title: Sorafenib Therapy for Pediatric Acute Myeloid Leukemia with FMS-like Tyrosine Kinase 3-internal Tandem Duplication Mutations: 2 Case Reports.
abstract: Sorafenib is a promising agent for treating pediatric refractory acute myeloid leukemia (AML) exhibiting FMS-like tyrosine kinase 3-internal tandem duplication (FLT3-ITD); however, its optimal use needs to be established. We report 2 cases of refractory pediatric FLT3-ITD-positive AML treated with sorafenib. Case 1 underwent stem cell transplantation (SCT) without entering remission, despite the use of chemotherapy. This patient relapsed despite receiving post-SCT sorafenib. Chemotherapy combined with sorafenib successfully achieved complete remission in case 2. This patient received post-SCT sorafenib and remains in complete remission. The combination of pre-SCT and post-SCT sorafenib may thus be effective for pediatric refractory FLT3-ITD-positive AML.
authors: *Department of Pediatrics, Graduate School of Medical Science, Kyoto Prefectural University of Medicine, Kyoto †Department of Pediatrics, Matsushita Memorial Hospital, Moriguchi, Japan.
authors: Shinya
authors: S
authors: Osone
authors: Toshihiko
authors: T
authors: Imamura
authors: Takuyo
authors: T
authors: Kanayama
authors: Yusuke
authors: Y
authors: Tsuma
authors: Sachiko
authors: S
authors: Kawashima-Goto
authors: Takuya
authors: T
authors: Nakatani
authors: Atsuya
authors: A
authors: Sugimoto
authors: Akari
authors: A
authors: Takai
authors: Mitsuru
authors: M
authors: Miyachi
authors: Shinichi
authors: S
authors: Tamura
authors: Hiroyuki
authors: H
authors: Ishida
authors: Hajime
authors: H
authors: Hosoi
mesh_terms: D002648:Child; D002675:Child, Preschool; D004334:Drug Administration Schedule; D005260:Female; D018380:Hematopoietic Stem Cell Transplantation / Q000379:methods*; D006801:Humans; D015470:Leukemia, Myeloid, Acute / Q000188:drug therapy* / Q000235:genetics; D008297:Male; D009154:Mutation; D009536:Niacinamide / Q000008:administration & dosage / Q000031:analogs & derivatives*; D010671:Phenylurea Compounds / Q000008:administration & dosage*; D016879:Salvage Therapy / Q000379:methods; D000077157:Sorafenib; D020080:Tandem Repeat Sequences; D016896:Treatment Outcome; D051941:fms-Like Tyrosine Kinase 3 / Q000235:genetics*
chemical_list: D010671:Phenylurea Compounds; D009536:Niacinamide; D000077157:Sorafenib; D051941:fms-Like Tyrosine Kinase 3