Abstract
The following is a case of a 40-year-old man who showed acute myeloid leukemia (AML) with paroxysmal nocturnal hemoglobinuria (PNH). Unrelated bone marrow transplantation using a myeloablative conditioning regimen (cyclophosphamide, 120mg/kg; total-body irradiation, 12Gy) was performed 6 years after the diagnosis of PNH. We administered eculizumab seven times, until 4 days before transplantation; hemolysis findings improved before transplantation. We observed a mild, temporary increase in LDH after transplantation, but the level was normalized after day 19, and no acute hemolytic reactions due to withdrawal of eculizumab were identified. Complete donor engraftment was observed after transplantation, and AML remained in remission. Use of allogeneic hematopoietic stem cell transplantation for PNH is the only treatment that can lead to complete recovery in patients who have developed hematopoietic deficiency and malignancy. The effects of eculizumab on allogeneic hematopoietic stem cell transplantation are unclear, but engraftment was immediately achieved and no serious graft-versus-host disease or infection occurred in this case. The utility and safety of using eculizumab with allogeneic hematopoietic stem cell transplantation needs to be determined by accumulating additional cases.