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hrp0095fc2.4 | Bone, Growth Plate and Mineral Metabolism | ESPE2022

Patient-reported outcomes from a randomized open-label phase 3 trial comparing burosumab vs conventional therapy in children with X-linked hypophosphatemia: results from the 24-week treatment extension period

Padidela Raja , Whyte Michael P , Glorieux Francis H , Munns Craig F , Ward Leanne M , Nilsson Ola , Portale Anthony A , Simmons Jill H , Namba Noriyuki , Cheong Hae Il , Pitukcheewanont Pisit , Sochett Etienne , Högler Wolfgang , Muroya Koji , Tanaka Hiroyuki , Gottesman Gary S , Biggin Andrew , Perwad Farzana , Williams Angela , Nixon Annabel , Sun Wei , Chen Angel , Skrinar Alison , Imel Erik A

In a randomized open-label phase 3 trial in 62 children (1–12 years) with X-linked hypophosphatemia (XLH) (NCT 02915705), switching from conventional therapy (oral phosphate plus active vitamin D) to burosumab, a monoclonal antibody targeting fibroblast growth factor 23, significantly improved serum phosphate concentration, rickets, lower-extremity deformities, growth, mobility, and patient-reported outcomes (PROs) at 64 weeks. Children in Europe, USA, Canada, and Australia wh...

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ESPE Abstracts

Patient-reported outcomes from a randomized open-label phase 3 trial comparing burosumab vs conventional therapy in children with X-linked hypophosphatemia: results from the 24-week treatment extension period

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