Adam Lamble, MD: We Could Do Better Treating Pediatric Acute Myeloid Leukemia

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Data from the largest investigation of TP53 mutations in pediatric patients with acute myeloid leukemia (AML) is presented at the ASH 2022 Annual Meeting.

A study from the American Society of Hematology (ASH) Annual Meeting and Exposition presented by lead investigator Adam Lamble, MD, Attending Physician, Assistant Professor of Pediatrics, Cancer & Blood Disorders Center, High-Risk Leukemia Program, Leukemia & Lymphoma Program, Seattle Children's Hospital, was the largest investigation of TP53 mutations in pediatric patients with acute myeloid leukemia (AML).

"A big effort at the Children's Oncology Group, and where my work is, is trying to see if we can better define, or identify those patients that would benefit from a stem cell transplant," Lamble said in an interview with HCPLive.

By comparing disease characteristics and clinical outcomes in the patients with and without the mutation, Lamble and his team found that the rare pathogenic TP53 variants in this population are prognostically significant.

"I think that we all agree that we could do better treating AML," he said.

There are 2 main hematologic malignancies when it comes to pediatric patients: acute lymphoblastic leukemia (ALL), and acute myeloid leukemia. Some patients can be cured with chemotherapy and some need more intensive treatment and undergo hematopoietic stem cell transplantation (HSCT).

"Historically, we haven't always known who would benefit from a stem cell transplant and who wouldn't, which probably led to some patients getting a stem cell transplant when they didn't need it, and then other patients not getting it and ultimately relapsing," Lamble explained.

Cytomolecular status of the patient, or the types of mutations present in the disease, is an important factor in determining who will benefit from stem cell transplant.

"TP53 is a very commonly mutated gene, across all different types of cancer. It's probably the most commonly mutated gene, and actually, this mutation is very well described in adult patients with AML, and unfortunately, is associated with a inferior prognosis compared to patients without this type of mutation," he said. "But in pediatrics, it's a lot more rare."

According to the study abstract, the data suggest TP53 pathogenic mutations should be considered high-risk defining lesions in pediatric AML, because poorer outcomes were observed compared with patients who did not have the mutation. Further investigation is needed, but these findings helped to fill the gap of information surrounding pediatric AML.