A study published recently in The New England Journal of Medicine has demonstrated that acute myeloid leukemia (AML) patients with disease that harbors TP53 mutations may respond well to the chemotherapy drug decitabine.
In this single institution trial, researchers from the Washington University School of Medicine in St. Louis (MO, USA) sought to identify the effect of TP53 mutations on AML response to decitabine.
The team sequenced the genetic profiles of 116 AML patients and highlighted that those individuals who responded better to decitabine also had a mutation in TP53 – a form of AML which usually correlates with an extremely poor prognosis.
Lead author Timothy J Ley (Washington University in St. Louis) commented: “What’s really unique here is that all the patients in the study with TP53 mutations had a response to decitabine and achieved an initial remission. With standard aggressive chemotherapy, we only see about 20–30% of these patients achieving remission, which is the critical first step to have a chance to cure patients with additional therapies.”
Ley concluded: “The findings need to be validated in a larger trial but they do suggest that TP53 mutations can reliably predict responses to decitabine, potentially prolonging survival in this ultra-high-risk group of patients and providing a bridge to transplantation in some patients who might not otherwise be candidates.”
The group are planning to conduct a larger trial to evaluate the efficacy of decitabine in AML patients of all ages who carry TP53 mutations.
Sources: Welch JS, Petti AA, Miller CA et al.TP53 and Decitabine in Acute Myeloid Leukemia and Myelodysplastic Syndromes. N. Engl. J. Med. 375, 2023–2036 (2016); https://medicine.wustl.edu/news/highly-lethal-type-leukemia-cancer-gene-predicts-treatment-response/