Allogeneic hematopoietic stem cell transplantation for paroxysmal nocturnal hemoglobinuria in the era of complement inhibition

The only potentially curative treatment for paroxysmal nocturnal hemoglobinuria (PNH) is allogeneic hematopoietic stem cell transplantation (allo-HSCT). However, its use has been largely abandoned following the introduction of efficient symptomatic treatment with complement inhibition. Nevertheless, the population of PNH patients is diverse, and some of them might still gain advantage from allo-HSCT, while anti-complement treatment would be the first choice for others. Both treatment modalities may be also sequentially applied in the same patient when needed. This review aimed to present the current status of allo-HSCT in the treatment of patients with PNH, with special reference to Poland where the previous unavailability of anti-complement therapy enabled the acquisition of extensive experience in performing allo-HSCT for PNH, a treatment option currently restricted only to selected patients who are not candidates for eculizumab.