SO050LONG-TERM FOLLOW-UP STUDY OF ECULIZUMAB FOR PREVENTION OF THROMBOTIC MICROANGIOPATHY IN PATIENTS WITH ATYPICAL HEMOLYTIC UREMIC SYNDROME
Bibliographic record
Abstract
Introduction and Aims: In previous prospective clinical trials and a retrospective study, eculizumab prevented thrombotic microangiopathy (TMA) and improved renal function and hematologic parameters in 130 patients (pts) with atypical hemolytic uremic syndrome (aHUS). Pts could enroll in a long-term follow-up study to evaluate TMA event rates during eculizumab treatment and discontinuation. Methods: This is an ongoing, observational, multicenter study (NCT01522170) of aHUS pts treated with eculizumab in 5 prior clinical studies. Primary endpoint is TMA event rate post-parent study during on- (ON; among pts receiving eculizumab) and off-treatment (OFF; among pts who discontinued) periods. A secondary endpoint is TMA event rate ON per label (dose and interval) vs OFF. Results: The current study included 87 pts: 76 with ON and 39 with OFF periods with median follow-up of 26.1 and 20.1 months. Baseline clinical characteristics differed between groups (Table). OFF pts had higher estimated glomerular filtration rates at time of discontinuation than ON pts; 17 (44%) reinitiated eculizumab. TMA event rate was 63% lower ON vs OFF, and 74% lower for labeled dosing vs OFF (Table). OFF TMA events were more frequently severe (Table). Overall, TMA event rates were higher among pts with genetic mutations (9.8/100 patient-years for ON vs 34.5 for OFF) vs no mutation (2.8 vs 7.8) (hazard ratio, 4.5; P=0.0082).
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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 | 0.000 | 0.000 |
| 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) | 0.000 | 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".