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 mentioned in a meta-analysis review published in PLOS Medicine. In phase I clinical trials, researchers determined how safe was the prescribed dosage of drugs used to fight cancer.
According to the guidelines of regulatory authorities in the US, limits on permissible risk were determined with respect to minors. Researchers systematically scoured the phase I clinical trials of pediatric patients, which were published from 2004 to 2015. They found that there were 170 studies related to pediatric cancer and they included a total of 4,604 patients. They objectively determined the rate of response by pediatric patients, and they graded their intensities as follows: 3, 4, or 5 (fatal). These events had led to an adverse reaction 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 tumors in the solid state (3.17, 95% CI 2.62 to 3.72) was significantly greater than that for malignancies that occurred hematologically (27.90, 95% CI 20.53 to 35.27).
The overall rate of adverse events of the grade 5 type 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 adverse events related to drugs administered to each person. The response rates and adverse events were similar to those observed in adults that participated in the phase I clinical trials of cancer patients.
This study has following limitations: we evaluated cancers of the heterogeneous type and investigated the treatment provided in the included clinical trials; we relied on only published data. We also included the outcomes of clinical trials that were of low-quality or had incomplete reports.
The data was carefully combined with the findings of ethical analysis, providing an empirical platform for further investigation on the therapeutic value of phase 1 clinical trials in pediatric cancer patients. They provided evidences for improving the risk/advantages of phase I clinical trials and for identifying studies, which impose greater challenges for complying with the standards of tolerable risk in children.