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Updated: May 27, 2026

Pre-clinical Evaluation of Tyrosine Kinase Inhibitors for Treatment of Acute Leukemia
Published on: September 18, 2013
Sorafenib as treatment for relapsed or refractory pediatric acute myelogenous leukemia
1Department of Pediatrics, Division of Hematology/Oncology, Emory University School of Medicine and Children's Healthcare of Atlanta, Atlanta, Georgia, USA.
Abstract:
The prognosis for children with acute myelogenous leukemia (AML) has improved with overall survival rates of up to 65% [Pui et al. J Clin Oncol 2011; 29: 551-565]. However, the cure rate for AML lags behind that of acute lymphoblastic leukemia. Advances in AML leukemogenesis are leading to refined risk stratification. FMS like tyrosine kinase 3 (FLT3) mutations are independently associated with a poor prognosis. Newer kinase inhibitors, including sorafenib, have shown promise in adult studies. We report three pediatric patients with relapsed AML who achieved a sustained remission with sorafenib. Further trials are necessary to understand the role of sorafenib in pediatric AML.
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