2019 Volume 56 Issue 2 Pages 198-201
A 10-year-old girl was admitted to the hospital because of fever and headache. A blood examination revealed leukocytosis with blasts. Bone marrow examination confirmed the diagnosis of acute myeloid leukemia (FAB-M2). She harbored an FLT3 internal tandem duplication mutation, so she required hematopoietic stem cell transplantation. After 4 courses of chemotherapy, she underwent cord blood transplantation and achieved complete remission (CR). The conditioning regimen consisted of cyclophosphamide (120 mg/kg body weight) and total body irradiation (12 Gy), and the prophylaxis for graft-versus-host disease consisted of tacrolimus and short-term methotrexate. Engraftment was performed on day 32. Bone marrow examination on day 35 revealed CR, but peripheral Wilms’ tumor 1 (WT1) mRNA was detected on day 34. She received sorafenib (oral FLT3 inhibitor, 200 mg/kg body weight) daily from day 40 as maintenance therapy. Subsequently, she remained on CR and was negative for WT1 mRNA for 2 years under daily sorafenib maintenance therapy. In this patient, sorafenib maintenance therapy appeared safe and effective.