학술논문
Development of a core outcome set for use in adult primary glioma phase III interventional trials: A mixed methods study.
Document Type
Academic Journal
Author
Retzer A; Centre for Patient Reported Outcomes Research (CPROR), Institute of Applied Health Research, University of Birmingham, Birmingham, UK.; National Institute for Health Research (NIHR) Applied Research Collaboration West Midlands (ARC WM), Birmingham, UK.; NIHR Birmingham Biomedical Research Centre (BRC), University of Birmingham, Birmingham, UK.; Baddeley E; Marie Curie Palliative Care Research Centre, Division of Population Medicine, Cardiff University School of Medicine, Cardiff, UK.; Sivell S; Marie Curie Palliative Care Research Centre, Division of Population Medicine, Cardiff University School of Medicine, Cardiff, UK.; Scott H; Division of Research and Evaluation, Office for Standards in Education, Childrens' Services and Skills (OFSTED), Bristol, UK.; Nelson A; Marie Curie Palliative Care Research Centre, Division of Population Medicine, Cardiff University School of Medicine, Cardiff, UK.; Bulbeck H; Brainstrust - The Brain Cancer People, UK.; Seddon K; Cardiff University, Cardiff, UK.; Grant R; Department of Clinical Neurosciences, Royal Infirmary of Edinburgh, Edinburgh, UK.; Adams R; Centre for Trials Research, Cardiff University, Cardiff, UK.; Watts C; Institute of Cancer and Genomic Sciences, University of Birmingham, UK.; Aiyegbusi OL; Centre for Patient Reported Outcomes Research (CPROR), Institute of Applied Health Research, University of Birmingham, Birmingham, UK.; National Institute for Health Research (NIHR) Applied Research Collaboration West Midlands (ARC WM), Birmingham, UK.; Birmingham Health Partners Centre for Regulatory Science and Innovation, University of Birmingham, Birmingham, UK.; NIHR Birmingham Biomedical Research Centre (BRC), University of Birmingham, Birmingham, UK.; NIHR Birmingham-Oxford Blood and Transplant Research Unit (BTRU) in Precision Transplant and Cellular Therapeutics, University of Birmingham, Birmingham, UK.; Kearns P; Institute of Cancer and Genomic Sciences, University of Birmingham , UK.; NIHR Birmingham Biomedical Research Centre (BRC), University of Birmingham, Birmingham, UK.; Cancer Research UK Clinical Trials Unit, Institute of Cancer and Genomic Sciences, University of Birmingham , UK.; Rivera SC; Centre for Patient Reported Outcomes Research (CPROR), Institute of Applied Health Research, University of Birmingham, Birmingham, UK.; Birmingham Health Partners Centre for Regulatory Science and Innovation, University of Birmingham, Birmingham, UK.; Dirven L; Department of Neurology, Leiden University Medical Center, Leiden, The Netherlands.; Department of Neurology, Haaglanden Medical Center, The Hague, The Netherlands.; Calvert M; Centre for Patient Reported Outcomes Research (CPROR), Institute of Applied Health Research, University of Birmingham, Birmingham, UK.; National Institute for Health Research (NIHR) Applied Research Collaboration West Midlands (ARC WM), Birmingham, UK.; Birmingham Health Partners Centre for Regulatory Science and Innovation, University of Birmingham, Birmingham, UK.; NIHR Birmingham Biomedical Research Centre (BRC), University of Birmingham, Birmingham, UK.; Midlands Health Data Research UK, Birmingham, UK.; NIHR Birmingham-Oxford Blood and Transplant Research Unit (BTRU) in Precision Transplant and Cellular Therapeutics, University of Birmingham, Birmingham, UK.; Byrne A; Marie Curie Palliative Care Research Centre, Division of Population Medicine, Cardiff University School of Medicine, Cardiff, UK.
Source
Publisher: Oxford University Press Country of Publication: England NLM ID: 101755003 Publication Model: eCollection Cited Medium: Internet ISSN: 2632-2498 (Electronic) Linking ISSN: 26322498 NLM ISO Abbreviation: Neurooncol Adv Subsets: PubMed not MEDLINE
Subject
Language
English
Abstract
Background: Glioma interventional studies should collect data aligned with patient priorities, enabling treatment benefit assessment and informed decision-making. This requires effective data synthesis and meta-analyses, underpinned by consistent trial outcome measurement, analysis, and reporting. Development of a core outcome set (COS) may contribute to a solution.
Methods: A 5-stage process was used to develop a COS for glioma trials from the UK perspective. Outcome lists were generated in stages 1: a trial registry review and systematic review of qualitative studies and 2: interviews with glioma patients and caregivers. In stage 3, the outcome lists were de-duplicated with accessible terminology, in stage 4 outcomes were rated via a 2-round Delphi process, and stage 5 comprised a consensus meeting to finalize the COS. Patient-reportable COS outcomes were identified.
