{"doi":"10.1002/cncr.32947","title":"Outcomes among pediatric patients with cancer who are treated on trial versus off trial: A matched cohort study","abstract":"BACKGROUND: Approximately 50% of children with cancer in the United States who are aged <15 years receive primary treatment on a therapeutic clinical trial. To the authors' knowledge, it remains unknown whether trial enrollment has a clinical benefit compared with the best alternative standard therapy and/or off trial (ie, clinical trial effect). The authors conducted a retrospective matched cohort study to compare the morbidity and mortality of pediatric patients with cancer who are treated on a phase 3 clinical trial compared with those receiving standard therapy and/or off trial. METHODS: Subjects were aged birth to 19 years; were diagnosed between 2000 and 2010 with acute lymphocytic leukemia (ALL), acute myeloid leukemia (AML), rhabdomyosarcoma, or neuroblastoma; and had received initial treatment at the Children's Hospital of Philadelphia. On-trial and off-trial subjects were matched based on age, race, ethnicity, a diagnosis of Down syndrome (for patients with ALL or AML), prognostic risk level, date of diagnosis, and tumor type. RESULTS: A total of 428 participants were matched in 214 pairs (152 pairs for ALL, 24 pairs for AML, 32 pairs for rhabdomyosarcoma, and 6 pairs for neuroblastoma). The 5-year survival rate did not differ between those treated on trial versus those treated with standard therapy and/or off trial (86.9% vs 82.2%; P = .093). On-trial patients had a 32% lower odds of having worse (higher) mortality-morbidity composite scores, although this did not reach statistical significance (odds ratio, 0.68; 95% confidence interval, 0.45-1.03 [P = .070]). CONCLUSIONS: There was no statistically significant difference in outcomes noted between those patients treated on trial and those treated with standard therapy and/or off trial. However, in partial support of the clinical trial effect, the results of the current study indicate a trend toward more favorable outcomes in children treated on trial compared with those treated with standard therapy and/or off trial. These findings can support decision making regarding enrollment in pediatric phase 3 clinical trials.","journal":"Cancer","year":2020,"id":70989,"datarank":0.0,"base_score":0.0,"endowment":0.0,"self_citation_contribution":0.0,"citation_network_contribution":0.0,"self_endowment_contribution":0.0,"citer_contribution":0.0,"corpus_percentile":null,"corpus_rank":null,"citation_count":18,"citer_count":0,"citers_with_citation_signal":0,"citers_with_endowment":0,"datacite_reuse_total":0,"is_dataset":false,"is_dataset_confidence":0.9564,"is_data_producer":false,"deposit_databanks":null,"is_oa":true,"file_count":0,"downloads":0,"has_version_chain":false,"published_date":"2020-01-01","fair_score":null,"fair_percentile":null,"algorithm_id":"datarank_citation_only_1hop_v6","ranking_scope":"data_only","authors":[{"id":375719,"name":"Evelyn Stevens","orcid":"0000-0002-5003-1812","position":1,"is_corresponding":false},{"id":375720,"name":"James Sharpe","orcid":"0000-0002-4357-718X","position":2,"is_corresponding":false},{"id":317739,"name":"Lauren L. Hochman","orcid":"0000-0001-7515-0589","position":3,"is_corresponding":false},{"id":317737,"name":"Joseph G. Reiter","orcid":"0000-0003-3806-4192","position":4,"is_corresponding":false},{"id":375721,"name":"Shawna R. Calhoun","orcid":"0000-0003-2990-2369","position":5,"is_corresponding":false},{"id":375722,"name":"Shivani A. Shah","orcid":"0000-0002-4568-0279","position":6,"is_corresponding":false},{"id":377441,"name":"Leonard C. Bailey","orcid":null,"position":7,"is_corresponding":false},{"id":375723,"name":"Rochelle Bagatell","orcid":"0000-0002-5729-8819","position":8,"is_corresponding":false},{"id":317741,"name":"Jeffrey H. Silber","orcid":"0000-0001-6415-7910","position":9,"is_corresponding":false},{"id":250644,"name":"Eric Tai","orcid":"0000-0001-5184-9037","position":10,"is_corresponding":false},{"id":375724,"name":"Lamia P. Barakat","orcid":"0000-0002-0938-4712","position":11,"is_corresponding":false},{"id":375718,"name":"Marilyn M. Schapira","orcid":"0000-0002-3775-9462","position":0,"is_corresponding":true}],"reference_count":38,"raw_metadata":null,"created_at":"2026-07-18T21:43:57.004133Z","pmid":"32453441","pmcid":null,"fwci":null,"citation_percentile":null,"influential_citations":0,"oa_status":null,"license":null,"views":0,"total_file_size_bytes":0,"version_count":0,"fair_f":null,"fair_a":null,"fair_i":null,"fair_r":null,"fair_zscore":null,"fair_rationale":null,"fair_model":null,"fair_agent_version":null,"fair_fulltext_source":null,"fair_has_llm":null,"fair_computed_at":null,"clinical_trials":[],"software_tools":[],"db_accessions":[],"linked_datasets":[],"topics":[]}