Improving Pediatric Outcomes in Oncology through Genomics

Within the past decade, target therapies have been prescribed using genomic data to more precisely diagnose cancer and predict future outcomes. The pediatric field is lacking by not targeting mutated oncogenic drivers and rare ALK translocated malignancies. Pediatric cancer does not have as many mutations, and many studies on focused on the disease state. New evidence shows that post therapy relapse samples accumulate more mutations and may lead to chemotherapy resistance.

A study with 102 children and young adults (up to age 22) involved exam sequencing of paired blood mononuclear cell and tumor DNAs along with tumor RNA sequencing. 69% had solid tumors. The study was designed to identify germ-line mutations that could cause an effect, tumor-specific alterations that would alter the histopathologic diagnosis, change risk status or both, and medically targetable somatic mutations.

Potentially actionable findings were found in 46% of cases and action was taken in 23 of those 42 patients (54%). A change of therapy was initiated for 14 patients (15%), a gremlin mutation was found to be clinically relevant in 9 patients, and 9 of the 14 patients had clinical benefit from the intervention.

This data is very relevant and suggests a promising field of research. With genomic sequencing, therapies were personalized in order to better target the cancer. As pharmacists, we should consider this and acknowledge that patients may require different treatment even with the same disease state. The research and sequencing in adults can and should be applied to children to better care for their cancer.

JAMA. 2015;314(9):881-883. 

1 thought on “Improving Pediatric Outcomes in Oncology through Genomics”

  1. This is really interesting to me because I would have thought that sequencing would have been a commonality for pediatric cancer. Pediatrics are extremely different that adult but thats what makes sequencing such a good option. I completely agree with you that we need to consider each patient as a different treatment plan. Targeting cancer such an interesting topic because each patient is a new puzzle that the medical team must try and solve. We as pharmacists have such a hard job with determining potential drug targets of the cancer, and I think that sequencing of the cancer would be very beneficial to us because we can see where the mutations are to target. Overall sequencing is, in my opinion, very beneficial for treatment of pediatric cancer.

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