E Charton1,2, B Cuer3,4, F Cottone5, F Efficace5, C Touraine3, Z Hamidou6,7, F Fiteni3,8, F Bonnetain9,10,7, M-C Woronoff-Lemsi10,11, C Bascoul-Mollevi3,4,7, A Anota9,10,7. 1. Methodology and Quality of Life Unit in Oncology (INSERM UMR 1098), University Hospital of Besançon, 3 Boulevard Fleming, 25030, Besançon, France. echarton@chu-besancon.fr. 2. University Bourgogne Franche-Comté, INSERM, EFS BFC, UMR1098, Interactions Hôte-Greffon-Tumeur/Ingénierie Cellulaire et Génique, Besançon, France. echarton@chu-besancon.fr. 3. Montpellier Cancer Institute (ICM), University of Montpellier, Montpellier, France. 4. Institute of Cancer Research of Montpellier (IRCM), ICM, INSERM, Montpellier, France. 5. Data Center and Health Outcomes Research Unit, Italian Group for Adult Hematologic Diseases (GIMEMA), Rome, Italy. 6. EA3279 Self-perceived Health Assessment Research Unit, Aix-Marseille University, Marseille, France. 7. French National Platform Quality of Life and Cancer, Besançon, France. 8. Department of Medical Oncology, University Hospital of Nîmes, Nîmes, France. 9. Methodology and Quality of Life Unit in Oncology (INSERM UMR 1098), University Hospital of Besançon, 3 Boulevard Fleming, 25030, Besançon, France. 10. University Bourgogne Franche-Comté, INSERM, EFS BFC, UMR1098, Interactions Hôte-Greffon-Tumeur/Ingénierie Cellulaire et Génique, Besançon, France. 11. Department of Clinical Research and Innovation, University Hospital of Besançon, Besançon, France.
Abstract
PURPOSE: The time to deterioration (TTD) approach has been proposed as a modality of longitudinal analysis of patient-reported outcomes (PROs) in cancer randomized clinical trials (RCTs). The objective of this study was to perform a systematic review of how the TTD approach has been used in phase III RCTs to analyze longitudinal PRO data. METHODS: A systematic literature search was conducted in PubMed/MEDLINE, the Cochrane Library and through manual search to identify studies published between January 2014 and June 2018. All phase III cancer RCTs including a PRO endpoint using the TTD approach were considered. We collected general information about the study, PRO assessment and the TTD approach, such as the event definition, the choice of reference score and whether the deterioration was definitive or not. RESULTS: A total of 1549 articles were screened, and 39 studies were finally identified as relevant according to predefined criteria. Among these 39 studies, 36 (92.3%) were in advanced and/or metastatic cancer. Several different deterioration definitions were used in RCTs, 10 studies (25.6%) defined the deterioration as "definitive", corresponding to a deterioration maintained over time until the last PRO assessment available for each patient. The baseline score was explicitly stated as the reference score to qualify the deterioration for most studies (n = 31, 79.5%). CONCLUSION: This review highlights the lack of standardization of the TTD approach for the analysis of PRO data in RCTs. Special attention should be paid to the definition of "deterioration", and this should be based on the specific cancer setting.
PURPOSE: The time to deterioration (TTD) approach has been proposed as a modality of longitudinal analysis of patient-reported outcomes (PROs) in cancer randomized clinical trials (RCTs). The objective of this study was to perform a systematic review of how the TTD approach has been used in phase III RCTs to analyze longitudinal PRO data. METHODS: A systematic literature search was conducted in PubMed/MEDLINE, the Cochrane Library and through manual search to identify studies published between January 2014 and June 2018. All phase III cancer RCTs including a PRO endpoint using the TTD approach were considered. We collected general information about the study, PRO assessment and the TTD approach, such as the event definition, the choice of reference score and whether the deterioration was definitive or not. RESULTS: A total of 1549 articles were screened, and 39 studies were finally identified as relevant according to predefined criteria. Among these 39 studies, 36 (92.3%) were in advanced and/or metastatic cancer. Several different deterioration definitions were used in RCTs, 10 studies (25.6%) defined the deterioration as "definitive", corresponding to a deterioration maintained over time until the last PRO assessment available for each patient. The baseline score was explicitly stated as the reference score to qualify the deterioration for most studies (n = 31, 79.5%). CONCLUSION: This review highlights the lack of standardization of the TTD approach for the analysis of PRO data in RCTs. Special attention should be paid to the definition of "deterioration", and this should be based on the specific cancer setting.
Entities:
Keywords:
Patient-reported outcomes; Randomized clinical trials; Systematic review; Time to deterioration
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