Long-Term Outcomes in Eculizumab-Treated Patients Enrolled in the Global aHUS Registry
Bibliographic record
Abstract
Background: The global atypical hemolytic uremic syndrome (aHUS) Registry (NCT01522183) is the largest repository of real-world data on patients with aHUS. We report long-term (LT) outcomes with eculizumab (ecu) treatment in two patient cohorts: those with LT follow-up (FU) without progression to end-stage kidney disease (ESKD) and those who developed ESKD during treatment. Our aim was to determine whether LT ecu preserves kidney function. Methods: The analysis included registry patients enrolled between Apr 2012-Nov 2021 and treated with ecu for ≥90 days. Cohort A included patients with ≥3-year FU, no ESKD, and two creatinine values; near treatment start and after ≥3-year observation. Cohort B included patients who developed ESKD after treatment with no FU duration specified. Results: Demographics and laboratory parameters (baseline [BL], last FU [LFU]) are shown in the Table. For Cohort A, 249 patients met inclusion criteria; at LFU, eGFR improved, 7% received acute dialysis (9% of adults, 6% of children) and two adults died. For Cohort B, 56 patients met inclusion criteria; at LFU, 48% received a kidney transplant (38% of adults, 68% of children), 89% received dialysis (95% of adults, 79% of children) and eight died (six adults, two children). BL eGFR was lower in patients who progressed to ESKD (Cohort B, N=56) than in patients who did not progress (Cohort A, N=249). Platelet and LDH levels improved in both cohorts at LFU. Conclusions: In a real-world setting, eGFR was stable in most patients receiving ecu. Some patients demonstrated hematologic benefits but no renal response; they had lower BL eGFR and initiated treatment late. Funding: Commercial Support - Alexion, AstraZeneca Rare Disease
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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 | 0.000 |
| 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 | 0.001 |
| Scholarly communication | 0.000 | 0.000 |
| Open science | 0.001 | 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".