Multicenter phase 2 trial of post HSCT eprenetapopt plus azacitidine maintenance for TP53-mutant AML and MDS

Patients with TP53-mutant AML and MDS have a poor prognosis, even after allogeneic hematopoietic stem-cell transplant (HSCT). Eprenetapopt (APR-246) is a first-in-class, small-molecule p53 reactivator. A multicenter phase 2 trial in the USA evaluated maintenance therapy with eprenetapopt plus azacitidine after HSCT for TP53-mutant AML or MDS. The maintenance therapy was well tolerated. Relapse-free survival and overall survival outcomes were encouraging in this high-risk population. The authors conclude that maintenance therapy with the eprenetapopt and azacitidine combination has the potential to improve post-HSCT outcomes in patients with TP53-mutant AML or MDS.