Systematic Review: Efficacy of Medical Therapy on Outcomes Important to Pediatric Patients With X-Linked Hypophosphatemia
Authors' conclusion
Our review indicates that burosumab likely provides benefits to children by preventing lower limb deformity and improving physical health QoL while potentially increasing height. However, burosumab may also increase adverse events. Our review found limited evidence regarding the impact of conventional therapy compared to no treatment on final height. Further research is required to understand the long-term effect of medical therapy in children.
Study context
- Population
- Individuals younger than 18 years with clinically or genetically confirmed X-linked hypophosphatemia (XLH)1 RCT, 1 post hoc, 1 observational
- Comparison
- Burosumab vs conventional therapy or no treatment; conventional therapy vs no treatment
- Outcome
- Burosumab vs conventional therapy
Methodological detailsGRADE certainty assessment, independent dual review, Searched Embase, MEDLINE, Web of Science and Cochrane Central up to May 2023; 4114 records screened, 254 full texts assessed
- GRADE certainty assessment
- Independent dual review
- Searched Embase, MEDLINE, Web of Science and Cochrane Central up to May 2023; 4114 records screened, 254 full texts assessed
Pooled result
As reported in the abstract
- Burosumab probably prevents lower limb deformity and improves physical health quality of life (QoL) (moderate certainty)
- Burosumab may increase height and enhance the burden of symptoms related to chronic hypophosphatemia (low certainty)
- Open-label RCT at high risk of bias (RoB); certainty moderate to very low
Further pooled results
Final height, conventional therapy vs no treatment
- One observational study at high RoB; very low certainty evidence on final height
Safety
Adverse events, burosumab vs conventional therapy
- Burosumab probably increases treatment-emergent adverse events (moderate certainty)
- Burosumab may increase dental abscesses (low certainty)
Original publication
Systematic Review: Efficacy of Medical Therapy on Outcomes Important to Pediatric Patients With X-Linked Hypophosphatemia. J Clin Endocrinol Metab. Published 9 January 2025. doi:10.
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