Measuring the impact of steroid therapy on health-related quality of life in patients with rheumatic diseases: international development of a glucocorticoid treatment–specific patient-reported outcome measure

Abstract

Objectives Glucocorticoids (GCs) (‘steroids’) are used to treat rheumatic diseases but adverse effects are common. We aimed to explore the impact of GC therapy on health-related quality of life (HRQoL), to inform the development of a treatment-specific patient-reported outcome measure (PROM) for use in clinical trials and practice. Methods Semi-structured qualitative interviews were conducted with patients from the UK, USA and Australia, treated for a rheumatic condition with GCs in the last two years. Purposive sampling was used to select participants with a range of demographic and disease features. An initial conceptual framework informed interview prompts and cues. Interviews elicited GC-related physical and psychological symptoms and salient aspects of HRQoL in relation to GC therapy. Interview data were analysed inductively to develop initial individual themes and domains. Candidate questionnaire items were developed and refined. Results Sixty semi-structured qualitative interviews were conducted (UK n = 34, USA n = 10, Australia n = 16). Mean age 58 years; 39/60 female; 18 rheumatic diseases were represented. 126 individual themes were identified and organised into six domains: physical symptoms; psychological symptoms; psychological impact of steroids; impact of steroids on participation; impact of steroids on relationships; and benefits of steroids. Candidate questionnaire items were tested and refined by piloting with patient research partners, iterative rounds of cognitive interviews, and linguistic translatability assessment, informing a draft questionnaire. Conclusion We describe an international qualitative study to develop candidate items for a treatment-specific PROM for patients with rheumatic diseases. A future survey will enable the validation of a final version of the PROM.

Validation of a new glucocorticoid-specific Patient-Reported Outcome Questionnaire (the Steroid PRO)

“…This study has used modern psychometric methods to develop the Steroid PRO, the first generic measure of the impact of GC therapy on HRQoL for patients with a rheumatic disease. The Steroid PRO development has been underpinned by patient involvement at every stage, including in-depth qualitative and cognitive interviews with patients across the major rheumatic diseases, in the UK, Australia and the USA 6. The final Steroid PRO with its four subscales (Social impact, Impact on appearance, Psychological impact and Treatment concerns) has been designed to be highly relevant to patients.…”

“…The Glucocorticoid Toxicity Index is a measure developed for clinicians to use in the assessment of GC-specific outcomes 16. The Steroid PRO focuses on patients’ perceptions of steroid impact and salience which may differ from clinical outcomes or concerns 6. The Systemic Lupus Erythematosus (SLE) Steroid Questionnaire (SSQ)17 is a disease-specific GC impact PROM for SLE 10.…”

“…Lastly, while the Steroid PRO worked well across four cultures (UK, USA, Australia and New Zealand), these are all English-speaking countries, therefore a cross-cultural validation will be required if the tool is to be used in other countries or for multinational comparisons. Linguistic translatability assessment has already been performed as part of the development of the initial candidate items, to ensure the wording and structure of items will be suitable for formal translation in the future 6…”

“…Underpinning semistructured qualitative interviews (n=60) have been performed with rheumatology patients from the UK, USA and Australia, receiving glucocorticoids (GCs) currently or within the last 2 years, for a range of rheumatic conditions. The interviews explored the impact of the medication on their symptoms and health-related quality of life (HRQoL) 6. The themes identified were: physical symptoms, psychological symptoms, psychological impact of steroids, impact of steroids on participation, impact of steroids on relationships and benefits of steroids 6.…”

“…The interviews explored the impact of the medication on their symptoms and health-related quality of life (HRQoL) 6. The themes identified were: physical symptoms, psychological symptoms, psychological impact of steroids, impact of steroids on participation, impact of steroids on relationships and benefits of steroids 6. These themes informed a long-form PRO measure (the draft Steroid PRO) with 40 candidate items, which was piloted with patient research partners (PRPs) and cognitive interviews with patients were conducted in the three countries 6.…”