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

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

Ward, Leanne M. (author): Department of Pediatrics, Faculty of Medicine, University of Ottawa, Ottawa Ontario, Canada

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

Glorieux, Francis H. (author): Department of Surgery, Pediatrics, and Human Genetics, Shriners Hospitals for Children and McGill University, Montreal Quebec, Canada

Portale, Anthony A. (author): Department of Pediatrics, University of California, San Francisco CA, United States

Whyte, Michael P. (author): Shriners Hospitals for Children St Louis, St Louis MO, United States

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

Nilsson, Ola, 1970- (author): 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. (author): Division of Endocrinology and Diabetes, Department of Pediatrics, Vanderbilt University School of Medicine, Vanderbilt University, Nashville TN, United States

Padidela, Raja (author): Department of Paediatric Endocrinology, Royal Manchester Children’s Hospital, Manchester, United Kingdom

Namba, Noriyuki (author): Department of Pediatrics, Osaka Hospital, Japan Community Healthcare Organization, Osaka, Japan; Osaka University Graduate School of Medicine, Osaka, Japan

Cheong, Hae, II (author): Department of Pediatrics, Hallym University Sacred Heart Hospital, Dongan-gu, Anyang, South Korea

Sochett, Etienne (author): Department of Pediatrics, Hospital for Sick Children, Toronto Ontario, Canada

Muroya, Koji (author): Department of Endocrinology, Kanagawa Children’s Medical Center, Yokohama, Kanagawa Japan

Tanaka, Hiroyuki (author): Division of Pediatrics, Okayama Saiseikai General Hospital Outpatient Center, Kita-ku, Okayama, Japan

Pitukcheewanont, Pisit (author): Center of Endocrinology, Diabetes and Metabolism, Children’s Hospital of Los Angeles, Los Angeles CA, United States

Gottesman, Gary S. (author): Shriners Hospitals for Children St Louis, St Louis MO, United States

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

Perwad, Farzana (author): Department of Pediatrics, University of California, San Francisco CA, United States

Chen, Angel (author): Ultragenyx Pharmaceutical Inc., Novato CA, United States

Merritt, John Lawrence (author): Ultragenyx Pharmaceutical Inc., Novato CA, United States

Imel, Erik A. (author): Department of Medicine, Indiana University School of Medicine, Indianapolis IN, United States

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Oxford University Press, 2024
2024
English.
In: JBMR PLUS. - : Oxford University Press. - 2473-4039. ; 8:1

Related links:: https://doi.org/10.1...; show more...; https://urn.kb.se/re...; https://doi.org/10.1...; show less...

Journal article (peer-reviewed)

Abstract Subject headings

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.

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MEDICAL AND HEALTH SCIENCES: MEDICAL AND HEAL ...; and Clinical Medicin ...; and Endocrinology an ...

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

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