Observational studyPloS one2026
The lived experience of adolescents with X-linked hypophosphataemia treated with burosumab at end of skeletal growth: a mixed-methods analysis.
Observational study in PloS one, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT05181839 (An Observational, Prospective, European, Multicentre, Mixed Methods Study to Describe the Lived Experience of X-Linked Hypophosphatemia), which is not on this map. Cited by 1 paper.
What it found
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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.
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.
An Observational, Prospective, European, Multicentre, Mixed Methods Study to Describe the Lived Experience of X-Linked Hypophosphatemia (XLH) for Adolescents at End of Skeletal Growth
Who cites it
1 citing paper in PubMed.
- Adolescents' experience of living with X-linked hypophosphataemia (XLH): a mixed-methods analysis of those who continued and discontinued burosumab treatment after end of skeletal growth.Orphanet journal of rare diseases · 2026Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
22 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
X-linked hypophosphataemia is a rare, genetic, lifelong disorder caused by phosphate-regulating endopeptidase homologue X-linked pathogenic variants and, if left untreated, is associated with a progressive accumulation of musculoskeletal manifestations. Burosumab is a fully human monoclonal antibody that targets circulating fibroblast growth factor 23 and directly inhibits its activity, thereby correcting the abnormal phosphate homoeostasis in people with X-linked hypophosphataemia (XLH). The efficacy and safety of burosumab has been demonstrated in a programme of clinical trials in children and adults. Few data describe the experience of adolescents with XLH receiving burosumab treatment before and after skeletal growth ends. This prospective, multicentre, mixed-methods study described the lived experience of adolescents with XLH treated with burosumab at the end of skeletal growth (NCT05181839). Using patient-reported outcomes, wearable devices, and interviews, we found low median symptom severity scores for pain (0.00), stiffness (0.00), and fatigue (1.75) on a 0-10 scale. Symptoms were usually triggered by physical activity but rarely interfered with daily life. Some adolescents reported emotional concerns related to XLH and treatment transition. These insights can inform patient support during transition to adult care.
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Registered trials
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