Berthelsen, Dorthe, Horgan, Ben, Simon, Lee, Ioannidis, John P. A., Goel, Niti and Choy, Ernest  ORCID: https://orcid.org/0000-0003-4459-8609
2026.
Development of a single‑item patient‑reported measure of treatment side‑effect burden in clinical trials of rheumatic and musculoskeletal diseases using stakeholder input and large language model refinement.
Seminars in Arthritis and Rheumatism
79
, 153026.
10.1016/j.semarthrit.2026.153026
|
Preview |
PDF
- Published Version
Available under License Creative Commons Attribution. Download (2MB) | Preview |
Abstract
Objectives : To build on existing evidence regarding single-item measurement instruments of patient-reported bother or trouble from medical side effects in individuals with rheumatic and musculoskeletal diseases (RMDs). Further, to collect input from the OMERACT community through a structured survey that rated and ranked available options and to seek agreement to advance one or more of these measures for use as exploratory outcomes in future clinical trials. Methods : At OMERACT 2025 we presented and discussed survey results for domain match, feasibility and ranking of six candidate instruments of bother or trouble from side effects. Collaborator feedback - including comments from patients, clinicians, and researchers - was synthesized with a large-language-model (LLM) to identify key concerns and guide refinement of the instrument’s relevance, clarity, and acceptability. The LLM-assisted synthesis of participant comments resulted in a new, single-item instrument designed to improve patient safety reporting from the patient’s perspective. Results : The merged and modified version of the instrument was presented at the OMERACT 2025 meeting, where 33 participants approved it as a reasonable approach to incorporate collaborator input. The proposed instrument is feasible (32 [97%]) and voting supported advancing its further assessment (30 [91%]) as an exploratory outcome measurement instrument in coming RMD trials. Conclusion : We developed a novel single-item instrument. This is the first known application of LLMs in refining a patient-reported outcome instrument for clinical trials. It is designed to capture the patient perspective on symptomatic treatment-related side effects in RMDs and is supported for exploratory use in trials.
| Item Type: | Article |
|---|---|
| Date Type: | Publication |
| Status: | Published |
| Schools: | Schools > Medicine |
| Additional Information: | For full list of authors see: https://doi.org/10.1016/j.semarthrit.2026.153026 License information from Publisher: LICENSE 1: URL: http://creativecommons.org/licenses/by/4.0/, Start Date: 2026-06-19 |
| Publisher: | Elsevier |
| ISSN: | 0049-0172 |
| Date of First Compliant Deposit: | 26 June 2026 |
| Last Modified: | 05 Aug 2026 15:30 |
| URI: | https://orca.cardiff.ac.uk/id/eprint/187752 |
Actions (repository staff only)
 |
Edit Item |
Altmetric
Altmetric