Burosumab Improves Patient-Reported Outcomes in Adults With Tumor-Induced Osteomalacia: Mixed-Methods Analysis
Authors' conclusion
Thus, this exploratory mixed-methods analysis provides deeper understanding of patients' perception of clinical meaningfulness beyond that articulated in validated patient-reported outcome instruments.
Study context
- Population
- Adults with tumor-induced osteomalacia (TIO), phase 2 trial UX023T-CL2018 interviewed
- Participants
- 14 adults
- Comparison
- Change from baseline after 144 weeks of burosumab
- Burosumabn = 14
Methodological detailsexit interviews, single-arm
- Exit interviews
- Single-arm
Primary result
As reported in the abstract
Patient-reported outcomes:
- Pain
- Fatigue
- HRQL
- Improvements in pain and fatigue after treatment, some achieving statistically and/or clinically meaningful thresholds
- SF-36 v2 improvements most pronounced in Physical Component Score, Physical Function and Bodily Pain domains
- Exploratory analysis
Further findings
Exit interviews (n = 8)
- Compromised health-related quality of life (HRQL) and pain rated most important to treat; more bothersome than fatigue and compromised physical function and activities of daily living
- Described symptomatic improvement and its clinical meaningfulness, incl. physical function, activities of daily living and mental well-being
Original publication
Burosumab Improves Patient-Reported Outcomes in Adults With Tumor-Induced Osteomalacia: Mixed-Methods Analysis. J Bone Miner Res. Published 4 September 2023. doi:10. 1002/ jbmr. 4900
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