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Effect of Burosumab...
Effect of Burosumab Compared With Conventional Therapy on Younger vs Older Children With X-linked Hypophosphatemia
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- Ward, Leanne M. (author)
- Department of Pediatrics, Faculty of Medicine, University of Ottawa, Ottawa, Ontario, Canada
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- Glorieux, Francis H. (author)
- Shriners Hospitals for Children, Canada, McGill University, Montreal, QC, Canada
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- Whyte, Michael P. (author)
- Shriners Hospitals for Children St Louis, St Louis, MO, USA
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- Munns, Craig F. (author)
- Child Health Research Centre, The University of Queensland, Brisbane, QLD, Australia; Department of Endocrinology and Diabetes, Queensland Children's Hospital, Brisbane, QLD, Australia
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- Portale, Anthony A. (author)
- Department of Pediatrics, University of California, San Francisco, San Francisco, CA, USA
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- Högler, Wolfgang (author)
- Department of Pediatrics and Adolescent Medicine, Johannes Kepler University Linz, Linz, Austria; Institute of Metabolism and Systems Research, University of Birmingham, Birmingham, UK
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- Simmons, Jill H. (author)
- Department of Pediatrics, Division of Endocrinology and Diabetes, Vanderbilt University School of Medicine, Vanderbilt University, Nashville, TN, USA
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- Gottesman, Gary S. (author)
- Shriners Hospitals for Children St Louis, St Louis, MO, USA
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- Padidela, Raja (author)
- Department of Paediatric Endocrinology, Royal Manchester Children's Hospital, Manchester, UK
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- Namba, Noriyuki (author)
- Department of Pediatrics, Osaka Hospital, Japan Community Healthcare Organization and Osaka University Graduate School of Medicine, Osaka, Japan; Division of Pediatrics and Perinatology, Tottori University Faculty of Medicine, Yonago, Japan
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- Cheong, Hae Il (author)
- Department of Pediatrics, Seoul National University Children's Hospital, Seoul, South Korea
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- Nilsson, Ola, 1970- (author)
- Karolinska Institutet,Örebro universitet,Institutionen för medicinska vetenskaper,Division of Pediatric Endocrinology and Center for Molecular Medicine, Karolinska Institutet, Stockholm, Sweden; Department of Pediatrics, University Hospital, Örebro, Sweden
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- Mao, Meng (author)
- Ultragenyx Pharmaceutical Inc., Novato, CA, USA
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- Chen, Angel (author)
- Ultragenyx Pharmaceutical Inc., Novato, CA, USA
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- Skrinar, Alison (author)
- Ultragenyx Pharmaceutical Inc., Novato, CA, USA
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- Roberts, Mary Scott (author)
- Ultragenyx Pharmaceutical Inc., Novato, CA, USA
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- Imel, Erik A. (author)
- Department of Medicine and Department of Pediatrics, Indiana University School of Medicine, Indianapolis, IN, USA
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(creator_code:org_t)
- 2022-05-09
- 2022
- English.
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In: Journal of Clinical Endocrinology and Metabolism. - : Oxford University Press. - 0021-972X .- 1945-7197. ; 107:8, s. e3241-e3253
- Related links:
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https://doi.org/10.1...
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Abstract
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- CONTEXT: Younger age at treatment-onset with conventional therapy (Pi/D) is associated with improved growth and skeletal outcomes in children with X-linked hypophosphatemia (XLH). The impact of age on burosumab efficacy and safety in XLH is unknown.OBJECTIVE: Explore the efficacy and safety of burosumab versus Pi/D in younger (<5 years) and older (5 to 12 years) children with XLH.DESIGN: Post-hoc analysis of 64-week, open-label, randomized controlled study.SETTING: Sixteen academic centers.PATIENTS OR OTHER PARTICIPANTS: Sixty-one children 1-12 years of age with XLH (younger, n=26; older, n=35).INTERVENTIONS: Children received burosumab starting at 0.8 mg/kg every 2 weeks (younger, n=14; older, n=15) or continued Pi/D individually titrated per recommended guidelines (younger, n=12; older, n=20).MAIN OUTCOME MEASURE: Least squares means difference (LSMD) in Radiographic Global Impression of Change (RGI-C) rickets total score from baseline to week 64.RESULTS: The LSMD in outcomes through 64 weeks on burosumab versus conventional therapy by age group were as follows: RGI-C rickets total score (younger, +0.90; older, +1.07), total rickets severity score (younger, -0.86; older, -1.44), RGI-C lower limb deformity score (younger, +1.02; older, +0.91), recumbent length or standing height Z-score (younger, +0.20; older, +0.09), and serum alkaline phosphatase (younger, -31.15% of upper normal limit [ULN]; older, -52.11% of ULN). On burosumab, dental abscesses were not reported in younger children but were in 53% of older children.CONCLUSIONS: Burosumab appears to improve outcomes in both younger and older children with XLH, including rickets, lower limb deformities, growth, and alkaline phosphatase, compared with Pi/D.
Subject headings
- MEDICIN OCH HÄLSOVETENSKAP -- Klinisk medicin -- Pediatrik (hsv//swe)
- MEDICAL AND HEALTH SCIENCES -- Clinical Medicine -- Pediatrics (hsv//eng)
Keyword
- X-linked hypophosphatemia
- burosumab
- children
- fibroblast growth factor 23
- rickets
Publication and Content Type
- ref (subject category)
- art (subject category)
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- By the author/editor
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Ward, Leanne M.
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Glorieux, Franci ...
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Whyte, Michael P ...
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Munns, Craig F.
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Portale, Anthony ...
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Högler, Wolfgang
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Simmons, Jill H.
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Gottesman, Gary ...
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Padidela, Raja
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Namba, Noriyuki
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Cheong, Hae Il
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Nilsson, Ola, 19 ...
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Mao, Meng
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Chen, Angel
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Skrinar, Alison
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Roberts, Mary Sc ...
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Imel, Erik A.
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- About the subject
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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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and Pediatrics
- Articles in the publication
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Journal of Clini ...
- By the university
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Örebro University
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Karolinska Institutet