ESPE Abstracts (2022) 95 FC2.4

ESPE2022 Free Communications Bone, Growth Plate and Mineral Metabolism (6 abstracts)

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

Raja Padidela 1,2 , Michael P Whyte 3 , Francis H Glorieux 4 , Craig F Munns 5,6 , Leanne M Ward 7,8 , Ola Nilsson 9,10 , Anthony A Portale 11 , Jill H Simmons 12 , Noriyuki Namba 13,14 , Hae Il Cheong 15 , Pisit Pitukcheewanont 16 , Etienne Sochett 17 , Wolfgang Högler 18,19 , Koji Muroya 20 , Hiroyuki Tanaka 21 , Gary S Gottesman 22 , Andrew Biggin 5 , Farzana Perwad 11 , Angela Williams 23 , Annabel Nixon 24 , Wei Sun 25 , Angel Chen 26 , Alison Skrinar 27 & Erik A Imel 26

1Department of Paediatric Endocrinology, Royal Manchester Children's Hospital, Manchester, United Kingdom; 2Faculty of Biology, Medicine and Health, University of Manchester, Manchester, United Kingdom; 3Shriners Hospitals for Children -Washington University School of Medicine in St Louis, St Louis, MO, USA; 4Shriners Hospital for Children – Canada, McGill University, Montreal, QC, Canada; 5The University of Sydney Children's Hospital Westmead Clinical School, The Children's Hospital at Westmead, Westmead, NSW, Australia; 6Department of Endocrinology, The Children's Hospital at Westmead, Westmead, NSW, Australia; 7Department of Pediatrics, University of Ottawa, Ottawa, ON, Canada; 8Division of Endocrinology and Metabolism, Children's Hospital of Eastern Ontario, Ottawa, ON, Canada; 9Division of Pediatric Endocrinology & Center for Molecular Medicine, Karolinska Institute, Stockholm, Sweden; 10School of Medical Sciences, Örebro University, Örebro, Sweden; 11Department of Pediatrics, University of California, San Francisco, CA, USA; 12Department of Pediatrics, Division of Endocrinology and Diabetes, Vanderbilt University School of Medicine, Vanderbilt University, Nashville, TN, USA; 13Department of Pediatrics, Osaka Hospital, Japan Community Healthcare Organization, Osaka, Japan; 14Department of Pediatrics, Osaka University Graduate School of Medicine, Osaka, Japan; 15Department of Pediatrics, Hallym University Sacred Heart Hospital, Anyang, Republic of South Korea; 16Center of Endocrinology, Diabetes and Metabolism, Children's Hospital Los Angeles, Los Angeles, CA, USA; 17Department of Paediatrics, Hospital for Sick Children, Toronto, ON, Canada; 18Department of Paediatrics and Adolescent Medicine, Johannes Kepler University Linz, Linz, Austria; 19Institute of Metabolism and Systems Research, University of Birmingham, Birmingham, United Kingdom; 20Department of Endocrinology and Metabolism, Kanagawa Children's Medical Center, Yokohama, Japan; 21Okayama Saiseikai General Hospital Outpatient Center, Okayama, Japan; 22Shriners Hospitals for Children, St Louis, MO, USA; 23Kyowa Kirin International, Marlow, United Kingdom; 24Chilli Consultancy, Salisbury, United Kingdom; 25Kyowa Kirin Pharmaceutical Development, Princeton, NJ, USA; 26Department of Medicine and Department of Pediatrics, Indiana University School of Medicine, Indianapolis, IN, USA; 27Ultragenyx Pharmaceutical, Novato, CA, USA

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.

Change from baseline in PROMIS and SF-10 scores

Instrument Domain Week Burosumab Continuation group (n=15) Crossover group (n=20)
PROMIS Pain Interference 64a −3.8 (10.03) −0.5 (7.14)
88b −8.2 (10.70) −3.3 (9.61)
Physical Function Mobility 64a 2.8 (6.96) 0.9 (4.41)
88b 2.7 (7.21) 2.7 (5.13)
Fatigue 64a −3.6 (8.97) −2.1 (8.36)
88b −2.7 (9.89) −4.9 (8.80)
SF-10 Health Survey for Children Physical Health 64a 6.1 (8.47) 0.3 (10.81)
88b 4.8 (10.62) 7.0 (8.70)
Psychosocial Health 64a 1.3 (8.18) 1.2 (6.24)
88b 1.2 (9.50) 2.7 (7.92)
Values are mean (SD); bold indicates that PROMIS-specific MID was reached. Negative change scores indicate improved Pain Interference and Fatigue, positive change scores indicate improved Physical Function Mobility and SF-10 scores.

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.

Volume 95

60th Annual ESPE (ESPE 2022)

Rome, Italy
15 Sep 2022 - 17 Sep 2022

European Society for Paediatric Endocrinology 

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