Use of tamibarotene, a potent and selective RARα agonist, in combination with azacitidine in patients with relapsed and refractory AML with RARA gene overexpression.
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| 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 (RARA) gene overexpression. Approximately, 50% of higher-risk myelodysplastic syndrome (MDS) patients and approximately 30% of AML patients are positive for RARA overexpression using a blood-based biomarker test that measures RARA expression in peripheral blasts. A phase 2 study investigating the activity of tamibarotene in patients with RARA overexpression was conducted in patients with AML and MDS (NCT02807558). In 28 patients with R/R AML and RARA 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 RARA overexpression.
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| Authors | Eytan M Stein, Stephane de Botton, Thomas Cluzeau, Arnaud Pigneux, Jane L Liesveld, Rachel J Cook, Philippe Rousselot, David A Rizzieri, Thorsten Braun, Gail J Roboz, Delphine Lebon, Mael Heiblig, Kristen Baker, Angela Volkert, Sofia Paul, Nisha Rajagopal, David A Roth, Michael Kelly, Pierre Peterlin |
| Journal | Leukemia & lymphoma (Leuk Lymphoma) Vol. 64 Issue 12 Pg. 1992-2001 (12 2023) ISSN: 1029-2403 [Electronic] United States |
| PMID | 37571998 (Publication Type: Journal Article, Research Support, Non-U.S. Gov't) |
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