Linda Dirven1, Martin J B Taphoorn2, Jaap C Reijneveld3, Jane Blazeby4, Marc Jacobs5, Andrea Pusic6, Edoardo La Sala7, Roger Stupp8, Peter Fayers9, Fabio Efficace7. 1. VU University Medical Center, Department of Neurology, Amsterdam, The Netherlands. Electronic address: l.dirven@vumc.nl. 2. VU University Medical Center, Department of Neurology, Amsterdam, The Netherlands; Medical Center Haaglanden, Department of Neurology, The Hague, The Netherlands. 3. VU University Medical Center, Department of Neurology, Amsterdam, The Netherlands; Academic Medical Center, Department of Neurology, Amsterdam, The Netherlands. 4. University of Bristol, Centre for Surgical Research and Division of Surgery, Head & Neck, University Hospitals Bristol NHS Foundation Trust, Bristol, United Kingdom. 5. Academic Medical Center, University of Amsterdam, Department of Medical Psychology, Amsterdam, The Netherlands. 6. Memorial Sloan Kettering Cancer Center, Department of Surgery, New York, NY, USA. 7. Italian Group for Adult Hematologic Diseases (GIMEMA), Data Center and Health Outcomes Research Unit, Rome, Italy. 8. University Hospital Zurich, Department of Oncology and Cancer Centre, Zurich, Switzerland. 9. University of Aberdeen, Institute of Applied Health Sciences, Aberdeen, United Kingdom; Norwegian University of Science and Technology, Department of Cancer Research and Molecular Medicine, Faculty of Medicine, Trondheim, Norway.
Abstract
BACKGROUND: To determine the net clinical benefit of a new treatment strategy, information on both survival and patient-reported outcomes (PROs) is required. However, to make an adequately informed decision, PRO evidence should be of sufficiently high quality. OBJECTIVE: To investigate the methodological quality of PRO reporting in randomised controlled trials (RCTs) in patients with brain tumours, and to assess the proportion of studies that should impact clinical decision-making. METHODS: We conducted a systematic literature search in several databases covering January 2004 to March 2012. We selected relevant RCTs and retrieved the following data: (1) basic trial demographics and PRO characteristics, (2) quality of PRO reporting and (3) risk of bias. Studies that should impact clinical decision-making based on their methodological robustness were analysed systematically. RESULTS: We identified 14 RCTs, representing over 3000 glioma patients. Only two RCTs (14%) satisfied sufficiently many key methodological criteria to provide high-quality PRO evidence, and should therefore impact clinical decision-making. Important methodological limitations in other studies were lack of reporting of the extent (43%) and reasons (86%) of missing data and statistical approaches to handle this (71%). PRO results were not interpreted in 79% of the studies and clinical significance was not discussed in 86%. Studies with high-quality PRO evidence generally showed lower risk of bias. CONCLUSIONS: Investigators involved in brain tumour research should pay special attention to methodological challenges identified in current work. The level of PRO reporting should continue to improve in order to facilitate a critical appraisal of study results.
BACKGROUND: To determine the net clinical benefit of a new treatment strategy, information on both survival and patient-reported outcomes (PROs) is required. However, to make an adequately informed decision, PRO evidence should be of sufficiently high quality. OBJECTIVE: To investigate the methodological quality of PRO reporting in randomised controlled trials (RCTs) in patients with brain tumours, and to assess the proportion of studies that should impact clinical decision-making. METHODS: We conducted a systematic literature search in several databases covering January 2004 to March 2012. We selected relevant RCTs and retrieved the following data: (1) basic trial demographics and PRO characteristics, (2) quality of PRO reporting and (3) risk of bias. Studies that should impact clinical decision-making based on their methodological robustness were analysed systematically. RESULTS: We identified 14 RCTs, representing over 3000 glioma patients. Only two RCTs (14%) satisfied sufficiently many key methodological criteria to provide high-quality PRO evidence, and should therefore impact clinical decision-making. Important methodological limitations in other studies were lack of reporting of the extent (43%) and reasons (86%) of missing data and statistical approaches to handle this (71%). PRO results were not interpreted in 79% of the studies and clinical significance was not discussed in 86%. Studies with high-quality PRO evidence generally showed lower risk of bias. CONCLUSIONS: Investigators involved in brain tumour research should pay special attention to methodological challenges identified in current work. The level of PRO reporting should continue to improve in order to facilitate a critical appraisal of study results.
Authors: Rebecca Mercieca-Bebber; Julie Rouette; Melanie Calvert; Madeleine T King; Lori McLeod; Patricia Holch; Michael J Palmer; Michael Brundage Journal: Qual Life Res Date: 2017-02-07 Impact factor: 4.147
Authors: Fabio Efficace; Peter Fayers; Andrea Pusic; Yeliz Cemal; Jane Yanagawa; Marc Jacobs; Andrea la Sala; Valentina Cafaro; Katie Whale; Jonathan Rees; Jane Blazeby Journal: Cancer Date: 2015-06-16 Impact factor: 6.860
Authors: Amir H Zamanipoor Najafabadi; Marthe C M Peeters; Linda Dirven; Daniel J Lobatto; Justus L Groen; Marieke L D Broekman; Saskia M Peerdeman; Wilo C Peul; Martin J B Taphoorn; Wouter R van Furth Journal: Neuro Oncol Date: 2017-07-01 Impact factor: 12.300
Authors: Louis Garnier; Emilie Charton; Antoine Falcoz; Sophie Paget-Bailly; Dewi Vernerey; Marine Jary; François Ducray; Elsa Curtit Journal: Neurooncol Pract Date: 2020-11-11