FC 115: Long-Term Outcomes in Eculizumab-Treated Kidney Transplant Patients Enrolled in the Global Atypical Haemolytic Uraemic Syndrome Registry
Bibliographic record
Abstract
Abstract BACKGROUND AND AIMS The global atypical haemolytic uraemic syndrome (aHUS) registry (NCT01522183), the largest repository of real-world data on patients with aHUS, provides key insights into disease course, natural history, and patient (pt) outcomes with and without complement inhibitor therapy. Prior to eculizumab availability, pts with aHUS had a reportedly high risk of disease recurrence and graft loss following a kidney transplant (KTx). We report the long-term clinical outcomes of eculizumab-treated pts who received a KTx. METHOD This analysis included pts enrolled from April 2012–November 2021. Pts were included in a long-term treatment outcomes study if they had ever been treated with eculizumab and were excluded if they had DGKE mutations or their diagnosis was later revised to any condition other than aHUS. The current analysis set included pts who had received a KTx no more than 30 days prior to eculizumab start or up to 60 days post eculizumab start date, had ≥3 years of follow-up (FU) post-transplant and had serum creatinine (SCr) measurements at baseline (BL) and post 3 years FU. Changes in kidney function, haematological parameters and clinical characteristics at last follow-up (LFU) were assessed. RESULTS A total of 329/1970 adult and paediatric registry pts met the inclusion criteria and received a KTx; 78 pts from 12 countries met sub-group inclusion criteria. BL demographics are shown in Table 1. Mean (SD) treatment duration was 5.7 (2.4) years and 47 (60%) pts had pathogenic genetic variants. Lab parameters at BL (closest measurement at or following KTx) and LFU are reported in Table 2. Despite large ranges, median SCr levels remained stable at LFU. Median eGFR and platelet levels were similar at BL and LFU, while lower median lactose dehydrogenase levels were seen at LFU. At LFU: four pts (5%)—three adult—had subsequent KTx(s); eight (10%)—seven adults—had received dialysis by last observation; and two (3%) had received plasma exchange/infusion. Two pts (3%) died during the observation period. CONCLUSION This study assessed a large, real-world population of pts at high risk of KTx failure. Lab parameters suggest most pts benefitted from eculizumab treatment, with few TMA-related complications and stable kidney function and haematological parameters for ≥3 years. Overall, the proportions of female pts and of pts with pathogenic genetic variants were consistent with previous studies. A potential study limitation was the relatively stringent sub-population-specific inclusion criteria. However, these data further demonstrate the beneficial impact of eculizumab treatment in the long-term management of aHUS in pts requiring KTx.
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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 | 0.001 |
| Science and technology studies | 0.000 | 0.000 |
| Scholarly communication | 0.000 | 0.000 |
| Open science | 0.001 | 0.000 |
| Research integrity | 0.000 | 0.000 |
| Insufficient payload (model declined to judge) | 0.001 | 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 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".