Experimental Treatment Combination Shows Efficacy in Acute Myeloid Leukemia and Myelodysplastic Syndromes


Researchers acknowledge that the primary cause of failure of stem cell transplants is disease relapse. This holds true specifically for allogeneic hematopoietic stem cell transplants where a cancer patient receives a donor’s healthy stem cells.

As reported in CureToday, patients with high-risk TP53-mutated acute myeloid leukemia and myelodysplastic syndromes welcome the news that a new study finds post-transplant treatment with azacytidine (Vidaza) and a novel drug called eprenetapopt resulted in progression-free survival. Eprenetapopt is a medication that reactivates mutant p53 proteins.


About the Study

A phase II trial was initiated to investigate whether Vidaza and eprenetapopt improved the odds of relapse-free survival (RFS). The study researchers determined that the drug combination would be considered successful after a period of one year if it evidenced a fifty percent survival rate. That should mean that the patients were alive one year hence with no signs of cancer.

Eighty-four patients were initially screened but only fifty-five patients received the stem cell transplant. Then, thirty-three patients were treated for purposes of maintenance with the combination of Vidaza and eprenetapopt.

The follow-up was approximately fourteen months for each patient. Results of maintenance therapy were 12.5 months of  RFS. The cumulative one-year survival rate, relapse-free, was 59.9%. The overall rate of survival for one year was approximately seventy-eight percent for any cause.

About Adverse Events

Frequent side effects were a decreased number of white blood cells, anemia, fever, and hypertension.

Two patients died. One death was unrelated to the study and the second patient died of disease progression after study completion.

In conclusion, the study authors explained that the prognosis accompanying the TP53 mutation is poor. However, post-maintenance treatment with Vidaza plus eprenetapopt led to positive survival outcomes. Data suggest that a phase 3 trial is recommended for a future study of patients with TP53 mutant myeloid malignancies.

Rose Duesterwald

Rose Duesterwald

Rose became acquainted with Patient Worthy after her husband was diagnosed with Acute Myeloid Leukemia (AML) six years ago. During this period of partial remission, Rose researched investigational drugs to be prepared in the event of a relapse. Her husband died February 12, 2021 with a rare and unexplained occurrence of liver cancer possibly unrelated to AML.

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