SUN-749 Impact of Treatment with Burosumab versus Conventional Therapy on Patient-Important Outcomes in Adult X-Linked Hypophosphatemia: A Retrospective and Prospective Cohort Study
Bibliographic record
Abstract
Abstract Disclosure: D.S. Ali: None. A. Kubba: None. M.H. Alabdely: None. R. Alnajimi: None. A.A. Alraddadi: None. E. Morgante: None. M. Brandi: Takeda, Eli Lilly & Company, Calcilytix, Kyowa Kirin, UCB, Abiogen, Alexion, Amolyt, Amorphical, Bruno Farmaceutici, CoGeDi, Echolight. A.A. Khan: Takeda, Amgen Inc, Alexion, Ascendis, Amolyt, Calcilytix.. Background: X-linked hypophosphatemia (XLH) is a rare inherited disorder affecting BONE AND MINERAL METABOLISM, causing osteomalacia, and other non-skeletal complications in adults. Historically, XLH has been managed with conventional therapy (CT) consisting of phosphate salts and active vitamin D. However, the advent of burosumab, offers a novel treatment pathway. To date, there is limited evidence on the comparative effectiveness of burosumab versus CT on patient-important outcomes in adults including pain, fatigue, quality of life (QoL), and physical function. Methods: We retrospectively reviewed charts of all patients with XLH, confirmed by DNA analysis, at our tertiary referral center in Canada. Phone interviews assessed pain and stiffness using the BPI-SF and WOMAC scales in individuals on burosumab versus CT. Higher BPI-SF scores indicate greater pain severity and life interference, while higher WOMAC scores reflect increased pain, stiffness, and functional limitation. Biochemical outcomes were compared between groups at two time points: 6 months before starting burosumab and 12-24 months after. Results: We included 16 patients (12 on burosumab, 4 on CT) with a mean (SD) age of 42.3 (18) years and BMI of 28.7 (5.6) kg/m². Half of the patients are living with obesity (BMI>30). The mean duration of burosumab use was 3 years. Of our patients, 50% of patients pathogenic PHEX variants, while 12.5% had variants of unknown significance. Over 12-24 months, ALP levels decreased by 8% in the burosumab group but increased by 23% in the CT group. The mean BPI pain score was lower with burosumab (3.5 vs. 4.9, p = 0.431). WOMAC physical function (27.3 vs. 44.3, p = 0.124) and total WOMAC scores (40.0 vs. 62.3, p = 0.122) were also lower with burosumab. Conclusions: Patients on burosumab had lower BPI pain, WOMAC physical function, and total WOMAC scores than those on CT, suggesting less pain, stiffness, and functional limitation. Lower WOMAC physical function scores for patients on burosumab indicate fewer difficulties completing daily tasks such as walking, sitting, and climbing stairs compared to CT. However, this was not statistically significant. More research with larger sample size is needed to confirm these findings and to further assess the impact of burosumab vs CT on outcomes important to adults with XLH. Presentation: Sunday, July 13, 2025
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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 | 0.000 | 0.001 |
| Meta-epidemiology (narrow) | 0.000 | 0.000 |
| Meta-epidemiology (broad) | 0.000 | 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 | 0.000 |
| Research integrity | 0.000 | 0.000 |
| Insufficient payload (model declined to judge) | 0.002 | 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".