Patient-reported outcomes from a randomized open-label phase 3 trial comparing burosumab versus conventional therapy in children with X-linked hypophosphatemia: results from the 24-week treatment extension period

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Patient-reported outcomes from a randomized open-label phase 3 trial comparing burosumab versus conventional therapy in children with X-linked hypophosphatemia : results from the 24-week treatment extension period

Padidela, Raja (author): Department of Paediatric Endocrinology, Royal Manchester Children’s Hospital, Manchester, United Kingdom; Faculty of Biology, Medicine and Health, University of Manchester, Manchester, United Kingdom

Whyte, Michael P. (author): Shriners Hospitals for Children - Washington University School of Medicine in St Louis, St Louis MO, USA

Glorieux, Francis H. (author): Shriners Hospital for Children – Canada, McGill University, Montreal QC, Canada

Munns, Craig F. (author): The University of Sydney Children’s Hospital Westmead Clinical School, The Children’s Hospital at Westmead, Westmead, NSW, Australia; Department of Endocrinology, The Children’s Hospital at Westmead, Westmead NSW, Australia

Ward, Leanne M. (author): Department of Pediatrics, University of Ottawa, Ottawa ON, Canada; Division of Endocrinology and Metabolism, Children’s Hospital of Eastern Ontario, Ottawa ON, Canada

Nilsson, Ola, 1970- (author): Örebro universitet,Institutionen för medicinska vetenskaper,Division of Pediatric Endocrinology & Center for Molecular Medicine, Karolinska Institute, Stockholm, Sweden

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

Simmons, Jill H. (author): Department of Pediatrics, Division of Endocrinology and Diabetes, Vanderbilt University School of Medicine, Vanderbilt University, Nashville TN, USA

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

Cheong, Hae Il (author): Department of Pediatrics, Hallym University Sacred Heart Hospital, Anyang, Republic of South Korea

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

Sochett, Etienne (author): Department of Paediatrics, Hospital for Sick Children, Toronto ON, 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, Birmingham, United Kingdom

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

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

Biggin, Andrew (author): The University of Sydney Children’s Hospital Westmead Clinical School, The Children’s Hospital at Westmead, Westmead NSW, Australia

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

Williams, Angela (author): Kyowa Kirin International, Marlow, United Kingdom

Nixon, Annabel (author): Chilli Consultancy, Salisbury, United Kingdom

Sun, Wei (author): Kyowa Kirin Pharmaceutical Development, Princeton NJ, USA

Chen, Angel (author): Department of Medicine and Department of Pediatrics, Indiana University School of Medicine, Indianapolis IN, USA

Skrinar, Alison (author): Ultragenyx Pharmaceutical, Novato CA, USA

Imel, Erik A. (author): Department of Medicine and Department of Pediatrics, Indiana University School of Medicine, Indianapolis IN, USA

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S. Karger, 2022
2022
English.
In: Hormone Research in Paediatrics. - : S. Karger. - 1663-2818 .- 1663-2826. ; 95:Suppl. 2, s. 29-30

Related links:: https://urn.kb.se/re...

Journal article (other academic/artistic)

Abstract Subject headings

In a randomized open-label phase 3 trial in 62 children (1–12 years) with X-linked hypophosphatemia (XLH) (NCT 02915705), switching from conventional therapy (oral phosphate plus active vitamin D) to burosumab, a monoclonal antibody targeting fibroblast growth factor 23, significantly improved serum phosphate concentration, rickets, lower-extremity deformities, growth, mobility, and patient-reported outcomes (PROs) at 64 weeks. Children in Europe, USA, Canada, and Australia who completed 64 weeks’ treatment could continue to receive burosumab in the extension period (burosumab continuation group) or cross over from conventional therapy to burosumab (crossover group) to 124 weeks. A Patient-Reported Outcomes Measurement Information System (PROMIS) questionnaire was used in children aged ≥5 years to measure Pain Interference, Physical Function Mobility, and Fatigue; health-related quality of life was measured using the SF-10 Health Survey for Children (n=35). Here, we describe changes in PROs from baseline to weeks 64 and 88, and report whether the 3-point minimal important difference (MID) was reached for PROMIS domains (Thissen et al., 2016; PMID 26118768). The mean change from baseline exceeded the MID for Pain Interference at weeks 64 and 88 and for Fatigue at week 64 in the burosumab continuation group, and for Pain Interference and Fatigue at week 88 in the crossover group. Similar improvements in SF-10 Physical Health were seen baseline to week 64 in the burosumab continuation group, and week 64 to 88 in the cross-over group. SF-10 Psychosocial Health changed little in either group at the two timepoints.Treatment with burosumab improved Pain Interference and Fatigue beyond the MID in children with XLH who switched from conventional therapy to receive 24 weeks of burosumab. Improvements were also maintained in children who received an additional 24 weeks’ burosumab treatment.

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

MEDICAL AND HEALTH SCIENCES: MEDICAL AND HEAL ...; and Clinical Medicin ...; and Pediatrics

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By the university: Örebro University

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Patient-reported outcomes from a randomized open-label phase 3 trial comparing burosumab versus conventional therapy in children with X-linked hypophosphatemia : results from the 24-week treatment extension period

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