Observational studyThe Journal of clinical endocrinology and metabolism2026
Real-world impact of treatment on growth in children with X-linked hypophosphatemia.
Observational study in The Journal of clinical endocrinology and metabolism, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to 3 registered trials, which are not on this map. Not yet cited in PubMed.
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 International, Multicentre, Prospective, Non-interventional Observational Registry for Patients With X-Linked Hypophosphatemia (XLH)
X-linked Hypophosphatemia Disease Monitoring Program (XLH-DMP)
An International, Multicenter, Prospective, Longitudinal Observational Study for Patient With X-linked Hypophosphatemic Rickets/Osteomalacia
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Authors and funding
13 authors.
Funding
Abstract
contextX-linked hypophosphatemia (XLH) is a rare, genetic, progressive, lifelong disorder manifest by impaired growth and disproportionate short stature. Burosumab, a monoclonal antibody against fibroblast growth factor 23, is approved for treating patients with XLH.
objectiveTo understand the impact of burosumab treatment on growth in a real-world setting.
designInterim data from 3 ongoing, real-world observational studies (NCT03651505, NCT03193476, and NCT03745521), were unified into a single study (APEX).
settingOutpatient clinics. PATIENTS: Children aged 2-17 years with XLH. INTERVENTION(S): Subcutaneous burosumab vs oral phosphate salts and active vitamin D or no treatment. MAIN OUTCOME MEASURE(S): Retrospective and prospective height data from children enrolled in APEX were analyzed to provide long-term estimation of the impact of treatment on growth. Growth and height were estimated by a mixed regression model with separate models for children and adolescents.
resultsIn total, 641 participants were analyzed (402 female) with 498 burosumab-treated and 143 burosumab naïve. Median (interquartile range [IQR]) enrollment ages were 8 (5-12) years for burosumab-treated and 10 (4-14) years for burosumab-naïve participants. Burosumab-treated participants experienced improved growth over burosumab-naïve participants (additional growth velocity: 0.085 Z-score/year for children [P < .0001]; 0.121 Z-score/year for adolescents [P < .0001]). Modeling predicted greater adult height among burosumab-treated participants.
conclusionBurosumab had a robust, positive association with improved growth outcomes in male and female children and adolescents with XLH. Modeling based on a median of 3.3 years of follow-up predicted burosumab can support improvement of growth that will likely result in greater adult height.
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Registered trials
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