Real-World Effectiveness of Burosumab in Adults with X-Linked Hypophosphataemia (XLH) in the UK
Bibliographic record
Abstract
X-linked hypophosphataemia (XLH) is a genetic phosphate-wasting disorder caused by excess fibroblast growth factor 23 (FGF23), which leads to skeletal morbidities, pain, stiffness, and impairments in physical function and health-related quality of life. Burosumab inhibits excess circulating FGF23, restoring bone biochemistry. Here we report real-world data from adults with debilitating XLH symptoms who started treatment with burosumab through a UK early access programme. Change from baseline was assessed for bone biochemistry and patient-reported outcomes (PROs) collected from patients' medical records from September 2019 to December 2022. The proportion of patients (n = 136; 66% female, median age 44.0 years [range 18-83]) with normal serum phosphate increased from 5% (6/126) at baseline to 63% (52/82) after 6 months' burosumab treatment; mean serum phosphate increased significantly from baseline. Significant improvements from baseline were observed in Brief Pain Inventory short-form Worst Pain, Pain Severity and Pain Interference scores (mean [SD] improvement at 6 months: 1.8 [2.3], 1.6 [2.1] and 1.9 [2.2] points, respectively). Western Ontario and McMaster Universities Arthritis Index Stiffness, Pain, Physical Function and total scores improved significantly at 6 months (15.9 [29.7], 11.4 [24.3], 15.7 [19.7] and 15.4 [18.3], respectively), as did EuroQol five-dimension five-level (EQ-5D-5L) utility and visual analogue scale (VAS) scores (0.16 [0.22] and 17.0 [21.6]). Most improvements were clinically meaningful (where benchmarks exist). This study demonstrates the effectiveness of burosumab in real-world practice, supporting findings from clinical trials, and provides new evidence that burosumab treatment substantially improves EQ-5D-5L utility and VAS scores in adults with XLH.
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How this classification was reachedexpand
Full frame machine prediction
Teacher imitationNot calibrated prevalence, not ground truth. Human validation pending. The Gemma side is a direct model label for every work in the frame, read from the title-only record. The Codex side is a classifier learned from the 10,348 direct Codex labels and calibrated to design-weighted sample rates; fields without enough sample support carry no Codex call. Candidate is the union of the two sides; consensus is their intersection. These outputs are machine_predicted_unvalidated and are not human labels.
Distilled classifier scores by category (both heads)
| Category | Codex | Gemma |
|---|---|---|
| Metaresearch | 0.001 | 0.007 |
| Meta-epidemiology (narrow) | 0.000 | 0.000 |
| Meta-epidemiology (broad) | 0.001 | 0.001 |
| Bibliometrics | 0.000 | 0.001 |
| Science and technology studies | 0.000 | 0.000 |
| Scholarly communication | 0.001 | 0.000 |
| Open science | 0.000 | 0.001 |
| Research integrity | 0.001 | 0.001 |
| Insufficient payload (model declined to judge) | 0.001 | 0.000 |
Machine scores (provisional)
The two teacher heads of the student model, read on this work. A score orders the frame for review; it never asserts a category, and the validation status ships verbatim with every row.
Baseline scores from an immature model (maturity gate not passed, 7 training rounds). Scores rank; they never assert a category.
score_only:v0-immature-baseline · verbatim from the scoring run: score_only means the number may rank works, and no category label ships from itClassification
machine, unvalidatedMachine predicted; a candidate call from one source (direct Gemma or distilled Codex), not a consensus.
How this classification was reached, model by model and score by score, is at the end of the page under "How this classification was reached".