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Burosumab vs conventional therapy in children with X-linked hypophosphataemia: A randomised, active-controlled, open-label, phase 3 trial

The Lancet May 22, 2019

Imel EA, et al. - In this randomized, active-controlled, open-label, phase 3 trial, researchers compared the safety and effectiveness of continuing conventional therapy (oral phosphate and active vitamin D) vs switching to burosumab (a fully human monoclonal antibody against fibroblast growth factor 23) in pediatric X-linked hypophosphatemia. Participants in the study were children with X-linked hypophosphatemia (aged 1-12 years). Participants in the study were randomly assigned (1:1) either to receive subcutaneous burosumab starting at 0.8 mg/kg every 2 weeks (burosumab group) or conventional therapy prescribed by investigators (conventional therapy group). According to findings, rickets severity, growth, and biochemistries among children with X-linked hypophosphatemia treated with burosumab showed significantly greater clinical improvements vs those continuing conventional therapy.

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