Burosumab treatment in adults with X-linked hypophosphataemia: 96-week patient-reported outcomes and ambulatory function from a randomised phase 3 trial and open-label extension

Karine Briot; Anthony A Portale; Maria Luisa Brandi; Thomas O Carpenter; Hae Ii Cheong; Martine Cohen-Solal; Rachel K Crowley; Richard Eastell; Yasuo Imanishi; Steven Ing; Karl Insogna; Nobuaki Ito; Suzanne Jan de Beur; Muhammad K Javaid; Peter Kamenicky; Richard Keen; Takuo Kubota; Robin H Lachmann; Farzana Perwad; Pisit Pitukcheewanont; Stuart H Ralston; Yasuhiro Takeuchi; Hiroyuki Tanaka; Thomas J Weber; Han-Wook Yoo; Annabel Nixon; Mark Nixon; Wei Sun; Angela Williams; Erik A Imel

doi:10.1136/rmdopen-2021-001714

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Burosumab treatment in adults with X-linked hypophosphataemia: 96-week patient-reported outcomes and ambulatory function from a randomised phase 3 trial and open-label extension

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Karine Briot Department of Rheumatology, Hôpital Cochin, Paris, France PubMed articles Google scholar articles
Anthony A Portale Department of Pediatrics, Division of Nephrology, University of California, San Francisco, California, USA PubMed articles Google scholar articles
Maria Luisa Brandi FIRMO Foundation, Florence, Italy PubMed articles Google scholar articles
Thomas O Carpenter Department of Pediatrics and Department of Orthopedics and Rehabilitation, Yale School of Medicine, New Haven, Connecticut, USA PubMed articles Google scholar articles
Hae Ii Cheong Department of Pediatrics, Seoul National University Children’s Hospital, Seoul, Republic of Korea PubMed articles Google scholar articles
Martine Cohen-Solal Department of Rheumatology, Hôpital Lariboisière, Paris, FranceINSERM U1132 BIOSCAR, University of Paris, Paris, France PubMed articles Google scholar articles
Rachel K Crowley Department of Endocrinology, St Vincent’s University Hospital, Dublin, IrelandUniversity College Dublin School of Medicine, Dublin, Ireland PubMed articles Google scholar articles
Richard Eastell Department of Oncology and Metabolism, The University of Sheffield, Sheffield, UK PubMed articles Google scholar articles
Yasuo Imanishi Department of Metabolism, Endocrinology and Molecular Medicine, Osaka City University Graduate School of Medicine, Osaka, Japan PubMed articles Google scholar articles
Steven Ing Division of Endocrinology, Diabetes, and Metabolism, The Ohio State University Wexner Medical Center, Columbus, Ohio, USA PubMed articles Google scholar articles
Karl Insogna Department of Internal Medicine, Yale School of Medicine, New Haven, Connecticut, USA PubMed articles Google scholar articles
Nobuaki Ito Division of Nephrology and Endocrinology, The University of Tokyo Hospital, Tokyo, Japan PubMed articles Google scholar articles
Suzanne Jan de Beur Division of Endocrinology and Metabolism, Department of Medicine, Johns Hopkins University School of Medicine, Baltimore, Maryland, USA PubMed articles Google scholar articles
Muhammad K Javaid Nuffield Department of Orthopaedics, Rheumatology and Musculoskeletal Sciences, National Institute for Health Research Oxford Biomedical Research Centre, University of Oxford, Oxford, UK PubMed articles Google scholar articles
Peter Kamenicky Endocrine Physiology and Pathophysiology, INSERM, Paris-Saclay University, Paris, FranceHospital Bicetre, Paris, France PubMed articles Google scholar articles
Richard Keen Metabolic Bone Disease Unit, Royal National Orthopaedic Hospital NHS Trust, London, UK PubMed articles Google scholar articles
Takuo Kubota Department of Pediatrics, Osaka University Graduate School of Medicine, Osaka, Japan PubMed articles Google scholar articles
Robin H Lachmann Charles Dent Metabolic Unit, National Hospital for Neurology and Neurosurgery, London, UK PubMed articles Google scholar articles
Farzana Perwad Department of Pediatrics, Division of Nephrology, University of California, San Francisco, California, USA PubMed articles Google scholar articles
Pisit Pitukcheewanont Department of Pediatric Endocrinology, Children’s Hospital Los Angeles, University of Southern California Keck School of Medicine, Los Angeles, California, USA PubMed articles Google scholar articles
Stuart H Ralston Rheumatology and Bone Diseases Unit, Medical Research Council Institute of Genetics and Molecular Medicine, The University of Edinburgh, Edinburgh, UK PubMed articles Google scholar articles
Yasuhiro Takeuchi Endocrine Center, Toranomon Hospital, Tokyo, Japan PubMed articles Google scholar articles
Hiroyuki Tanaka Department of Pediatrics, Okayama Saiseikai General Hospital, Okayama, Japan PubMed articles Google scholar articles
Thomas J Weber Division of Endocrinology, Metabolism and Nutrition, Department of Medicine, Duke University Medical Center, Durham, North Carolina, USA PubMed articles Google scholar articles
Han-Wook Yoo Department of Pediatrics, Asan Medical Center Children’s Hospital, Seoul, Republic of Korea PubMed articles Google scholar articles
Annabel Nixon Chilli Consultancy, Salisbury, UK PubMed articles Google scholar articles
Mark Nixon Chilli Consultancy, Salisbury, UK PubMed articles Google scholar articles
Wei Sun Biostatistics Department, Kyowa Kirin Pharmaceutical Development, Inc, Princeton, New Jersey, USA PubMed articles Google scholar articles
Angela Williams Health Economics and Outcomes Research Department, Kyowa Kirin International PLC, Marlow, UK PubMed articles Google scholar articles
Erik A Imel Department of Medicine and Department of Pediatrics, Indiana University School of Medicine, Indianapolis, Indiana, USA PubMed articles Google scholar articles

Correspondence to Dr Karine Briot; karine.briot{at}aphp.fr

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Briot K, Portale AA, Brandi ML, et al

Burosumab treatment in adults with X-linked hypophosphataemia: 96-week patient-reported outcomes and ambulatory function from a randomised phase 3 trial and open-label extension

RMD Open 2021;7:e001714. doi: 10.1136/rmdopen-2021-001714

Publication history

Received April 27, 2021
Accepted August 16, 2021
First published September 21, 2021.

Online issue publication

July 06, 2022

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© Author(s) (or their employer(s)) 2021. Re-use permitted under CC BY-NC. No commercial re-use. See rights and permissions. Published by BMJ. http://creativecommons.org/licenses/by-nc/4.0/This is an open access article distributed in accordance with the Creative Commons Attribution Non Commercial (CC BY-NC 4.0) license, which permits others to distribute, remix, adapt, build upon this work non-commercially, and license their derivative works on different terms, provided the original work is properly cited, appropriate credit is given, any changes made indicated, and the use is non-commercial. See: http://creativecommons.org/licenses/by-nc/4.0/.

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