Systematic Review: Efficacy of Medical Therapy on Outcomes Important to Adult Patients With X-Linked Hypophosphatemia
Authors' conclusion
No formal comparisons between burosumab and conventional therapy in adults exist. Evidence for conventional therapy vs no treatment is very uncertain. Our review highlights the need for more data on the long-term effects of burosumab and conventional therapy on patient-important outcomes in adult patients with XLH.
Study context
- Population
- Adults aged 18+ with clinically or genetically confirmed X-linked hypophosphatemia (XLH)1 RCT, 2 observational studies
- Comparison
- Burosumab vs no treatment or conventional therapy; conventional therapy vs no treatment
- Outcome
- Patient-important outcomes, burosumab vs no treatment
- Follow-up
- 24 weeks
Methodological detailsRandomized controlled trials (RCTs) and observational studies, GRADE certainty assessment, Searched Embase, MEDLINE, Web of Science and Cochrane Central up to May 2023; 4114 records screened, 254 full texts assessed
- RCTs and observational studies
- GRADE certainty assessment
- 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 improves pain from fracture/pseudofracture healing (moderate certainty)
- Little or no impact on direct pain measures (moderate certainty)
- May reduce need for parathyroidectomy (low certainty)
- Little or no impact on fatigue (high certainty), stiffness (moderate certainty) and mobility (low certainty)
- RCT had low risk of bias (RoB)
Further pooled results
Burosumab vs conventional therapy
- Indirect evidence only; low certainty
Conventional therapy vs no treatment
- 2 observational studies with high RoB; very low certainty on patient-important outcomes
Safety
- Burosumab may increase dental abscess risk (low certainty)
Original publication
Systematic Review: Efficacy of Medical Therapy on Outcomes Important to Adult Patients With X-Linked Hypophosphatemia. J Clin Endocrinol Metab. Published 23 December 2024. doi:10.
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