Compassionate use of sorafenib in FLT3-ITD-positive acute myeloid leukemia: sustained regression before and after allogeneic stem cell transplantation - PubMed (original) (raw)
Case Reports
. 2009 Jun 25;113(26):6567-71.
doi: 10.1182/blood-2009-03-208298. Epub 2009 Apr 23.
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- PMID: 19389879
- DOI: 10.1182/blood-2009-03-208298
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Case Reports
Compassionate use of sorafenib in FLT3-ITD-positive acute myeloid leukemia: sustained regression before and after allogeneic stem cell transplantation
Stephan Metzelder et al. Blood. 2009.
Free article
Abstract
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.
Comment in
- Toxic effects of sorafenib when given early after allogeneic hematopoietic stem cell transplantation.
Yokoyama H, Lundqvist A, Su S, Childs R. Yokoyama H, et al. Blood. 2010 Oct 14;116(15):2858-9. doi: 10.1182/blood-2010-06-291104. Blood. 2010. PMID: 20947691 Free PMC article. No abstract available.
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