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Patient-Reported Outcomes from a Randomized, Active-Controlled, Open-Label, Phase 3 Trial of Burosumab Versus
Raja Padidela1,2, Michael P Whyte3, Francis H Glorieux4
1Department of Paediatric Endocrinology, Royal Manchester Children's Hospital, Manchester, UK. Raja.Padidela@mft.nhs.uk.
Insights
Burosumab significantly improved patient-reported outcomes, including pain and physical function, in children with X-linked hypophosphatemia compared to conventional therapy. This treatment shift demonstrated clinically meaningful benefits for quality of life.
Area of Science:
- Pediatric Endocrinology
- Rare Genetic Diseases
- Pharmacology and Therapeutics
Background:
- X-linked hypophosphatemia (XLH) is a rare genetic disorder characterized by impaired phosphate homeostasis, leading to rickets and skeletal deformities.
- Current management of XLH in children involves oral phosphate and active vitamin D, which may not fully address all disease manifestations.
- Fibroblast growth factor 23 (FGF23) plays a central role in phosphate regulation, and its inhibition is a therapeutic target for XLH.
Purpose of the Study:
- To evaluate the impact of switching from conventional therapy to burosumab on patient-reported outcomes (PROs) in children with X-linked hypophosphatemia.
- To assess changes in pain interference, physical function, fatigue, and overall health using validated questionnaires (PROMIS, SF-10).
Main Methods:
- A randomized, open-label, phase 3 trial compared subcutaneous burosumab to continued conventional therapy in children aged 1-12 years with XLH.
- Patient-reported outcomes were collected from participants aged ≥5 years using the Patient-Reported Outcomes Measurement Information System (PROMIS) and SF-10 Health Survey for Children.
- PROs were analyzed at weeks 40 and 64 to compare treatment effects on pain, mobility, fatigue, and physical health.
Main Results:
- Burosumab treatment led to significant improvements in PROMIS pain interference, physical function mobility, and fatigue scores from baseline.
- Patients receiving burosumab showed clinically meaningful reductions in pain interference and fatigue compared to those on conventional therapy.
- SF-10 physical health scores significantly improved with burosumab at weeks 40 and 64, indicating enhanced overall physical well-being.
Conclusions:
- Switching to burosumab therapy significantly improved patient-reported outcomes, including pain and physical health, in children with X-linked hypophosphatemia.
- Burosumab demonstrated statistically significant advantages over conventional therapy in specific PRO measures, particularly pain interference and physical health.
- These findings highlight the positive impact of burosumab on the quality of life for pediatric patients with XLH.
Abstract:
Changing to burosumab, a monoclonal antibody targeting fibroblast growth factor 23, significantly improved phosphorus homeostasis, rickets, lower-extremity deformities, mobility, and growth versus continuing oral phosphate and active vitamin D (conventional therapy) in a randomized, open-label, phase 3 trial involving children aged 1-12 years with X-linked hypophosphatemia. Patients were randomized (1:1) to subcutaneous burosumab or to continue conventional therapy. We present patient-reported outcomes (PROs) from this trial for children aged ≥ 5 years at screening (n = 35), using a Patient-Reported Outcomes Measurement Information System (PROMIS) questionnaire and SF-10 Health Survey for Children. PROMIS pain interference, physical function mobility, and fatigue scores improved from baseline with burosumab at weeks 40 and 64, but changed little with continued conventional therapy. Pain interference scores differed significantly between groups at week 40 (- 5.02, 95% CI - 9.29 to - 0.75; p = 0.0212) but not at week 64. Between-group differences were not significant at either week for physical function mobility or fatigue. Reductions in PROMIS pain interference and fatigue scores from baseline were clinically meaningful with burosumab at weeks 40 and 64 but not with conventional therapy. SF-10 physical health scores (PHS-10) improved significantly with burosumab at week 40 (least-squares mean [standard error] + 5.98 [1.79]; p = 0.0008) and week 64 (+ 5.93 [1.88]; p = 0.0016) but not with conventional therapy (between-treatment differences were nonsignificant). In conclusion, changing to burosumab improved PRO measures, with statistically significant differences in PROMIS pain interference at week 40 versus continuing with conventional therapy and in PHS-10 at weeks 40 and 64 versus baseline.Trial registration: ClinicalTrials.gov NCT02915705.
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