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Molecular Profiling Feasible for Recurrent Pediatric Cancer

69 percent of patients had at least one genetic alteration leading to targeted treatment suggestion

WEDNESDAY, March 16, 2022 (HealthDay News) — Molecular profiling is feasible for cancer recurrence among pediatric patients, according to a study published online March 16 in Cancer Discovery.

Pablo Berlanga, M.D., Ph.D., from the Université Paris-Saclay, and colleagues conducted a prospective precision medicine trial aiming to define tumor molecular profiles in 787 pediatric patients with recurrent/refractory malignancies from February 2016 to July 2020.

The researchers found that in 69 percent of patients, at least one genetic alteration leading to targeted treatment suggestion was identified with successful sequencing; 10 percent of these were considered to be ‘ready for routine use’. A total of 356 patients had follow-up beyond 12 months; 30 percent of those patients received one or more matched targeted therapies, 56 percent within early clinical trials. The objective response rate was 17 percent for matched treatment and was 38 percent among patients with ‘ready for routine use’ alterations. Seventy-six percent of actionable alterations detected in tumor tissue were also identified in circulating cell free DNA among patients with extra-cerebral tumors.

“The sequencing efforts on pediatric tumors at relapse underscore the low proportion of high level evidence or ‘ready for routine use’ alterations,” the authors write. “This strongly underlines that high-throughput molecular profiling together with matched targeted therapies, the innovative clinical trials, the development of new technologies that allow cancer characterization beyond the genetic level, the development of combination strategies, and analysis of the personal immune response of each patient are needed to overcome the current limitations.”

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