferative disease of hematopoietic stem cells that is clinically characterized by hemolysis, thrombosis, and bone marrow failure. In recent years, significant progress has been made in the complement inhibitor-based treatment of PNH, but the only curative treatment is still hematopoietic stem cell transplantation (HSCT). This article reviews the research progress on allogeneic HSCT for PNH, systematically summarizes the overview, indications, influencing factors, and treatment outcomes of allogeneic HSCT for PNH, and provides an analysis of HSCT for PNH.
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Li et al. (2025) studied this question.
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