Patient-Reported Outcomes from a Randomized, Active-Controlled, Open-Label, Phase 3 Trial of Burosumab Versus Conventional Therapy in Children with X-Linked Hypophosphatemia

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Patient-Reported Outcomes from a Randomized, Active-Controlled, Open-Label, Phase 3 Trial of Burosumab Versus Conventional Therapy in Children with X-Linked Hypophosphatemia

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

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): Karolinska Institutet,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, San Francisco, CA, USA

Simmons, Jill H. (author): Departments 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): Seoul National University Children's Hospital, Seoul, Republic of 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, UK

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

Tanaka, Hiroyuki (author): Okayama Saiseikai General Hospital Outpatient Center, Okayama, 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, San Francisco, CA, USA

Williams, Angela (author): Kyowa Kirin International, Marlow, UK

Nixon, Annabel (author): Chilli Consultancy, Salisbury, UK

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

Chen, Angel (author): Ultragenyx Pharmaceutical, Novato, CA, 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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2021-01-23
2021
English.
In: Calcified Tissue International. - : Springer. - 0171-967X .- 1432-0827. ; 108, s. 622-633

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

Journal article (peer-reviewed)

Abstract Subject headings

Changing to burosumab, a monoclonal antibody targeting fibroblast growth factor 23, significantly improved phosphorus homeostasis, rickets, lower-extremity deformities, mobility, and growth versus continuing oral phosphate and active vitamin D (conventional therapy) in a randomized, open-label, phase 3 trial involving children aged 1-12 years with X-linked hypophosphatemia. Patients were randomized (1:1) to subcutaneous burosumab or to continue conventional therapy. We present patient-reported outcomes (PROs) from this trial for children aged ≥ 5 years at screening (n = 35), using a Patient-Reported Outcomes Measurement Information System (PROMIS) questionnaire and SF-10 Health Survey for Children. PROMIS pain interference, physical function mobility, and fatigue scores improved from baseline with burosumab at weeks 40 and 64, but changed little with continued conventional therapy. Pain interference scores differed significantly between groups at week 40 (- 5.02, 95% CI - 9.29 to - 0.75; p = 0.0212) but not at week 64. Between-group differences were not significant at either week for physical function mobility or fatigue. Reductions in PROMIS pain interference and fatigue scores from baseline were clinically meaningful with burosumab at weeks 40 and 64 but not with conventional therapy. SF-10 physical health scores (PHS-10) improved significantly with burosumab at week 40 (least-squares mean [standard error] + 5.98 [1.79]; p = 0.0008) and week 64 (+ 5.93 [1.88]; p = 0.0016) but not with conventional therapy (between-treatment differences were nonsignificant). In conclusion, changing to burosumab improved PRO measures, with statistically significant differences in PROMIS pain interference at week 40 versus continuing with conventional therapy and in PHS-10 at weeks 40 and 64 versus baseline.

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MEDICAL AND HEALTH SCIENCES: MEDICAL AND HEAL ...; and Health Sciences; and Physiotherapy

Articles in the publication: Calcified Tissue ...

By the university: Örebro University; Karolinska Institutet

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Patient-Reported Outcomes from a Randomized, Active-Controlled, Open-Label, Phase 3 Trial of Burosumab Versus Conventional Therapy in Children with X-Linked Hypophosphatemia

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