Sorafenib Therapy for Pediatric Acute Myeloid Leukemia with FMS-like Tyrosine Kinase 3-internal Tandem Duplication Mutations: 2 Case Reports

J Pediatr Hematol Oncol. 2017 May;39(4):e199-e202. doi: 10.1097/MPH.0000000000000672.

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.

Publication types

  • Case Reports

MeSH terms

  • Child
  • Child, Preschool
  • Drug Administration Schedule
  • Female
  • Hematopoietic Stem Cell Transplantation / methods*
  • Humans
  • Leukemia, Myeloid, Acute / drug therapy*
  • Leukemia, Myeloid, Acute / genetics
  • Male
  • Mutation
  • Niacinamide / administration & dosage
  • Niacinamide / analogs & derivatives*
  • Phenylurea Compounds / administration & dosage*
  • Salvage Therapy / methods
  • Sorafenib
  • Tandem Repeat Sequences
  • Treatment Outcome
  • fms-Like Tyrosine Kinase 3 / genetics*

Substances

  • Phenylurea Compounds
  • Niacinamide
  • Sorafenib
  • fms-Like Tyrosine Kinase 3