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Sökning: L773:0021 972X > (2020-2024) > Burosumab Versus Ph...

Burosumab Versus Phosphate/Active Vitamin D in Pediatric X-Linked Hypophosphatemia : A Sub-group Analysis by Dose Level

Imel, Erik A. (författare)
Indiana University School of Medicine, Indianapolis, IN, USA
Glorieux, Francis H. (författare)
Shriners Hospital for Children-Canada, Montreal, Canada
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
Munns, Craig F. (författare)
The University of Queensland, Queensland Children's Hospital, South Brisbane, Australia
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
Simmons, Jill H. (författare)
Vanderbilt University Medical Center, Nashville, TN, USA
Padidela, Raja (författare)
Paediatric Endocrinology, Royal Manchester Children's Hospital and Faculty of Biology, Medicine and Health, University of Manchester, Manchester, United Kingdom
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
Cheong, Hae Il (författare)
Department of Pediatrics, Hallym University Sacred Heart Hospital, Anyang-si, Gyeonggi-do, South Korea
Pitukcheewanont, Pisit (författare)
Children's Hospital Los Angeles, Los Angeles, CA and Keck School of Medicine of USC, Los Angeles, CA, USA
Sochett, Etienne (författare)
Department of Endocrinology, The Hospital for Sick Children, Toronto, Canada
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
Muroya, Koji (författare)
Kanagawa Children's Medical Center, Yokohama, Japan
Tanaka, Hiroyuki (författare)
Okayama Saiseikai General Hospital, Okayama, Japan
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
Biggin, Andrew (författare)
Children's Hospital Westmead, Westmead, Australia
Perwad, Farzana (författare)
Department of Pediatrics, University of California, San Francisco, San Francisco, CA, USA
Chen, Angel (författare)
Ultragenyx Pharmaceutical Inc., Novato, CA, USA
Roberts, Mary Scott (författare)
Formerly of Ultragenyx Pharmaceutical Inc., Novato, CA, USA
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.
Ingår i: Journal of Clinical Endocrinology and Metabolism. - : Oxford University Press. - 0021-972X .- 1945-7197. ; 108:11, s. 2990-2998
Relaterad länk:
https://doi.org/10.1...
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https://urn.kb.se/re...
https://doi.org/10.1...
http://kipublication...
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  • Tidskriftsartikel (refereegranskat)
Abstract Ämnesord
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  • 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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