Comparison of Patients with Primary Hyperoxaluria 1 and Late-Stage CKD from the BONAPH1DE Registry and ILLUMINATE-C Lumasiran Trial
Bibliographic record
Abstract
Background: Patients (pts) with primary hyperoxaluria type 1 (PH1) and late-stage chronic kidney disease (CKD) from the real-world observational BONAPH1DE study (BPH1; NCT04982393) and Phase 3 ILLUMINATE-C trial (ILLUM-C; NCT04152200) were reviewed to assess if pt characteristics and outcomes in these real-world and clinical studies are concordant. Methods: BPH1 (N=174; all ages; any stage CKD) included 19 lumasiran ever-treated pts on dialysis and 11 not on dialysis with BL eGFR ≤45 mL/min/1.73m2. ILLUM-C (N=21; all ages; late-stage CKD) included 15 pts on dialysis and 6 not on dialysis with BL eGFR ≤45; all initiated lumasiran. Outcomes after isolated kidney transplant (iKT) in a subset of 7 BPH1 pts (lumasiran-treated at iKT; ≥1 year follow-up; ≥1 post-iKT POx and eGFR value) were compared with 5 similar ILLUM-C pts. Results: In BPH1, pts not on dialysis had a mean (SD) BL eGFR of 18.9 (15.5) and BL POx of 110.5 (108.0) µmol/L; those on dialysis had a mean (SD) BL POx of 185.4 (348.6). Before lumasiran treatment, ILLUM-C pts with BL eGFR ≤45 and not on dialysis had a mean (SD) BL eGFR of 19.8 (9.6) and BL POx of 64.7 (41.3); those on dialysis had a mean (SD) BL POx of 108.4 (29.5; Figure 1). Overall, among lumasiran-treated pts in both studies who underwent iKT, eGFR increased and POx declined post-iKT. In ILLUM-C, POx was already reduced from BL at the time of iKT (19.2-64.5) and declined further post-iKT. Conclusion: BPH1 and ILLUM-C data showed comparable POx reduction, eGFR improvement, and iKT outcomes with lumasiran treatment in both real-world and clinical trial studies. Funding: Commercial Support - Alnylam Pharmaceuticals
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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 | 0.001 |
| Bibliometrics | 0.000 | 0.000 |
| Science and technology studies | 0.000 | 0.000 |
| Scholarly communication | 0.001 | 0.001 |
| Open science | 0.001 | 0.001 |
| Research integrity | 0.001 | 0.001 |
| 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".