Sorafenib Therapy for Pediatric Acute Myeloid Leukemia with FMS-like Tyrosine Kinase 3-internal Tandem Duplication Mutations: 2 Case Reports. Issue 4 (May 2017)

• Record Type: Journal Article
• Authors: Osone, Shinya; Imamura, Toshihiko; Kanayama, Takuyo; Tsuma, Yusuke; Kawashima-Goto, Sachiko; Nakatani, Takuya; Sugimoto, Atsuya; Takai, Akari; Miyachi, Mitsuru; Tamura, Shinichi; Ishida, Hiroyuki; Hosoi, Hajime
• Is Part Of: Journal of pediatric hematology/oncology. Volume 39:Issue 4(2017)
• Publication Date: 2017-05
• Subjects: acute myeloid leukemia -- sorafenib -- FLT3-ITD -- stem cell transplantation -- children; Pediatric hematology -- Periodicals; Tumors in children -- Periodicals; 618.9215
• DOI: 10.1097/MPH.0000000000000672
• Languages: English
• ISSNs: 1077-4114

Abstract

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.