[Unrelated bone marrow transplantation for acute myeloid leukemia evolved from paroxysmal nocturnal hemoglobinuria].

Abstract

Paroxysmal nocturnal hemoglobinuria (PNH) is a hematopoietic stem cell disease that results from clonal expansion of hematopoietic stem cells with gene mutations. Patients with PNH are known to have an increased risk of developing myelodysplastic syndromes (MDS) and acute myeloid leukemia (AML). We report a case of a 42-year-old woman diagnosed with AML 13 years after a diagnosis of PNH. Gene mutations associated with MDS were detected. She did not achieve complete remission (CR) after induction therapy with idarubicin and cytarabine. Unrelated bone marrow transplantation was performed with a myeloablative conditioning regimen with cyclophosphamide (120 mg/kg) and total body irradiation (12 Gy). Ravulizumab was administered until 47 days after transplantation. She achieved CR after transplantation with complete donor engraftment. The transplantation was successful without severe complications such as graft-versus-host disease or sinusoidal obstruction syndrome. Further accumulation of cases is necessary to determine the efficacy and safety of anti-complement inhibitors in allogeneic hematopoietic stem cell transplantation.