Assessing patient-reported outcomes (PROs) and patient-related outcomes in randomized cancer clinical trials for older adults: Results of DATECAN-ELDERLY initiative.

Martine Puts, Siri Rostoft, Angéline Galvin, Pierre Soubeyran, Etienne Brain, Kwok-Leung Cheung, Marije E Hamaker, Ravindran Kanesvaran, Murielle Mauer, Supriya Mohile, Isacco Montroni, Hans Wildiers, Simone Mathoulin-Pélissier, Carine Bellera

Journal: Journal of geriatric oncology 2023;15(1):101611

PMID: 37679204

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Abstract

As older adults with cancer are underrepresented in randomized clinical trials (RCT), there is limited evidence on which to rely for treatment decisions for this population. Commonly used RCT endpoints for the assessment of treatment efficacy are more often tumor-centered (e.g., progression-free survival). These endpoints may not be as relevant for the older patients who present more often with comorbidities, non-cancer-related deaths, and treatment toxicity. Moreover, their expectation and preferences are likely to differ from younger adults. The DATECAN-ELDERLY initiative combines a broad expertise, in geriatric oncology and clinical research, with interest in cancer RCT that include older patients with cancer. In order to guide researchers and clinicians coordinating cancer RCT involving older patients with cancer, the experts reviewed the literature on relevant domains to assess using patient-reported outcomes (PRO) and patient-related outcomes, as well as available tools related to these domains. Domains considered relevant by the panel of experts when assessing treatment efficacy in RCT for older patients with cancer included functional autonomy, cognition, depression and nutrition. These were based on published guidelines from international societies and from regulatory authorities as well as minimum datasets recommended to collect in RCT including older adults with cancer. In addition, health-related quality of life, patients' symptoms, and satisfaction were also considered by the panel. With regards to tools for the assessment of these domains, we highlighted that each tool has its own strengths and limitations, and very few had been validated in older adults with cancer. Further studies are thus needed to validate these tools in this specific population and define the minimum clinically important difference to use when developing RCTs in this population. The selection of the most relevant tool should thus be guided by the RCT research question, together with the specific properties of the tool.

Address: Univ. Bordeaux, Inserm, Bordeaux Population Health Research Center, Epicene team, UMR 1219, Bordeaux, France.; Univ. Bordeaux, Inserm, UMR 1312, SIRIC BRIO, France; Department of medical oncology, Bergonie Institute, Comprehensive Cancer Center, Bordeaux, France.; Department of Medical Oncology, Institut Curie/Saint-Cloud, Saint-Cloud, France.; School of Medicine, University of Nottingham, Nottingham, United Kingdom.; Department of Geriatric Medicine, Diakonessenhuis Utrecht/ Zeist/Doorn, Zeist, the Netherlands.; Division of Medical Oncology, National Cancer Centre Singapore, Singapore.; Statistics Department, European Organization for Research and Treatment of Cancer, Brussels, Belgium.; Department of Medicine, University of Rochester Medical Center, Rochester, NY, United States.; Division of Colorectal Surgery, Ospedale Santa Maria delle Croci, Ravenna, Italy.; Lawrence S. Bloomberg Faculty of Nursing, University of Toronto, Toronto, Canada.; Department of Geriatric Medicine, Oslo University Hospital, Oslo, Norway; Institute of Clinical Medicine, University of Oslo, Oslo, Norway.; Department of General Medical Oncology and Multidisciplinary Breast Centre, University Hospitals Leuven, Leuven Cancer Institute, Belgium.; Univ. Bordeaux, Inserm, Bordeaux Population Health Research Center, Epicene team, UMR 1219, Bordeaux, France; INSERM CIC1401, Clinical and Epidemiological Research Unit, Bergonie Institute, Comprehensive Cancer Center, F-33000 Bordeaux, France.; Univ. Bordeaux, Inserm, Bordeaux Population Health Research Center, Epicene team, UMR 1219, Bordeaux, France; INSERM CIC1401, Clinical and Epidemiological Research Unit, Bergonie Institute, Comprehensive Cancer Center, F-33000 Bordeaux, France. Electronic address: [email protected].

DOI: 10.1016/j.jgo.2023.101611