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

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2024年9月11日

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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

摘要

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