title: [Successful treatment with sorafenib for primary refractory acute monoblastic leukemia with FLT3-ITD].
abstract: A 6-year-old boy was diagnosed with FLT3-ITD+acute monoblastic leukemia (AMoL). He showed resistance to 2 cycles of induction chemotherapy with etoposide, cytarabine, and mitoxantrone or idarubicin performed according to the Japan Pediatric Leukemia/Lymphoma Study Group (JPLSG) AML-05 protocol. His condition was also refractory to salvage FLAI-GO (fludarabine, cytarabine, idarubicin, and gemtuzumab ozogamicin) chemotherapy. Sequential administration of sorafenib at doses of up to 300 mg/day resulted in the first remission. He underwent bone marrow transplantation from his HLA 2-locus mismatched father. Recurrence was observed on post-transplantation day 71. A sustained partial response was observed after alternate-day readministration of sorafenib 150mg/day. In spite of a donor lymphocyte infusion, his blast cell count increased on day 245. Chemotherapy with an increased dose of sorafenib reduced the blast cell count. Although a second HLA-mismatched allogeneic peripheral blood stem cell transfusion was performed, the patient died of regimen-related toxicity. Herein, we report a pediatric case of primary refractory FLT3-ITD+ AMoL. Further prospective studies are necessary to validate the efficacy of sorafenib treatment.
authors.affiliation: Department of Pediatrics, Yamagata University, Japan.
authors.forename: Noriyuki
authors.initials: N
authors.lastname: Takahashi
authors.forename: Takako
authors.initials: T
authors.lastname: Kawakami
authors.forename: Hiroko
authors.initials: H
authors.lastname: Sato
authors.forename: Tadashi
authors.initials: T
authors.lastname: Onoda
authors.forename: Miyako
authors.initials: M
authors.lastname: Kanno
authors.forename: Tetsuo
authors.initials: T
authors.lastname: Mitsui
authors.forename: Kiyoshi
authors.initials: K
authors.lastname: Hayasaka
mesh_terms: D000971:Antineoplastic Combined Chemotherapy Protocols / Q000627:therapeutic use*; D016026:Bone Marrow Transplantation / Q000009:adverse effects; D002648:Child; D017809:Fatal Outcome; D006801:Humans; D055029:Inverted Repeat Sequences; D007948:Leukemia, Monocytic, Acute / Q000188:drug therapy* / Q000276:immunology / Q000628:therapy; D008297:Male; D009536:Niacinamide / Q000009:adverse effects / Q000031:analogs & derivatives* / Q000627:therapeutic use; D010671:Phenylurea Compounds / Q000009:adverse effects / Q000627:therapeutic use*; D000077157:Sorafenib; D051941:fms-Like Tyrosine Kinase 3 / Q000032:analysis
chemical_list: D010671:Phenylurea Compounds; D009536:Niacinamide; D000077157:Sorafenib; C497970:FLT3 protein, human; D051941:fms-Like Tyrosine Kinase 3