Results of a study of molecular tumor profiling in young patients revealed a high rate of genetic alterations with potential for impacting clinical care, including clarifying diagnoses and treatment with matched, precision cancer drugs.

Reporting in Nature Medicine, researchers from Dana-Farber/Boston Children’s Cancer and Blood Disorders Center said molecular profiling of solid tumors found clinically significant genetic variations in 298, or 6% of 345 pediatric patients. In 240 patients, the genetic “fingerprint” or pattern of cancer-related changes in the tumor’s DNA could be used to choose a targeted, precision therapy matched to those alterations. Of these patients, 200 were eligible for matched drug therapy.

Targeted therapies were used to treat 29 patients, and 24% of the patients responded to the targeted drugs or experienced a durable clinical benefit. In addition, the molecular profiling – done by a process called next-generation sequencing – clarified the diagnostic classification in 17 patients.

Source: Molecular tumor profiling in pediatric cancer patients significantly impacts clinical care

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