Effect of Burosumab on Muscle Function and Strength, and Rates of Adenosine Triphosphate (ATP) Synthesis in Skeletal Muscle in Adults With X-linked Hypophosphatemia (XLH)
Authors' conclusion
The improvement in the 6MWT and STS test without changes in muscle strength or ATP synthesis rates suggests that reductions in pain, fatigue, and stiffness may partly explain the improved performance. Intracellular phosphate in skeletal muscle is insulated from hypophosphatemia in XLH.
Study context
- Population
- Untreated, symptomatic adults with XLH
- Participants
- 10 participants
- Comparison
- Burosumab treatment vs baseline
- Burosumabn = 10
- Outcome
- ATP synthesis rates in skeletal muscle (right calf, 31P magnetic resonance spectroscopy saturation transfer)
- Follow-up
- 2 weeks ("peak") and 4 weeks ("trough") after the third dose
Main finding
As reported in the abstract
ATP synthesis rates · 3 months
- ATP synthesis rates did not change over the 3 months of the study
Further findings
Symptoms (pain, fatigue, stiffness, lower-extremity joint pain)
- All symptoms improved with treatment
6MWT and Sit to Stand (STS) test performance
- Performance on the 6MWT and STS tests improved
Muscle strength · 3 months
- Muscle strength did not change
Rate of change in ATP synthesis by 6MWT/STS performance (at/above vs below median)
- No difference in rate of change in ATP synthesis
Intracellular muscle phosphate
- Intracellular muscle concentrations of phosphate were normal
Original publication
Effect of Burosumab on Muscle Function and Strength, and Rates of ATP Synthesis in Skeletal Muscle in Adults With XLH. J Clin Endocrinol Metab. Published 1 November 2023. doi:10.
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