Risks and benefits of phase I trials in pediatric cancer patients

On an average, one out of ten children with pediatric phase I cancer improve after being treated for the illness. But one out of fifty children succumb to drug-related complications. This was published in a systematic review and meta-analysis in PLOS Medicine. In phase I clinical trials, the safety and dosage of anti-cancer drugs was determined.

According to national and international regulations, limits on permissible risk were determined with respect to minors. Researchers systematically scoured pediatric phase I cancer studies, which were published between 2004 and 2015. They identified 170 studies that included 4,604 patients. They determined objective response rates and graded intensities as follows: 3, 4, or 5 (fatal). These caused adverse reactions of drugs.

Among all clinical trials, the overall response rate was 10.29% (95% CI 8.33 to 12.25). The overall response rate for solid tumors (3.17, 95% CI 2.62 to 3.72) was significantly greater than that for hematological malignancies (27.90, 95% CI 20.53 to 35.27).

The overall rate of fatal grade 5 adverse events was 2.09% (95% CI 1.45 to 2.72). An average response rate of 1.32 was reported for grades 3 and 4, which were drug-related adverse events per person. The adverse events and response rates were similar to those observed in adults that participated in phase I cancer trials.

This study has following limitations: heterogeneous types of cancer and treatment in included trials, reliance on only published data, and low-quality or incomplete reports of some clinical trial outcomes.

The data was coupled with careful ethical analysis, providing an empirical basis for further discussions on therapeutic status of phase I clinical trials in pediatric cancer patients. They provided evidence for refining risk/benefit in phase I trials and for identifying studies, which present greater challenges that meet standards of acceptable risk in children.


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