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Burosumab vs Phosphate/Active Vitamin D in Pediatric X-Linked Hypophosphatemia: A Subgroup Analysis by Dose Level
Erik A Imel1, Francis H Glorieux2, Michael P Whyte3
1Departments of Medicine and Pediatrics, Indiana University School of Medicine, Indianapolis, IN 46202, USA.
Insights
Burosumab significantly improved rickets in children with X-linked hypophosphatemia (XLH) compared to conventional therapy. Switching to burosumab demonstrated superior skeletal improvements and reduced alkaline phosphatase levels.
Area of Science:
- Pediatric Endocrinology
- Skeletal Dysplasias
- Pharmacology
Background:
- X-linked hypophosphatemia (XLH) is a rare genetic disorder characterized by impaired phosphate reabsorption, leading to rickets and skeletal abnormalities.
- Conventional therapy involves active vitamin D and phosphate supplementation, but often results in suboptimal skeletal healing.
Purpose of the Study:
- To compare the efficacy of burosumab versus conventional therapy in improving rickets in children with XLH.
- To analyze skeletal responses based on different dosing strategies of conventional therapy.
Main Methods:
- An open-label, randomized, controlled, phase 3 trial involving 61 children aged 1-12 years with XLH.
- Patients were randomized to receive either burosumab or continue conventional therapy (higher/lower doses of phosphate and active vitamin D).
- Skeletal response was assessed using the Radiographic Global Impression of Change (RGI-C) for rickets at week 64.
Main Results:
- Burosumab treatment resulted in significantly higher RGI-C scores for rickets compared to conventional therapy across all pre-baseline and on-study dose groups.
- Serum alkaline phosphatase (ALP) levels decreased more substantially in the burosumab group.
- Prior conventional therapy doses did not impact the positive response to burosumab.
Conclusions:
- Switching from conventional therapy to burosumab leads to superior improvement in rickets and serum ALP levels in children with XLH.
- Burosumab represents a more effective treatment option for managing rickets in pediatric XLH patients.
Context:
In an open-label, randomized, controlled, phase 3 trial in 61 children aged 1 to 12 years with X-linked hypophosphatemia (XLH), burosumab improved rickets vs continuing conventional therapy with active vitamin D and phosphate.
Objective:
We conducted an analysis to determine whether skeletal responses differed when switching to burosumab vs continuing higher or lower doses of conventional therapy.
Methods:
Conventional therapy dose groups were defined as higher-dose phosphate [greater than 40 mg/kg] (HPi), lower-dose phosphate [40 mg/kg or less] (LPi), higher-dose alfacalcidol [greater than 60 ng/kg] or calcitriol [greater than 30 ng/kg] (HD), and lower-dose alfacalcidol [60 ng/kg or less] or calcitriol [30 ng/kg or less] (LD).
Results:
At week 64, the Radiographic Global Impression of Change (RGI-C) for rickets was higher (better) in children randomly assigned to burosumab vs conventional therapy for all prebaseline dose groups: HPi (+1.72 vs +0.67), LPi (+2.14 vs +1.08), HD (+1.90 vs +0.94), LD (+2.11 vs +1.06). At week 64, the RGI-C for rickets was also higher in children randomly assigned to burosumab (+2.06) vs conventional therapy for all on-study dose groups: HPi (+1.03), LPi (+1.05), HD (+1.45), LD (+0.72). Serum alkaline phosphatase (ALP) also decreased in the burosumab-treated patients more than in the conventional therapy group, regardless of on-study phosphate and active vitamin D doses.
Conclusion:
Prior phosphate or active vitamin D doses did not influence treatment response after switching to burosumab among children with XLH and active radiographic rickets. Switching from conventional therapy to burosumab improved rickets and serum ALP more than continuing either higher or lower doses of phosphate or active vitamin D.
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