Burosumab vs conventional therapy in children with X-linked hypophosphatemia: results of the open-label, phase 3 extension period.

In a randomized, open-label phase 3 study of 61 children aged 1-12 years old with X-linked hypophosphatemia (XLH) previously treated with conventional therapy, changing to burosumab every 2 weeks (Q2W) for 64 weeks improved the phosphate metabolism, radiographic rickets, and growth compared with conventional therapy. In this open-label extension period (weeks 64-88), 21 children continued burosumab Q2W at the previous dose or crossed over from conventional therapy to burosumab starting at 0.8 mg/kg Q2W with continued clinical radiographic assessments through week 88. Efficacy endpoints and safety observations were summarized descriptively for both groups (burosumab continuation,

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© The Author(s) 2024. Published by Oxford University Press on behalf of the American Society for Bone and Mineral Research.

Déclaration de conflit d'intérêts

L.M.W. has been a consultant to Ultragenyx and Kyowa Kirin and participated in clinical trials with Ultragenyx Pharmaceutical Inc., with funds to Dr Ward’s institution. W.H. served as an investigator in clinical trials with, and as a consultant for, Ultragenyx Pharmaceutical Inc. and serves as a clinical investigator in clinical trials with, and has received research funding from, Kyowa Kirin. F.H.G. has been a consultant to, and participated in clinical trials with, Ultragenyx Pharmaceutical Inc. and Kyowa Kirin. A.A.P. has been a consultant to, and served as an investigator in clinical trials with, Ultragenyx Pharmaceutical Inc. M.P.W. has lectured for Ultragenyx Pharmaceutical Inc. C.F.M. is a consultant for Kyowa Kirin and has received research funding from Kyowa Kirin. O.N. has received speakers’ honoraria from Kyowa Kirin, Abbott, and BioMarin, consulting fees from Kyowa Kirin and BioMarin, and research support from Kyowa Kirin. J.H.S. has received institutional research funding from and personal honoraria for participation in an advisory board from Ultragenyx Pharmaceutical Inc. R.P. has no conflicts to disclose. N.N. has been a consultant to, and participated in clinical trials with, Kyowa Kirin. H.I.C. has been a consultant to, and participated in clinical trials with, Ultragenyx Pharmaceutical Inc. H.T. has received research funding from Kyowa Kirin co. Ltd. P.P. has been an employee of Lumos Pharma Inc. and owns stock in Lumos Pharma Inc. and Ascendis Pharma. G.S.G. has been a consultant for Ultragenyx Pharmaceutical Inc. A.C. and J.L.M. are employees of and own stock in Ultragenyx Pharmaceutical Inc. E.A.I. has been a consultant to, and participated in clinical trials with, Ultragenyx Pharmaceutical Inc. E.S., K.M., A.B., and F.P. report no conflicts.