Bone Marrow Failure Disorders

This comprehensive review was written by 2 members of the Severe Aplastic Anaemia Working Party (SAAWP) of the European Group for Bone Marrow Transplantation (EBMT), Leiden, the Netherlands. The authors note the emergence of novel strategies of complement inhibition in the management of paroxysmal nocturnal hemoglobinuria, including newer anti-C5 agents that match eculizumab in safety and efficacy but whose administration is oral or subcutaneous and therefore more convenient for patients. The authors also review benefits and considerations for use of proximal complement inhibitors developed to address C3-mediated extravascular hemolysis.