The Future of Treatment for Children with Acute Myeloid Leukemia and TP53 Gene Mutation

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Dr. Adam Lamble recommends that pediatric patients with AML and TP53 status should now be considered for transplant in first remission, and novel interventions are warranted.

In an interview with HCPLive, Adam Lamble, MD, Attending Physician, Assistant Professor of Pediatrics, Cancer & Blood Disorders Center, High-Risk Leukemia Program, Leukemia & Lymphoma Program, Seattle Children's Hospital, shared what's anticipated for the future of acute myeloid leukemia (AML) research following his investigation of TP53 mutations in the pediatric AML population.

The retrospective study was the largest investigation to evaluate the TP53 gene mutation in children with the disease. The results confirmed how rare the mutation is in this population (1.5%), and supported classifying this subgroup as high-risk, according to Lamble.

"These patients need to be considered for transplant in first remission, which they're not necessarily being considered for at this time," he stated. "Then, I think that these patients also warrant novel interventions, at least in the relapse setting."

Data exhibited alarmingly poor outcomes for these children when compared with children who had AML but did not have the TP53 status. While many features of this disease is more common and well understood in adults with AML, evidence for pediatric patients is lacking.

"This is the accumulation of 4 different clinical trials, probably over the last 20 years, just to get these 26 patients, or 1.5%, of patients that had TP53," Lamble explained. "And so, I think that we're very fortunate that pediatric AML is as rare as it is, but I think that it has always really limited the amount of investigations."

The next step would be to validate the retrospective data in a prospective setting to confirm TP53 is a high-risk mutation in pediatric patients with acute myeloid leukemia. However, he also emphasized the need to start interventions in the clinical setting.