Post-Transplant Eprenetapopt Plus Azacitidine Maintenance Shows Promise in TP53-Mutant AML and MDS

In a phase 2 trial published in the Journal of Clinical Oncology, post-transplant eprenetapopt and azacitidine maintenance demonstrated promising safety and efficacy with the potential to improve outcomes in patients with TP53-mutant acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS), according to the findings of Asmita Mishra, MD, Houston Lee Moffitt Cancer Center, Tampa, FL and colleagues.

Patients received up to 12 cycles of intravenous eprenetapopt at a dose of 3.7 g once daily on days 1 to 4 combined with intravenous/subcutaneous azacitidine at a dose of 36 mg/m2 once daily on days 1 to 5 in 28-day cycles.

The primary end points of the trial were relapse-free survival (RFS) and safety.

A total of 84 patients were screened for eligibility before undergoing hematopoietic stem cell transplantation (HCT). Of these patients 55 received a transplant and 33 proceeded to maintenance therapy. The median number of cycles of eprenetapopt was 7, with a range of 1 to 12.

With a median follow-up of 14.5 months, the median RFS was 12.5 months (95% confidence interval [CI], 9.6 to not estimable [NE]) and the 1-year RFS probability was 59.9% (95% CI, 41 to 74). With a median follow-up of 17 months, the median overall survival (OS) was 20.6 months (95% CI, 14.2 to NE) and the 1-year OS probability was 78.8% (95% CI, 60.6 to 89.3).

Researchers noted the 30-day and 60-day mortalities from the first dose were 0% and 6% (n = 2), respectively. Acute and chronic (all grade) graft-versus-host disease adverse events were reported in 12% (n = 4) and 33% (n = 11) of patients, respectively.

“In patients with TP53[-mutant] AML and MDS, post-HCT maintenance therapy with eprenetapopt combined with azacitidine was well-tolerated,” Dr Mishra and coauthors concluded. “RFS and OS outcomes were encouraging in this high-risk population.”

Source:

Mishra A, Tamari R, DeZern AE, et al. Eprenetapopt Plus Azacitidine After Allogeneic Hematopoietic Stem-Cell Transplantation for TP53-Mutant Acute Myeloid Leukemia and Myelodysplastic Syndromes. J Clin Oncol. Published online July 11, 2022. doi:10.1200/jco.22.00181