Results: In Delphi round 1, 96 participants rated 35 outcomes identified in stages 1 and 2, to which a further 10 were added. Participants (77/96) rated the resulting 45 outcomes in round 2. Of these, 22 outcomes met a priori threshold for inclusion in the COS. After further review, a COS consisting of 19 outcomes grouped into 7 outcome domains (survival, adverse events, activities of daily living, health-related quality of life, seizure activity, cognitive function, and physical function) was finalized by 13 participants at the consensus meeting.
Conclusions: A COS for glioma trials was developed, comprising 7 outcome domains. Additional research will identify appropriate measurement tools and further validate this COS.
Competing Interests: RG: funding: NIHR, UCB Pharma. OLA: funding: NIHR Birmingham BRC, NIHR ARC WM, UKRI, Health Foundation, Janssen, Gilead, GlaxoSmithKline. Personal fees: Gilead Sciences, Merck, GlaxoSmithKline. LD: funding: Innovative Medicines Initiative, European Organisation for Research and Treatment of Cancer (EORTC) Quality of Life Group, Brain Tumour Charity. Neuro Oncology Journal Associate Editor. MC: Birmingham Health Partners Centre for Regulatory Science and Innovation and CPROR Director, NIHR senior investigator. Funding: NIHR, UKRI, NIHR Birmingham BRC, NIHR ARC WM, UK SPINE, European Regional Development Fund-Demand Hub and Health Data Research UK at the University of Birmingham and University Hospitals Birmingham NHS Foundation Trust, Innovate UK (part of UKRI), Macmillan Cancer Support, UCB Pharma, Janssen, GlaxoSmithKline, Gilead. Personal fees: Astellas, Aparito, CIS Oncology, Halfloop, Takeda, Merck, Daiichi Sankyo, Glaukos, GlaxoSmithKline, Patient-Centered Outcomes Research Institute. A relative owns GlaxoSmithKline shares.
(© The Author(s) 2023. Published by Oxford University Press, the Society for Neuro-Oncology and the European Association of Neuro-Oncology.)
Methods: A 5-stage process was used to develop a COS for glioma trials from the UK perspective. Outcome lists were generated in stages 1: a trial registry review and systematic review of qualitative studies and 2: interviews with glioma patients and caregivers. In stage 3, the outcome lists were de-duplicated with accessible terminology, in stage 4 outcomes were rated via a 2-round Delphi process, and stage 5 comprised a consensus meeting to finalize the COS. Patient-reportable COS outcomes were identified.
Results: In Delphi round 1, 96 participants rated 35 outcomes identified in stages 1 and 2, to which a further 10 were added. Participants (77/96) rated the resulting 45 outcomes in round 2. Of these, 22 outcomes met a priori threshold for inclusion in the COS. After further review, a COS consisting of 19 outcomes grouped into 7 outcome domains (survival, adverse events, activities of daily living, health-related quality of life, seizure activity, cognitive function, and physical function) was finalized by 13 participants at the consensus meeting.
Conclusions: A COS for glioma trials was developed, comprising 7 outcome domains. Additional research will identify appropriate measurement tools and further validate this COS.
Competing Interests: RG: funding: NIHR, UCB Pharma. OLA: funding: NIHR Birmingham BRC, NIHR ARC WM, UKRI, Health Foundation, Janssen, Gilead, GlaxoSmithKline. Personal fees: Gilead Sciences, Merck, GlaxoSmithKline. LD: funding: Innovative Medicines Initiative, European Organisation for Research and Treatment of Cancer (EORTC) Quality of Life Group, Brain Tumour Charity. Neuro Oncology Journal Associate Editor. MC: Birmingham Health Partners Centre for Regulatory Science and Innovation and CPROR Director, NIHR senior investigator. Funding: NIHR, UKRI, NIHR Birmingham BRC, NIHR ARC WM, UK SPINE, European Regional Development Fund-Demand Hub and Health Data Research UK at the University of Birmingham and University Hospitals Birmingham NHS Foundation Trust, Innovate UK (part of UKRI), Macmillan Cancer Support, UCB Pharma, Janssen, GlaxoSmithKline, Gilead. Personal fees: Astellas, Aparito, CIS Oncology, Halfloop, Takeda, Merck, Daiichi Sankyo, Glaukos, GlaxoSmithKline, Patient-Centered Outcomes Research Institute. A relative owns GlaxoSmithKline shares.
(© The Author(s) 2023. Published by Oxford University Press, the Society for Neuro-Oncology and the European Association of Neuro-Oncology.)