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

2017
Sorafenibis a promising agent for treating pediatric refractoryacute 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 refractorypediatric 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 sorafenibsuccessfully achieved complete remission in case 2. This patient received post-SCT sorafeniband remains in complete remission. The combination of pre-SCT and post-SCT sorafenibmay thus be effective for pediatric refractoryFLT3-ITD-positive AML.
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