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Burosumab Versus Ph...
Burosumab Versus Phosphate/Active Vitamin D in Pediatric X-Linked Hypophosphatemia : A Sub-group Analysis by Dose Level
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- Imel, Erik A. (författare)
- Indiana University School of Medicine, Indianapolis, IN, USA
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- Glorieux, Francis H. (författare)
- Shriners Hospital for Children-Canada, Montreal, Canada
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- Whyte, Michael P. (författare)
- Center for Metabolic Bone Disease and Molecular Research, Shriners Hospitals for Children-St. Louis; St. Louis, MO, USA
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- Portale, Anthony A. (författare)
- Department of Pediatrics, University of California, San Francisco, San Francisco, CA, USA
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- Munns, Craig F. (författare)
- The University of Queensland, Queensland Children's Hospital, South Brisbane, Australia
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- Nilsson, Ola, 1970- (författare)
- Karolinska Institutet,Örebro universitet,Institutionen för medicinska vetenskaper,Division of Pediatric Endocrinology and Center for Molecular Medicine, Department of Women's and Children's Health, Karolinska Institutet and University Hospital, Stockholm, Sweden; Department of Pediatrics, University Hospital, Örebro, Sweden
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- Simmons, Jill H. (författare)
- Vanderbilt University Medical Center, Nashville, TN, USA
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- Padidela, Raja (författare)
- Paediatric Endocrinology, Royal Manchester Children's Hospital and Faculty of Biology, Medicine and Health, University of Manchester, Manchester, United Kingdom
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- Namba, Noriyuki (författare)
- Department of Pediatrics, Osaka Hospital, Japan Community Healthcare Organization, Osaka, Japan; Department of Pediatrics, Osaka University Graduate School of Medicine, Suita, Japan, and currently at Division of Pediatrics and Perinatology, Tottori University Faculty of Medicine, Yonago, Japan
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- Cheong, Hae Il (författare)
- Department of Pediatrics, Hallym University Sacred Heart Hospital, Anyang-si, Gyeonggi-do, South Korea
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- Pitukcheewanont, Pisit (författare)
- Children's Hospital Los Angeles, Los Angeles, CA and Keck School of Medicine of USC, Los Angeles, CA, USA
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- Sochett, Etienne (författare)
- Department of Endocrinology, The Hospital for Sick Children, Toronto, Canada
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- Högler, Wolfgang (författare)
- Department of Pediatrics and Adolescent Medicine, Johannes Kepler University Linz, Linz, Austria; Institute of Metabolism and Systems Research, University of Birmingham, Birmingham, United Kingdom
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- Muroya, Koji (författare)
- Kanagawa Children's Medical Center, Yokohama, Japan
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- Tanaka, Hiroyuki (författare)
- Okayama Saiseikai General Hospital, Okayama, Japan
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- Gottesman, Gary S. (författare)
- Center for Metabolic Bone Disease and Molecular Research, Shriners Hospitals for Children-St. Louis; St. Louis, MO, USA; Washington University School of Medicine, St. Louis, MO, USA
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- Biggin, Andrew (författare)
- Children's Hospital Westmead, Westmead, Australia
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- Perwad, Farzana (författare)
- Department of Pediatrics, University of California, San Francisco, San Francisco, CA, USA
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- Chen, Angel (författare)
- Ultragenyx Pharmaceutical Inc., Novato, CA, USA
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- Roberts, Mary Scott (författare)
- Formerly of Ultragenyx Pharmaceutical Inc., Novato, CA, USA
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- Ward, Leanne M. (författare)
- Department of Pediatrics, University of Ottawa, Children's Hospital of Eastern Ontario, Ottawa, Ontario, Canada
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(creator_code:org_t)
- Oxford University Press, 2023
- 2023
- Engelska.
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Ingår i: Journal of Clinical Endocrinology and Metabolism. - : Oxford University Press. - 0021-972X .- 1945-7197. ; 108:11, s. 2990-2998
- Relaterad länk:
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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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http://kipublication...
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Abstract
Ämnesord
Stäng
- PURPOSE: In an open label, randomized, controlled, phase 3 trial in 61 children 1 to 12 years old with X-linked hypophosphatemia (XLH), burosumab improved rickets versus continuing conventional therapy with active vitamin D and phosphate. Here, we conducted an analysis to determine whether skeletal responses differed when switching to burosumab versus continuing higher or lower doses of conventional therapy.METHODS: Conventional therapy dose groups were defined as: higher dose phosphate >40 mg/kg [HPi], lower dose phosphate ≤40 mg/kg [LPi], higher dose alfacalcidol >60 ng/kg or calcitriol >30 ng/kg [HD], and lower dose alfacalcidol ≤60 ng/kg or calcitriol ≤30 ng/kg [LD].RESULTS: At Week 64, the Radiographic Global Impression of Change (RGI-C) for rickets was higher (better) in children randomized to burosumab versus conventional therapy for all pre-baseline dose groups: HPi (+1.72 versus +0.67), LPi (+2.14 versus +1.08), HD (+1.90 versus +0.94), LD (+2.11 versus +1.06). At Week 64, the RGI-C for rickets was also higher in children randomized to burosumab (+2.06) versus conventional therapy for all on-study dose groups: HPi (+1.03), LPi (+1.05), HD (+1.45), LD (+0.72). Serum alkaline phosphatase also decreased in the burosumab treated patients more than in the conventional therapy group, regardless of on-study phosphate and active vitamin D doses.MAIN CONCLUSIONS: Prior phosphate or active vitamin D doses did not influence treatment response after switching to burosumab among children with XLH and active radiographic rickets. Switching from conventional therapy to burosumab improved rickets and serum alkaline phosphatase more than continuing either higher or lower doses of phosphate or active vitamin D.
Ämnesord
- MEDICIN OCH HÄLSOVETENSKAP -- Klinisk medicin -- Pediatrik (hsv//swe)
- MEDICAL AND HEALTH SCIENCES -- Clinical Medicine -- Pediatrics (hsv//eng)
Nyckelord
- FGF23
- X-linked hypophosphatemia
- XLH
- active vitamin D
- burosumab
- oral phosphate
- rickets
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- Av författaren/redakt...
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Imel, Erik A.
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Glorieux, Franci ...
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Whyte, Michael P ...
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Portale, Anthony ...
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Munns, Craig F.
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Nilsson, Ola, 19 ...
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visa fler...
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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 Il
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Pitukcheewanont, ...
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Sochett, Etienne
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Högler, Wolfgang
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Muroya, Koji
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Tanaka, Hiroyuki
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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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Roberts, Mary Sc ...
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Ward, Leanne M.
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- Om ämnet
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- MEDICIN OCH HÄLSOVETENSKAP
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MEDICIN OCH HÄLS ...
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och Klinisk medicin
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och Pediatrik
- Artiklar i publikationen
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Journal of Clini ...
- Av lärosätet
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Örebro universitet
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Karolinska Institutet