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.
증례보고
2/5 보강
PICO 자동 추출 (휴리스틱, conf 2/4)
유사 논문P · Population 대상 환자/모집단
환자: early molecular progression (AML1-MTG16 burden elevated to 0
I · Intervention 중재 / 시술
추출되지 않음
C · Comparison 대조 / 비교
추출되지 않음
O · Outcome 결과 / 결론
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.
OpenAlex 토픽 ·
Acute Myeloid Leukemia Research
Retinoids in leukemia and cellular processes
Acute Lymphoblastic Leukemia research
ℹ️ 이 논문은 무료 전문이 아직 없습니다. 코퍼스 전체의 43.6%는 무료 가능 (통계 →) · 🏥 기관 EZproxy로 시도
[BACKGROUND] Acute myeloid leukemia (AML) with rare aberrations like AML1-MTG16 fusion encounters precision oncology challenges, exacerbated by the "Long-Tail" dilemma.
APA
Kuangguo Zhou, Zhiqiong Wang, et al. (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.. Clinical therapeutics. https://doi.org/10.1016/j.clinthera.2026.03.015
MLA
Kuangguo Zhou, et al.. "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.." Clinical therapeutics, 2026.
PMID
41986207 ↗
Abstract 한글 요약
[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.
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