Journal
BLOOD
Volume 113, Issue 26, Pages 6567-6571Publisher
AMER SOC HEMATOLOGY
DOI: 10.1182/blood-2009-03-208298
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Funding
- Deutsche Jose Carreras Leukamie-Stiftung eV [DJCLS 06/01v]
- Deutsche Forschungsgemeinschaft [KFO210, BU 1321/3-1, NE 310/14-1]
- University Medical Center Giessen and Marburg
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Acute myeloid leukemia (AML) patients with internal tandem duplication (ITD) mutations in the Fms-like tyrosine-3 (FLT3) gene have a dismal prognosis. Here we report compassionate-use results with the multikinase and FLT3-ITD inhibitor sorafenib for the treatment of relapsed or refractory FLT3-ITD-positive AML. Sorafenib induced clinically meaningful and very rapid responses in all 6 patients treated either before (n = 2), after (n = 3), or both before and after (n = 1) allogeneic stem cell transplantation (allo-SCT). Sorafenib-induced remissions facilitated allo-SCT in 2 of the 3 refractory patients. Two of the 4 patients who were treated after allo-SCT survived 216 and 221 days, respectively, whereas the other 2 remain in ongoing complete molecular remission. Sorafenib response was associated with an inhibition of the antiapoptotic FLT3-ITD target Stat-5 in vivo. Together, sorafenib monotherapy before or after allo-SCT has remarkable clinical activity in poor risk FLT3-ITD-positive AML and deserves further evaluation in prospective clinical trials. (Blood. 2009; 113: 6567-6571)
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