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Burosumab vs conven...
Burosumab vs conventional therapy in children with X-linked hypophosphatemia : results of the open-label, phase 3 extension period
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- Ward, Leanne M. (author)
- Department of Pediatrics, Faculty of Medicine, University of Ottawa, Ottawa Ontario, Canada
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- Högler, Wolfgang (author)
- Department of Paediatrics and Adolescent Medicine, Johannes Kepler University Linz, Linz, Austria; Institute of Metabolism and Systems Research, University of Birmingham, Edgbaston, Birmingham, United Kingdom
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- Glorieux, Francis H. (author)
- Department of Surgery, Pediatrics, and Human Genetics, Shriners Hospitals for Children and McGill University, Montreal Quebec, Canada
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- Portale, Anthony A. (author)
- Department of Pediatrics, University of California, San Francisco CA, United States
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- Whyte, Michael P. (author)
- Shriners Hospitals for Children St Louis, St Louis MO, United States
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- Munns, Craig F. (author)
- Faculty of Medicine, The University of Queensland, Brisbane Queensland, Australia; Faculty of Medicine, Child Health Research Centre, The University of Queensland, Brisbane Queensland, Australia; Department of Endocrinology and Diabetes, Queensland Children’s Hospital, Brisbane Queensland, Australia
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- Nilsson, Ola, 1970- (author)
- Karolinska Institutet,Örebro universitet,Institutionen för medicinska vetenskaper,Division of Pediatric Endocrinology, Department of Women’s and Children’s Health, Center for Molecular Medicine, Karolinska Institutet and University Hospital, Stockholm, Sweden; Department of Pediatrics, School of Medical Sciences, Örebro University and University Hospital, Örebro, Sweden
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- Simmons, Jill H. (author)
- Division of Endocrinology and Diabetes, Department of Pediatrics, Vanderbilt University School of Medicine, Vanderbilt University, Nashville TN, United States
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- Padidela, Raja (author)
- Department of Paediatric Endocrinology, Royal Manchester Children’s Hospital, Manchester, United Kingdom
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- Namba, Noriyuki (author)
- Department of Pediatrics, Osaka Hospital, Japan Community Healthcare Organization, Osaka, Japan; Osaka University Graduate School of Medicine, Osaka, Japan
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- Cheong, Hae, II (author)
- Department of Pediatrics, Hallym University Sacred Heart Hospital, Dongan-gu, Anyang, South Korea
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- Sochett, Etienne (author)
- Department of Pediatrics, Hospital for Sick Children, Toronto Ontario, Canada
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- Muroya, Koji (author)
- Department of Endocrinology, Kanagawa Children’s Medical Center, Yokohama, Kanagawa Japan
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- Tanaka, Hiroyuki (author)
- Division of Pediatrics, Okayama Saiseikai General Hospital Outpatient Center, Kita-ku, Okayama, Japan
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- Pitukcheewanont, Pisit (author)
- Center of Endocrinology, Diabetes and Metabolism, Children’s Hospital of Los Angeles, Los Angeles CA, United States
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- Gottesman, Gary S. (author)
- Shriners Hospitals for Children St Louis, St Louis MO, United States
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- Biggin, Andrew (author)
- Department of Endocrinology, The University of Sydney Children’s Hospital Westmead Clinical School, The Children’s Hospital at Westmead, Westmead New South Wales, Australia
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- Perwad, Farzana (author)
- Department of Pediatrics, University of California, San Francisco CA, United States
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- Chen, Angel (author)
- Ultragenyx Pharmaceutical Inc., Novato CA, United States
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- Merritt, John Lawrence (author)
- Ultragenyx Pharmaceutical Inc., Novato CA, United States
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- Imel, Erik A. (author)
- Department of Medicine, Indiana University School of Medicine, Indianapolis IN, United States
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(creator_code:org_t)
- Oxford University Press, 2024
- 2024
- English.
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In: JBMR Plus. - : Oxford University Press. - 2473-4039. ; 8:1
- Related links:
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https://doi.org/10.1...
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https://urn.kb.se/re...
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https://doi.org/10.1...
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Abstract
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- 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, n = 6; crossover, n = 15). At week 88 compared with baseline, improvements in the following outcomes were observed in the burosumab continuation and crossover groups, respectively: mean (SD) RGI-C rickets total score (primary outcome), +2.11 (0.27) and +1.89 (0.35); mean (SD) RGI-C lower limb deformity score, +1.61 (0.91) and +0.73 (0.82); and mean (SD) height Z-score + 0.41 (0.50) and +0.08 (0.34). Phosphate metabolism normalized rapidly in the crossover group and persisted in the continuation group. Mean (SD) serum alkaline phosphatase decreased from 169% (43%) of the upper limit of normal (ULN) at baseline to 126% (51%) at week 88 in the continuation group and from 157% (33%) of the ULN at baseline to 111% (23%) at week 88 in the crossover group. During the extension period, treatment-emergent adverse events (AEs) were reported in all 6 children in the burosumab continuation group and 14/15 children in the crossover group. The AE profiles in the randomized and extension periods were similar, with no new safety signals identified. Improvements from baseline in radiographic rickets continued in the extension period among children with XLH who remained on burosumab. Children who crossed over from conventional therapy to burosumab demonstrated a rapid improvement in phosphate metabolism and improved rickets healing over the ensuing 22 weeks.
Subject headings
- MEDICIN OCH HÄLSOVETENSKAP -- Klinisk medicin -- Endokrinologi och diabetes (hsv//swe)
- MEDICAL AND HEALTH SCIENCES -- Clinical Medicine -- Endocrinology and Diabetes (hsv//eng)
Keyword
- burosumab
- FGF23
- phosphate
- rare disease
- x-linked hypophosphatemia
Publication and Content Type
- ref (subject category)
- art (subject category)
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Ward, Leanne M.
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Högler, Wolfgang
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Glorieux, Franci ...
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Portale, Anthony ...
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Whyte, Michael P ...
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Munns, Craig F.
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Nilsson, Ola, 19 ...
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Simmons, Jill H.
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Padidela, Raja
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Namba, Noriyuki
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Cheong, Hae, II
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Sochett, Etienne
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Muroya, Koji
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Tanaka, Hiroyuki
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Pitukcheewanont, ...
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Gottesman, Gary ...
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Biggin, Andrew
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Perwad, Farzana
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Chen, Angel
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Merritt, John La ...
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Imel, Erik A.
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- MEDICAL AND HEALTH SCIENCES
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MEDICAL AND HEAL ...
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and Clinical Medicin ...
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Örebro University
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Karolinska Institutet