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Article Abstract
Tamibarotene-based therapy is a novel targeted approach for the treatment of relapsed/refractory (R/R) acute myeloid leukemia (AML) with retinoic acid receptor alpha () gene overexpression. Approximately, 50% of higher-risk myelodysplastic syndrome (MDS) patients and approximately 30% of AML patients are positive for overexpression using a blood-based biomarker test that measures expression in peripheral blasts. A phase 2 study investigating the activity of tamibarotene in patients with overexpression was conducted in patients with AML and MDS (NCT02807558). In 28 patients with R/R AML and overexpression treated with tamibarotene in combination with azacitidine, the median overall survival was 5.9 months. In 21 response-evaluable patients, the complete remission/complete remission with incomplete hematologic recovery (CR/CRi) rate was 19%, and median time to initial CR/CRi was 1.2 months. The favorable safety profile and preliminary clinical activity support the development of combination therapies with tamibarotene in myeloid malignancies with overexpression.
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| http://dx.doi.org/10.1080/10428194.2023.2243356 | DOI Listing |
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