SUN-757 Sustained Improvements in Patient-Reported Outcomes Across Age Groups in Burosumab-Treated X-linked Hypophosphatemia (XLH): Results From the XLH Disease Monitoring Program
Bibliographic record
Abstract
Abstract Disclosure: A.A. Khan: Ascendis, Alexion Pharmaceuticals, Inc., Amolyt, Bridge Bio, Takeda. T.O. Carpenter: Kyowa Kirin, Ultragenyx. J. Simmons: Kyowa Kirin, Ultragenyx. H. Cassinelli: None. E.A. Imel: Kyowa Kirin, Ultragenyx. P.F. Florenzano: Kyowa Kirin, Ultragenyx. B. Johnson: Kyowa Kirin, Kyowa Kirin. E. Yang: Ultragenyx. H.M. Heerssen: Kyowa Kirin. J. Li: Kyowa Kirin. L.M. Ward: Kyowa Kirin, Ultragenyx. Introduction: Burosumab, a fully human FGF23-neutralizing antibody, improved outcomes in adults and children with X-linked hypophosphatemia (XLH) in 24- and 64-week randomized controlled trials, respectively. This analysis describes real-world changes in patient-reported outcomes (PROs) over ≤3 years (y) in burosumab-treated individuals with XLH, including age groups with previously limited data (13-17 and >65y). Methods: Participants (pts) initiating burosumab after enrollment and before the Year (Y) 1 visit in the XLH Disease Monitoring Program (DMP); NCT03651505, a multi-country observational study) were evaluated. In children, change from baseline in Patient-Reported Outcomes Measurement Information System (PROMIS) T-scores at Y1 and 3 were evaluated to assess fatigue, pain interference, and physical function (PF) mobility. In adults, change from baseline in Western Ontario and McMaster Universities Osteoarthritis Index (WOMAC) scores and PROMIS PF T-scores at Y1 and 3 visits were evaluated to assess pain severity, joint stiffness, and PF. Results: At baseline (N=116) and Y1 (n=108), most groups included >10 pts, but the >65y group included 3. Y3 (n=76) group sample size range was 3-22. In children (n=83), mean ± standard deviation (SD) PROMIS pain interference improved significantly at Y1 (n=36: −3.6 ± 8.5, P=0.016) and Y3 (n=29: −4.8 ± 10.1, P=0.017). Mean PROMIS fatigue and PF mobility improved descriptively at Y1 and 3. The 5-12y group showed descriptive improvements in all PROMIS domains at Y1 and 3, with a significant change for pain at Y1 (n=25: −4.2 ± 7.3, P=0.008). The 13-17y group showed descriptive improvements in fatigue and pain interference at Y1 and 3. In adults (n=72), mean ± SD WOMAC scores improved significantly at Y1 (pain: −7.5 ± 18.8, P=0.001; stiffness: −12.2 ± 23.6, P<0.0001; PF: −6.3 ± 15.5, P=0.001), and Y3 (n=47) (pain: −11.3 ± 16.9, P<0.0001; stiffness: −16.8 ± 21.7, P<0.0001; PF: −10.8 ± 16.6; P<0.0001). Mean ± SD PROMIS PF score improved significantly at Y1 (1.5 ± 5.6, P=0.025) and remained stable at Y3 (1.6 ± 5.8). All adult groups had descriptive reductions from baseline in all WOMAC domains at Y1 and 3. Significant improvements in mean ± SD scores were seen at Y1 and 3 for pain in the 30-39y (Y1: −7.5 ± 11.3, P=0.0036; Y3: −10.3 ± 12.2, P=0.0054) and 50-64y (Y1: −13.3 ± 18.9, P=0.0333; Y3: −21.9 ± 13.1, P=0.0021) groups, stiffness in the 18-29y (Y1: −22.1 ± 28.5, P=0.057; Y3: −25.0 ± 23.2, P=0.0185) and 30-39y (Y1: −10.4 ± 17.9, P=0.0092; Y3: −13.3 ± 19.2, P=0.175) groups, and PF in the 50-64y (Y1: −8.7 ± 13.5, P=0.0467; Y3: −14.9 ± 9.1, P=0.0023) group. Conclusion: Children with XLH showed early and sustained improvement in pain interference. Adults with XLH also showed significant improvements at DMP Y1 and 3 in all WOMAC domains. Despite small sample sizes, this analysis provides real-world data on the positive impact of burosumab treatment on PROs. Presentation: Sunday, July 13, 2025
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How this classification was reachedexpand
Full frame distilled prediction
Teacher imitationNot calibrated prevalence, not ground truth. Human validation pending. Learned from the 10,348 direct Codex labels and 10,348 direct Gemma labels. Candidate is the union of thresholded teacher heads; consensus is their intersection. These outputs are machine_predicted_unvalidated and are not human labels or direct frontier model labels.
Codex and Gemma teacher scores by category
| Category | Codex | Gemma |
|---|---|---|
| Metaresearch | 0.001 | 0.001 |
| 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 | 0.000 | 0.000 |
| Open science | 0.000 | 0.000 |
| Research integrity | 0.000 | 0.001 |
| Insufficient payload (model declined to judge) | 0.000 | 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 teacher head, 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".