Rickets severity predicts clinical outcomes in children with X-linked hypophosphatemia: Utility of the radiographic Rickets Severity Score.
Tom D. Thacher,John M. Pettifor,Peter J. Tebben,Ana L. Creo,Alison Skrinar,Meng Mao,Chao-Yin Chen,Ting Chang,Javier San Martin,Thomas O. Carpenter +9 more
TLDR
The reliability of the Rickets Severity Score is shown, and it is demonstrated that higher RSS values are associated with greater biochemical, clinical, and functional impairments in children with XLH.About:
This article is published in Bone.The article was published on 2019-02-14 and is currently open access. It has received 43 citations till now.read more
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Burosumab versus conventional therapy in children with X-linked hypophosphataemia: a randomised, active-controlled, open-label, phase 3 trial
Erik A. Imel,Francis H. Glorieux,Michael P. Whyte,Craig F Munns,Leanne Ward,Ola Nilsson,Ola Nilsson,Jill H. Simmons,Raja Padidela,Noriyuki Namba,Hae Il Cheong,Pisit Pitukcheewanont,Etienne Sochett,Wolfgang Högler,Wolfgang Högler,Koji Muroya,Hiroyuki Tanaka,Gary S. Gottesman,Andrew Biggin,Farzana Perwad,Meng Mao,Chao-Yin Chen,Alison Skrinar,Javier San Martin,Anthony A. Portale +24 more
TL;DR: Significantly greater clinical improvements were shown in rickets severity, growth, and biochemistries among children with X-linked hypophosphataemia treated with burosumab compared with those continuing conventional therapy.
Journal ArticleDOI
Multidisciplinary patient care in X‐linked hypophosphatemic rickets: one challenge, many perspectives
Adalbert Raimann,Gabriel T. Mindler,Roland Kocijan,Katrin Bekes,Jochen Zwerina,Gabriele Haeusler,Rudolf Ganger +6 more
TL;DR: This review aims to depict different perspectives in XLH patient care in the setting of a multidisciplinary centre of expertise for rare bone diseases.
Journal ArticleDOI
Consensus Recommendations for the Diagnosis and Management of X-Linked Hypophosphatemia in Belgium
Michaël R. Laurent,Jean De Schepper,Dominique Trouet,Nathalie Godefroid,Emese Boros,Claudine Heinrichs,Bert Bravenboer,Brigitte Velkeniers,Johan Lammens,Pol Harvengt,Etienne Cavalier,Jean-François Kaux,Jacques Lombet,Kathleen De Waele,Charlotte Verroken,Koenraad van Hoeck,Geert Mortier,Elena Levtchenko,Johan Vande Walle +18 more
TL;DR: In this article, a national multidisciplinary group of Belgian experts have discussed translation of international best available evidence into locally feasible consensus recommendations for X-linked hypophosphatemia (XLH) patients.
Journal ArticleDOI
Exploring the Burden of X-Linked Hypophosphataemia: An Opportunistic Qualitative Study of Patient Statements Generated During a Technology Appraisal.
N. Ferizovic,Jade Marshall,Angela E. Williams,M Zulf Mughal,Nicholas Shaw,Catherine Mak,Oliver Gardiner,Pushpa Hossain,Sheela Upadhyaya +8 more
TL;DR: The findings suggest that the greatest burden experienced by children is associated with conventional therapy, co-reported with dosing regimen, adherence, distress and pain, and in adults, conventional therapy co- reported with bone deformity and orthopaedic surgery, as well as pain, mobility, fatigue and dental problems, featured highly.
Journal ArticleDOI
Patient-Reported Outcomes from a Randomized, Active-Controlled, Open-Label, Phase 3 Trial of Burosumab Versus Conventional Therapy in Children with X-Linked Hypophosphatemia
Raja Padidela,Michael P. Whyte,Francis H. Glorieux,Craig F Munns,Leanne M Ward,Ola Nilsson,Ola Nilsson,Anthony A. Portale,Jill H. Simmons,Noriyuki Namba,Hae Il Cheong,Pisit Pitukcheewanont,Etienne Sochett,Wolfgang Högler,Wolfgang Högler,Koji Muroya,Hiroyuki Tanaka,Gary S. Gottesman,Andrew Biggin,Farzana Perwad,Angela Williams,Annabel Nixon,Wei Sun,Angel Chen,Alison Skrinar,Erik A. Imel +25 more
TL;DR: In a randomized, open-label, phase 3 trial involving children aged 1-12 years with X-linked hypophosphatemia, burosumab, a monoclonal antibody targeting fibroblast growth factor 23, significantly improved phosphorus homeostasis, rickets, lower-extremity deformities, mobility, and growth versus continuing oral phosphate and active vitamin D (conventional therapy) as mentioned in this paper.
References
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Burosumab versus conventional therapy in children with X-linked hypophosphataemia: a randomised, active-controlled, open-label, phase 3 trial
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