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Sökning: WFRF:(Merritt F. S.) > (2020-2024) > Burosumab vs conven...

Burosumab vs conventional therapy in children with X-linked hypophosphatemia : results of the open-label, phase 3 extension period

Ward, Leanne M. (författare)
Department of Pediatrics, Faculty of Medicine, University of Ottawa, Ottawa Ontario, Canada
Högler, Wolfgang (författare)
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
Glorieux, Francis H. (författare)
Department of Surgery, Pediatrics, and Human Genetics, Shriners Hospitals for Children and McGill University, Montreal Quebec, Canada
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Portale, Anthony A. (författare)
Department of Pediatrics, University of California, San Francisco CA, United States
Whyte, Michael P. (författare)
Shriners Hospitals for Children St Louis, St Louis MO, United States
Munns, Craig F. (författare)
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
Nilsson, Ola, 1970- (författare)
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
Simmons, Jill H. (författare)
Division of Endocrinology and Diabetes, Department of Pediatrics, Vanderbilt University School of Medicine, Vanderbilt University, Nashville TN, United States
Padidela, Raja (författare)
Department of Paediatric Endocrinology, Royal Manchester Children’s Hospital, Manchester, United Kingdom
Namba, Noriyuki (författare)
Department of Pediatrics, Osaka Hospital, Japan Community Healthcare Organization, Osaka, Japan; Osaka University Graduate School of Medicine, Osaka, Japan
Cheong, Hae, II (författare)
Department of Pediatrics, Hallym University Sacred Heart Hospital, Dongan-gu, Anyang, South Korea
Sochett, Etienne (författare)
Department of Pediatrics, Hospital for Sick Children, Toronto Ontario, Canada
Muroya, Koji (författare)
Department of Endocrinology, Kanagawa Children’s Medical Center, Yokohama, Kanagawa Japan
Tanaka, Hiroyuki (författare)
Division of Pediatrics, Okayama Saiseikai General Hospital Outpatient Center, Kita-ku, Okayama, Japan
Pitukcheewanont, Pisit (författare)
Center of Endocrinology, Diabetes and Metabolism, Children’s Hospital of Los Angeles, Los Angeles CA, United States
Gottesman, Gary S. (författare)
Shriners Hospitals for Children St Louis, St Louis MO, United States
Biggin, Andrew (författare)
Department of Endocrinology, The University of Sydney Children’s Hospital Westmead Clinical School, The Children’s Hospital at Westmead, Westmead New South Wales, Australia
Perwad, Farzana (författare)
Department of Pediatrics, University of California, San Francisco CA, United States
Chen, Angel (författare)
Ultragenyx Pharmaceutical Inc., Novato CA, United States
Merritt, John Lawrence (författare)
Ultragenyx Pharmaceutical Inc., Novato CA, United States
Imel, Erik A. (författare)
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
Engelska.
Ingår i: JBMR PLUS. - : Oxford University Press. - 2473-4039. ; 8:1
Relaterad länk:
https://doi.org/10.1...
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https://urn.kb.se/re...
https://doi.org/10.1...
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  • Tidskriftsartikel (refereegranskat)
Abstract Ämnesord
Stäng  
  • 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.

Ämnesord

MEDICIN OCH HÄLSOVETENSKAP  -- Klinisk medicin -- Endokrinologi och diabetes (hsv//swe)
MEDICAL AND HEALTH SCIENCES  -- Clinical Medicine -- Endocrinology and Diabetes (hsv//eng)

Nyckelord

burosumab
FGF23
phosphate
rare disease
x-linked hypophosphatemia

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ref (ämneskategori)
art (ämneskategori)

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