Feasibility and first results of the trials-within-cohorts design in patients with lung cancer.

Abstract

Purpose or Objective ‘Trials-within-Cohorts’ (TwiCs), previously referred to as ‘cohort multiple RCT’ is a modern trial design that supports a quick and representative recruitment of patients for future trials. To our knowledge, the ‘COhort for Lung cancer Outcome Reporting and trial inclusion’ (COLOR) is the first TwiCs in patients with lung cancer. In this study we aimed to assess the feasibility and first year results of COLOR. Materials and Methods Since July 2020, all patients diagnosed with lung cancer referred to the Radiotherapy department were eligible to participate in the ongoing prospective COLOR study. At time of inclusion, written informed consent was requested for use of patient data, regular in quality of life (QoL) questionnaires and willingness to participate in future trials. After inclusion clinical baseline, treatment, toxicity, follow-up, survival and QoL data were registered. To assess QoL, validated questionnaires regarding global health, functional scales, fatigue, anxiety, depression and work were sent out to participants at predefined intervals (EORTC QLQ-C30, EORTC QLQ-LC13, EQ5D-5L, MFI, HADS and WAI). To assess representativeness of the cohort, the EORTC QLQ-C30 functioning scales of participants were compared with the published EORTC reference values. Results Of the 338 eligible patients between July 2020 and July 2021, 169 (50%) participated. Among 169 participants, 127 (75%) gave informed consent to participate in the QoL questionnaires, of which 101 (80%) completed the questionnaires at baseline, 81 (64%) after 1 month and 65 (51%) after 3 months. Of all patients, 110 (65%) were willing to participate in future clinical trials. At baseline, mean age was 68.4 years (+-9.2). Seventy-four patients (44%) had stage I disease, 14 (9%) stage II, 52 (31%) stage III and 25 (14%) stage IV (Table 1). The EORTC QLQ-C30 physical, role and cognitive functioning at baseline were comparable with the EORTC QLQ-C30 reference values, whereas the means of cognitive and emotional functioning were slightly higher in the COLOR study. Conclusion The TwiCs design appears feasible in patients with lung cancer with fair participation and response rates. With a planned expansion to other centers in the upcoming year, the COLOR study is expected to enable multiple (randomized) evaluations of experimental interventions with important advantages for recruitment, generalizability, and long-term outcome data collection.