DEVELOPMENT OF THE OIQOL-A- A HEALTH-RELATED QUALITY OF LIFE MEASURE FOR ADULTS WITH OSTEOGENISIS IMPERFECTA
Author(s)
Moshkovich O1, Benjamin K1, Macey J2, Doll HA3, Hill CL4, Hall AK5
1ICON, Gaithersburg, MD, USA, 2ICON, Abingdon, UK, 3ICON, London, UK, 4Sheffield Children's NHS Foundation Trust, Sheffield, UK, 5Mereo BioPharma Group plc, London, UK
OBJECTIVES: To develop a content-valid, condition-specific health-related quality of life (HRQoL) measure for adults with Osteogenesis Imperfecta (OI), a rare genetic disorder primarily characterised by brittle bones that break easily. METHODS: Initially, the Osteogenesis Imperfecta Specific Quality of Life Questionnaire (OIQoL), a condition-specific HRQoL paediatric measure, was adapted for adults with OI (OIQoL-A) based on a literature review, clinical expert interviews, and input from the OIQoL developer. The draft OIQoL-A was assessed for content validity in a hybrid concept elicitation (CE) and cognitive interview (CI) study. The CE portion followed a semi-structured interview guide focusing on OI symptoms and impacts, and was analysed thematically. The CI portion, analysed using a structured codebook, evaluated instrument relevance and ease of completion. The qualitative analysis was conducted using MAXQDA software. Concept saturation was reached after 15 interviews, therefore subsequent interviews were CI-only. RESULTS: Thirty UK and US/CAN OI adults representing a variety of OI types and severities participated: 15 in hybrid CE/CI interviews (60% female; aged 19-67); 15 across two CI waves (47% female; aged 23-70). Hybrid interview findings resulted in a significantly revised OIQoL-A reflecting differences between adult and paediatric OI populations. For example, OI adults rarely avoid or miss activities entirely, but may have challenges in performing daily activities. Also, OI adults fracture less frequently but many experience daily chronic pain. Finally, certain concepts (depression, sexual functioning) were not included in the paediatric measure but are relevant to OI adults. Subsequent rounds of CIs resulted in minor wording/structural changes to improve clarity and remove repetitive items. CONCLUSIONS: OI-specific HRQoL has different challenges in adulthood compared with children with OI that are important to include when measuring the HRQoL for this population. The OIQoL-A is a content valid measure for adults with OI; its psychometric properties will be validated in future studies.
Conference/Value in Health Info
2017-11, ISPOR Europe 2017, Glasgow, Scotland
Value in Health, Vol. 20, No. 9 (October 2017)
Code
PMS67
Topic
Patient-Centered Research
Topic Subcategory
Patient-reported Outcomes & Quality of Life Outcomes
Disease
Rare and Orphan Diseases