In vitro assessment of the sensitivity to APR‐246 + azacitidine combination predicts response to this combination in myelodysplastic/acute myeloid leukaemia patients.

Nineteen of these 33 patients had reached CR after three to six cycles of treatment, while 14 patients had failed to achieve CR (nCR). Keywords: TP53; MDS; AML; treatment; prognosis EN TP53 MDS AML treatment prognosis e77 e79 3 08/18/21 20210815 NES 210815 I TP53 i mutations, which abrogate normal p53 protein functions and may also induce deleterious "gain of function" of the protein, are a poor prognostic factor in myelodysplastic syndromes (MDS) and acute myeloid leukaemia (AML), especially when mutations are biallelic.1 Restoration of mutant p53 normal functions in those cases, especially by protein "reconformation", has been considered a relevant strategy and many drugs have been tested.2 APR-246 (Eprenetapopt) is a pro-drug that induces structural changes to mutant p53 and restores the active conformation of the protein.3 We recently reported the I in vitro i and I in vivo i synergy of azacitidine (AZA) in combination with APR-246. In vitro assessment of the sensitivity to APR-246 + azacitidine combination predicts response to this combination in myelodysplastic/acute myeloid leukaemia patients. [Extracted from the article]