Amélie Anota1,2, Astrid Pozet3, Hervé Lemasson4, Francois-Emery Cotté4, Antoine Falcoz1, Guillaume Eberst5, Guillaume Mouillet1, Stéphane Guerzider5, Émilie Charton1, Virginie Westeel1,5. 1. Methodology and Quality of Life Unit in Oncology (INSERM UMR 1098), University Hospital of Besançon, 3 Boulevard Alexandre Fleming, 25030, Besançon, France. 2. French National Platform Quality of Life and Cancer, Besançon, France. 3. Methodology and Quality of Life Unit in Oncology (INSERM UMR 1098), University Hospital of Besançon, 3 Boulevard Alexandre Fleming, 25030, Besançon, France. apozet@chu-besancon.fr. 4. Bristol-Myers Squibb, Market Access, Besançon, France. 5. Chest Disease Department, University Hospital of Besançon, Besançon, France.
Abstract
AIM: A systematic literature review of immuno-oncology trials was conducted to assess the potential impact of open-label vs double-blind trial design on patient-reported outcome (PRO) data. METHODS: A systematic search of indexed literature published from January 2009 to May 2019 was conducted using PubMed/MEDLINE, Cochrane Library, and EMBASE database. All randomized clinical trials (RCTs) of immuno-oncology therapies on advanced cancer patients reporting PRO data were identified. Descriptive analyses were performed to quantify differences at baseline and over time, by the type of study, regarding questionnaire completion rate and PRO scores. RESULTS: In total, 23 studies were retained (15 open-label, 8 blinded). At baseline, no difference in completion rate was observed between arms irrespective of trial design (absolute mean difference of 2.8% and 2.2% for open label and blinded studies, respectively). No clinically significant difference in baseline PRO scores was observed between arms. Over time, impact on PRO scores could not be identified due to the limited number of studies, heterogeneity of questionnaires and tumor types. CONCLUSIONS: Trial design had no impact on PRO completion rate or baseline scores. Future research should involve analyses by specific cancer types and ideally compare individual data from two similar RCTs (blinded vs. open-label).
AIM: A systematic literature review of immuno-oncology trials was conducted to assess the potential impact of open-label vs double-blind trial design on patient-reported outcome (PRO) data. METHODS: A systematic search of indexed literature published from January 2009 to May 2019 was conducted using PubMed/MEDLINE, Cochrane Library, and EMBASE database. All randomized clinical trials (RCTs) of immuno-oncology therapies on advanced cancer patients reporting PRO data were identified. Descriptive analyses were performed to quantify differences at baseline and over time, by the type of study, regarding questionnaire completion rate and PRO scores. RESULTS: In total, 23 studies were retained (15 open-label, 8 blinded). At baseline, no difference in completion rate was observed between arms irrespective of trial design (absolute mean difference of 2.8% and 2.2% for open label and blinded studies, respectively). No clinically significant difference in baseline PRO scores was observed between arms. Over time, impact on PRO scores could not be identified due to the limited number of studies, heterogeneity of questionnaires and tumor types. CONCLUSIONS: Trial design had no impact on PRO completion rate or baseline scores. Future research should involve analyses by specific cancer types and ideally compare individual data from two similar RCTs (blinded vs. open-label).
Authors: Guillaume Mouillet; Fabio Efficace; Antoine Thiery-Vuillemin; Emilie Charton; Mieke Van Hemelrijck; Francesco Sparano; Amélie Anota Journal: Cancer Med Date: 2020-08-26 Impact factor: 4.452
Authors: Maria Manuel Teixeira; Fábio Cardoso Borges; Paula Sousa Ferreira; João Rocha; Bruno Sepodes; Carla Torre Journal: Front Med (Lausanne) Date: 2022-08-12