Effects of Long-Term Burosumab Treatment on Kidney Health in X-Linked Hypophosphatemia: Results from the XLH Disease Monitoring Program
Bibliographic record
Abstract
Background: X-linked hypophosphatemia (XLH) is a rare disorder characterized by FGF23-mediated renal phosphate wasting leading to significant bone abnormalities. Previously, conventional therapy (CT) was oral phosphate and active vit. D, which confers risk of nephrocalcinosis (NC). Burosumab, a human FGF23-neutralizing antibody, improves renal phosphate wasting and vit. D synthesis. The present study aimed to document the frequency and evolution of NC in burosumab-treated patients with XLH. Methods: Children and adults in the longitudinal XLH Disease Monitoring Program combined with data from burosumab clinical trials were analyzed. We examined serial renal ultrasounds scored for NC and biochemistries including the change in estimated glomerular filtration rates (eGFR), for up to ~9 yrs of burosumab therapy. Results: Prior to burosumab (baseline; BL), 17% of 225 children (mean±SD age, 8±5 yrs) and 39% of 185 adults (40±13 yrs) had NC. Most had previously received CT. At last visit, patients received burosumab for a median (range) 4.0 (1, 9.5) yrs. Of those without BL NC (grade 0), NC scores at last visit remained zero in 96% of children and 94% of adults. Of those with BL NC (grades 1, 2 or 3), NC scores at last exam in children were unchanged in 66%, lower in 24%, and worsened in 11%, and in adults were unchanged in 44%, lower in 47%, and worsened in 10%. eGFR was decreased (CKD Stage 2) in 10% of children at BL and 10% of children at last examination, with no change from BL in mean eGFR regardless of NC status. eGFR in adults was decreased (CKD Stage 2, 3, 4) in 15% at BL and 24% at last exam. In adults, mean eGFR decreased from BL to last visit by ~1 ml/min/1.73m2 per yrs regardless of NC status. Serum phosphorus and alkaline phosphatase improvements were sustained, and serum Ca and parathyroid hormone remained stable. Conclusion: The risk of de novo or worsening NC during burosumab treatment was low. In those with NC at BL, NC scores were stable or improved in most patients during burosumab treatment. Continued monitoring of renal health is important in patients with XLH on burosumab or CT. Funding: Commercial Support - Ultragenyx Pharmaceutical Inc.
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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 | 0.003 | 0.003 |
| Meta-epidemiology (narrow) | 0.000 | 0.000 |
| Meta-epidemiology (broad) | 0.001 | 0.000 |
| Bibliometrics | 0.000 | 0.000 |
| Science and technology studies | 0.000 | 0.000 |
| Scholarly communication | 0.000 | 0.000 |
| Open science | 0.000 | 0.000 |
| Research integrity | 0.000 | 0.000 |
| 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".