New drug-chemotherapy combination after stem cell transplant linked to ‘favorable survival outcomes’ in group of rare blood cancers

Maintenance treatment with eprenetapopt, a new drug, plus Vidaza (azacitidine) chemotherapy after allogeneic hematopoietic stem cell transplantation was associated with better survival outcomes in a group of patients with high-risk acute myeloid leukemia TP53 and of myelodysplastic syndromes, according to new study findings.

Disease relapse is the main reason for the failure of allogeneic hematopoietic stem cell transplants, according to the study authors, who noted that new stem cell transplant strategies to reduce the risk of disease relapse are absolutely necessary.

It should be noted that an allogeneic hematopoietic stem cell transplant is a procedure in which a cancer patient receives healthy hematopoietic stem cells from a donor to replace their own stem cells that have been damaged by other treatments. against cancer such as radiotherapy or chemotherapy.

“Currently, maintenance strategies (which are used to reduce the risk of disease recurrence) are not the standard of care in allogeneic (hematopoietic stem cell transplantation) for (acute myeloid leukemia and myelodysplastic syndromes),” writes the authors of the study.

In preclinical trials, eprenetapopt – a small molecule drug that can reactivate mutant p53 proteins – was active alongside Vidaza. Additionally, results from previous studies have shown the combination to be safe and effective in patients with other forms of blood cancers.

Accordingly, the researchers sought to see if maintenance treatment with eprenetapopt and Vidaza would induce similar results in this high-risk patient population.

They conducted a multicenter phase 2 trial to analyze whether post-transplant eprenetapopt plus Vidaza improves relapse-free survival (i.e. the length of time after the end of primary treatment that a patient survives without signs or symptoms of this cancer). Based on previous results, the study researchers determined that a one-year relapse-free survival rate of 50% or greater would be considered a success.

A total of 84 patients were screened to determine if they were eligible for allogeneic hematopoietic stem cell transplantation. Of the 55 patients who received a transplant, 33 patients (14 with acute myeloid leukemia and 19 with myelodysplastic syndromes) received maintenance treatment with eprenetapopt and Vidaza.

At a median follow-up of 14.5 months, maintenance treatment was associated with a median relapse-free survival of 12.5 months and an estimated one-year relapse-free survival rate of 59.9%. In addition, the median overall survival (time between treatment and death from any cause) was 20.6 months for a median follow-up of 17 months. The one-year overall survival rate was also estimated to be 78.8%.

Some of the most common serious or worse side effects in patients included decreased white blood cell count (36%), anemia (27%) and hypertension (12%). There were four severe cases of fever and two severe cases of febrile neutropenia (fever during a period of low white blood cell count) and difficult or labored breathing.

There were a few occurrences of dose reduction (9%), interruption (6%) or delay (6%) due to treatment-related side effects.

Two patient deaths were reported during the study period – one associated with a side effect unrelated to study treatment and the other attributed to disease progression more than 30 days after completion. of the study treatment.

“The TP53 mutation is associated with a poor prognosis in (acute myeloid leukemia) and (myelodysplastic syndromes), even in patients who undergo allogeneic (hematopoietic stem cell transplantation),” the study authors wrote. “Post-(hematopoietic stem cell transplantation) maintenance treatment with eprenetapopt plus (Vidaza) in this high-risk population of patients with mutant TP53 (acute myeloid leukemia) or (myelodysplastic syndromes) led to survival outcomes These data support future exploration of this maintenance strategy in a Phase 3 study… of eprenetapopt plus (Vidaza) versus placebo plus (Vidaza) in patients with TP53 mutant myeloid malignancies.

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