Recovery from life-threatening transplantation-associated thrombotic microangiopathy using eculizumab in a patient with very severe aplastic anemia

摘要

Transplantation-associated thrombotic microangiopathy (TA-TMA) is a life-threatening complication of allogeneic hematopoietic SCT (HSCT). Particularly, prognosis of the TA-TMA with multiorgan impairment is poor because there is no definitive therapy. The pathogenesis of TA-TMA remains undetermined; however, Jodele etal. reported that dysregulation of the complement system may be involved in the pathogenesis of TA-TMA. Eculizumab, a humanized moAb against the complement component C5 that prevents tissue damage by blocking the formation of the membrane attack complex, has been successful in the treatment of paroxysmal nocturnal hemoglobinuria or atypical hemolytic uremic syndrome (aHUS). Thus, if uncontrolled activation of the complement system has an important role in the development of TA-TMA, eculizmab could be a targeted therapy for TA-TMA.