Development of a core outcome set for use in adult primary glioma phase III interventional trials: A mixed methods study

Ameeta Retzer; Elin Baddeley; Stephanie Sivell; Hannah Scott; Annmarie Nelson; Helen Bulbeck; Kathy Seddon; Robin Grant; Richard Adams; Colin Watts; Olalekan Lee Aiyegbusi; Pamela Kearns; Samantha Cruz Rivera; Linda Dirven; Melanie Calvert; Anthony Byrne

doi:10.1093/noajnl/vdad096

Development of a core outcome set for use in adult primary glioma phase III interventional trials: A mixed methods study

Neurooncol Adv. 2023 Aug 2;5(1):vdad096. doi: 10.1093/noajnl/vdad096. eCollection 2023 Jan-Dec.

Authors

Ameeta Retzer^{1

2

3}, Elin Baddeley⁴, Stephanie Sivell⁴, Hannah Scott⁵, Annmarie Nelson⁴, Helen Bulbeck⁶, Kathy Seddon⁷, Robin Grant⁸, Richard Adams⁹, Colin Watts¹⁰, Olalekan Lee Aiyegbusi^{1

2

11

3

12}, Pamela Kearns^{13

3

14}, Samantha Cruz Rivera^{1

11}, Linda Dirven^{15

16}, Melanie Calvert^{1

2

11

3

17

12}, Anthony Byrne⁴

Affiliations

¹ Centre for Patient Reported Outcomes Research (CPROR), Institute of Applied Health Research, University of Birmingham, Birmingham, UK.
² National Institute for Health Research (NIHR) Applied Research Collaboration West Midlands (ARC WM), Birmingham, UK.
³ NIHR Birmingham Biomedical Research Centre (BRC), University of Birmingham, Birmingham, UK.
⁴ Marie Curie Palliative Care Research Centre, Division of Population Medicine, Cardiff University School of Medicine, Cardiff, UK.
⁵ Division of Research and Evaluation, Office for Standards in Education, Childrens' Services and Skills (OFSTED), Bristol, UK.
⁶ Brainstrust - The Brain Cancer People, UK.
⁷ Cardiff University, Cardiff, UK.
⁸ Department of Clinical Neurosciences, Royal Infirmary of Edinburgh, Edinburgh, UK.
⁹ Centre for Trials Research, Cardiff University, Cardiff, UK.
¹⁰ Institute of Cancer and Genomic Sciences, University of Birmingham, UK.
¹¹ Birmingham Health Partners Centre for Regulatory Science and Innovation, University of Birmingham, Birmingham, UK.
¹² NIHR Birmingham-Oxford Blood and Transplant Research Unit (BTRU) in Precision Transplant and Cellular Therapeutics, University of Birmingham, Birmingham, UK.
¹³ Institute of Cancer and Genomic Sciences, University of Birmingham , UK.
¹⁴ Cancer Research UK Clinical Trials Unit, Institute of Cancer and Genomic Sciences, University of Birmingham , UK.
¹⁵ Department of Neurology, Leiden University Medical Center, Leiden, The Netherlands.
¹⁶ Department of Neurology, Haaglanden Medical Center, The Hague, The Netherlands.
¹⁷ Midlands Health Data Research UK, Birmingham, UK.

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.

Keywords: Delphi; neuro-oncology; outcomes; primary glioma; trials.