儿童和青少年癌症细胞治疗临床试验：美国 15 年趋势

We searched ClinicalTrials.gov for clinical trials involving cellular therapies enrolling patients under 18 years of age in the U.S. Trials were initially stratified into child-only (maximum age of eligibility <18 years), child/adolescent and young adult (AYA) (maximum age of eligibility ≤21 years), and child/adult (maximum age of eligibility >21 years). Descriptive characteristics and trends over time were analyzed.

We included 202 trials posted 2007-2022. Of the 202 trials, only three trials were child-only; thus, our subsequent analysis focused on comparing child/AYA (≤21 years) and child/adult trials (>21 years). One hundred sixty-nine (84%) enrolled both child and adult populations. The vast majority of trials were early phase (phase 1, 1/2, and 2, 198/202, 98%). Chimeric antigen receptor T cell therapies were most commonly studied (88/202, 44%), while natural-killer cell therapies were most common in child/AYA trials (42% vs. 16%). Most trials were single institution-only (130/202, 64%) and did not receive industry funding (163/202, 81%). Studies with industry funding were more likely to be multicenter (64% vs. 29%) and international (31% vs. 0.6%). Notably, no central nervous system tumor-specific trials had industry funding. There was no difference in therapy type based on funding source. Yearly new trial activations increased over the time period studied (p=0.01).

The frequency of cellular therapy trial activations enrolling child/AYA patients with cancer in the U.S. has increased over time. Most studies were phase 1 or 2, single institution-only, and not industry-supported. Future opportunities for cell therapy for pediatric cancer should include multi-institutional approaches.