持续地西他滨/全反式维甲酸治疗：延长一名患有多基因TP53病变和复杂单体核型的老年急性髓细胞性白血病患者的完全缓解期

IF 5.4 3区材料科学 Q2 CHEMISTRY, PHYSICAL ACS Applied Energy Materials Pub Date : 2024-09-11 DOI:10.1186/s13148-024-01737-4

Johanna Thomas, Usama-Ur Rehman, Helena Bresser, Olga Grishina, Dietmar Pfeifer, Etienne Sollier, Konstanze Döhner, Christoph Plass, Heiko Becker, Claudia Schmoor, Maike de Wit, Michael Lübbert

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引用次数: 0

摘要

DNA-甲基化药物（HMAs）可显著提高TP53突变的急性髓细胞性白血病/骨髓增生异常综合症患者的缓解率；然而，继发性耐药性往往发展迅速。在 DECIDER 试验（NCT00867672）中，老年急性髓细胞性白血病患者（也包括具有不良遗传学的患者）随机接受全反式维甲酸（ATRA）联合地西他滨（DEC）治疗后，产生耐药性的时间明显推迟。一位 82 岁的患者患有非破坏性、框架内 TP53 突变（p.Cys238_Asn239delinsTyr，VAF 90%）和复杂单体核型，在接受 DEC + ATRA 治疗后，血液学和细胞遗传学完全缓解，30 个治疗周期后存活 3.7 年，且耐受性良好。对于不符合接受更多强化治疗条件的不同遗传风险组别 AML/MDS 患者，似乎有必要进一步开展 HMA + ATRA 研究。试验注册：该试验已在 ClinicalTrials.gov 注册，标识符为NCT00867672

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Continued decitabine/all-trans retinoic acid treatment: extended complete remission in an elderly AML patient with multi-hit TP53 lesions and complex-monosomal karyotype

DNA-hypomethylating agents (HMAs) induce notable remission rates in AML/MDS patients with TP53 mutations; however, secondary resistance often develops rapidly. In the DECIDER trial (NCT00867672), elderly AML patients (also those with adverse genetics) randomized to all-trans retinoic acid (ATRA) added to decitabine (DEC) attained significantly delayed time-to-resistance. An 82-year-old patient with a non-disruptive, in-frame TP53 mutation (p.Cys238_Asn239delinsTyr, VAF 90%) and complex-monosomal karyotype attained a complete hematologic and cytogenetic remission with DEC + ATRA, with 3.7 years survival after 30 treatment cycles that were well-tolerated. Further HMA + ATRA studies appear warranted in AML/MDS patients of different genetic risk groups ineligible for more intensive treatment. Trial registration: This trial was registered at ClinicalTrials.gov identifier: NCT00867672

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来源期刊

ACS Applied Energy Materials Materials Science-Materials Chemistry

CiteScore

10.30

自引率

6.20%

发文量

1368

期刊介绍： ACS Applied Energy Materials is an interdisciplinary journal publishing original research covering all aspects of materials, engineering, chemistry, physics and biology relevant to energy conversion and storage. The journal is devoted to reports of new and original experimental and theoretical research of an applied nature that integrate knowledge in the areas of materials, engineering, physics, bioscience, and chemistry into important energy applications.