Comparison of Patients With Head and Neck Cancer in Randomized Clinical Trials and Clinical Practice

Abstract

IMPORTANCE When patient populations in randomized clinical trials deviate too much from the general population, it undermines the relevance for daily practice.OBJECTIVE To investigate if patients with head and neck cancer in randomized clinical trials are representative of the clinically treated population.EVIDENCE REVIEW A systematic literature search was performed for randomized clinical trials on head and neck cancer evaluating an intervention to improve outcome with total sample size of 100 patients or greater and published between 2009 and 2019. Outcome measures were age, performance status, and recruitment rate. National cancer registries provided reference data. Databases that were searched included MEDLINE and Epub Ahead of Print; Embase; Cochrane Central Register of Controlled Trials; and ClinicalTrials.gov. Abstracts of search results were retrieved to assess selection criteria by 2 reviewers independently. After the selection procedure was completed by both reviewers, the results were compared and reviewed once more to reach consensus. Full articles were downloaded to retrieve general study information and outcome data.FINDINGS A total of 16 927 publications were identified, resulting in 87 compliant randomized clinical trials with a total of 34 241 patients. Half of the trials included all major head and neck sites, and one-third were exclusively for nasopharynx cancers. The experimental intervention was systemic treatment in 47 (54%) studies, radiotherapy in 23 (26%), and other in 17 (20%). Median sample size was 332, and median duration of accrual was 4.6 years. Median accrual per center per year for head and neck and nasopharynx trials was 5.4 and 39.7 patients, respectively. Median age of patients in head and neck trials was 57 years, which was 7 years younger than in cancer registries. More than 70% of patients had a World Health Organization performance score of 0 to 1 or a Karnofsky performance status of 90 to 100. CONCLUSIONS AND RELEVANCEIn this systematic review, patients in head and neck randomized clinical trials had a very good performance status, and half of them were younger than 57 years, while half of the clinical population was older than 64 years. In more than 50% of the head and neck trials, the yearly accrual per center was less than 6 patients, suggesting overly restrictive recruitment. Critical appraisal of trial population characteristics is recommended before results are implemented in clinical guidelines and general practice.

Transoral Surgery in HPV‐Positive Oropharyngeal Carcinoma: Oncologic Outcomes in the Veterans Affairs System

“…However, patients selected for participation in clinical trials have been reported to display better performance status and younger age than patients treated in clinical practice. 28 Nevertheless, our cohort showed 2-year OS of 96.6%, 88.9%, and 94.2% for patients with low, intermediate, and highrisk diseases, respectively. Furthermore, we show favorable 5-year DSS of 100.0%, 90.0%, and 88.7% in patients with low, intermediate, and high-risk diseases.…”

“…Indeed, 2‐year OS in low‐risk patients was 96–100% irrespective of treatment arm or risk. However, patients selected for participation in clinical trials have been reported to display better performance status and younger age than patients treated in clinical practice 28 . Nevertheless, our cohort showed 2‐year OS of 96.6%, 88.9%, and 94.2% for patients with low, intermediate, and high‐risk diseases, respectively.…”

2011–2021 rising prevalence of HPV infection among oropharyngeal carcinoma in France

“…The data of Ang et al were based on a retrospective analysis of patients with stage III–IV 7 th ed. OPC included in the RTOG0129 randomized trial between 2002 and 2005 treated with high-dose cisplatin, with a subsequent selection that may have excluded older or unfit patients [ 29 , 30 ]. However, time-dependent age evaluation may be an issue for comparison of published cohorts when HPV-positive and HPV-negative patients are not studied over exactly the same period of time.…”