Clinical therapeutics · 2026
Mechanism-Driven Repurposing of All-Trans Retinoic Acid (ATRA) for AML1-MTG16⁺ Acute Myeloid Leukemia: A First-in-Human Case Report and Translational Roadmap to Overcome the "Long-Tail" Barrier.
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Zhou Kuangguo, Wang Zhiqiong, Xiong Jie, Huang Wei
Аннотация
BACKGROUND: Acute myeloid leukemia (AML) with rare aberrations like AML1-MTG16 fusion encounters precision oncology challenges, exacerbated by the "Long-Tail" dilemma. Preclinical data show all-trans retinoic acid (ATRA) restores myeloid differentiation in MTG16-deficient AML models, but its clinical translation remains unexplored.
CASE PRESENTATION: We treated a 73-year-old unfit AML patient with early molecular progression (AML1-MTG16 burden elevated to 0.2%) using ATRA plus azacitidine/venetoclax. Sustained measurable residual disease-negative remission (21 months) and undetectable fusion transcripts were achieved with only grade 2 neutropenia.
CONCLUSIONS: This case may preliminarily suggest ATRA's potential in AML1-MTG16⁺ AML, offering translational clues for ultra-rare leukemia subtypes, pending further validation.
CASE PRESENTATION: We treated a 73-year-old unfit AML patient with early molecular progression (AML1-MTG16 burden elevated to 0.2%) using ATRA plus azacitidine/venetoclax. Sustained measurable residual disease-negative remission (21 months) and undetectable fusion transcripts were achieved with only grade 2 neutropenia.
CONCLUSIONS: This case may preliminarily suggest ATRA's potential in AML1-MTG16⁺ AML, offering translational clues for ultra-rare leukemia subtypes, pending further validation.