Evidence map›Paper›PMID 41445553›Full record

ArticleJBMR plus2025

Patient and carer perceptions and acceptability of current management practices in paediatric X-linked hypophosphatemia treated with burosumab therapy.

Jessica L Sandy, Christine P Rodda, Aris Siafarikas, Andrew Biggin, Christie-Lee Wall, Lucy Collins, Aaron Schindeler, Peter J Simm, Craig F Munns

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in JBMR plus, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.

0numbers the graph read from it
0cells of the map it votes in
1citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

1 citing paper in PubMed.

  1. Article
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

9 authors.

Jessica L SandyInstitute of Endocrinology and Diabetes, The Children's Hospital at Westmead, Westmead, NSW 2145, Australia.ORCID https://orcid.org/0000-0002-2406-8419
Christine P RoddaCentre for Hormone Research, Murdoch Children's Research Institute, Melbourne, VIC 3002, Australia.
Aris SiafarikasDepartment of Endocrinology and Diabetes, Perth Children's Hospital, Nedlands, WA 6009, Australia.
Andrew BigginInstitute of Endocrinology and Diabetes, The Children's Hospital at Westmead, Westmead, NSW 2145, Australia.
Christie-Lee WallInstitute of Endocrinology and Diabetes, The Children's Hospital at Westmead, Westmead, NSW 2145, Australia.
Lucy CollinsDepartment of Endocrinology and Diabetes, Royal Children's Hospital, Melbourne, VIC 3052, Australia.ORCID https://orcid.org/0000-0002-4957-1483
Aaron SchindelerBioengineering and Molecular Medicine Laboratory, The Children's Hospital at Westmead and the Westmead Institute for Medical Research, Westmead, NSW 2145, Australia.
Peter J SimmCentre for Hormone Research, Murdoch Children's Research Institute, Melbourne, VIC 3002, Australia.
Craig F MunnsChild Health Research Centre, Faculty of Medicine, The University of Queensland, Brisbane, QLD 4072, Australia.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

X-linked hypophosphatemia (XLH) is an X-linked dominant condition where fibroblast growth factor-23 (FGF23) excess leads to hypophosphatemic rickets, lower limb bowing, and musculoskeletal pain. Burosumab, a monoclonal antibody against FGF23, has been shown to ameliorate the clinical phenotype of XLH and has recently been approved for use in many countries. This study aimed to evaluate patient and parental/caregiver perception of burosumab therapy and the acceptability of current management practices in Australia. Children with XLH and parents/carers were invited to respond to a survey on clinical and management information including use of telehealth, access to multidisciplinary team members, and perceptions and experience regarding burosumab therapy. This was a multi-centre, cross-sectional survey-based study involving 4 tertiary Australian children's hospitals. A total of 21 survey responses from parents/carers were received between December 2022 and October 2023. Mean (SD) age at time of survey was 12.7 (4.1) yr and median time on burosumab was 42 mo (range 2-100). Reported side effects of burosumab were limited to local skin reactions (38%,

Indexed as

burosumabhypophosphatemiapaediatricsXLHX-linked hypophosphatemia

Identifiers

PMID41445553
PMCPMC12723661

What OpenQuestion holds

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Registered trials

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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.