FC 115: Long-Term Outcomes in Eculizumab-Treated Kidney Transplant Patients Enrolled in the Global Atypical Haemolytic Uraemic Syndrome Registry
Notice bibliographique
Résumé
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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Comment cette classification a été obtenuedéplier
Prédiction distillée sur la base complète
Imitation des enseignantsNi prévalence calibrée, ni vérité terrain. Validation humaine à venir. Apprise à partir de 10 348 étiquettes directes de Codex et de 10 348 étiquettes directes de Gemma. Le mode candidate est l'union des têtes enseignantes seuillées; le consensus est leur intersection. Ces sorties portent le statut machine_predicted_unvalidated et ne sont ni des étiquettes humaines ni des étiquettes directes de modèles de pointe.
Scores Codex et Gemma par catégorie
| Catégorie | Codex | Gemma |
|---|---|---|
| Métarecherche | 0,000 | 0,000 |
| Méta-épidémiologie (sens strict) | 0,000 | 0,000 |
| Méta-épidémiologie (sens large) | 0,001 | 0,000 |
| Bibliométrie | 0,000 | 0,001 |
| Études des sciences et des technologies | 0,000 | 0,000 |
| Communication savante | 0,000 | 0,000 |
| Science ouverte | 0,001 | 0,000 |
| Intégrité de la recherche | 0,000 | 0,000 |
| Charge utile insuffisante (le modèle a refusé de juger) | 0,001 | 0,000 |
Scores machine (provisoires)
Les deux têtes enseignantes du modèle étudiant, lues sur ce travail. Un score ordonne la base pour la relecture; il n'affirme jamais une catégorie, et le statut de validation accompagne chaque rangée tel quel.
Scores de référence d'un modèle non mature (critères de maturité non atteints, 7 itérations). Un score ordonne; il n'affirme jamais une catégorie.
score_only:v0-immature-baseline · tel quel depuis la passe de notation : score_only signifie que le nombre peut ordonner les travaux, et qu'aucune étiquette de catégorie n'en découleClassification
machine, non validéePrédiction automatique; un appel candidat d’une seule tête enseignante, pas un consensus.
Le détail, modèle par modèle et score par score, se trouve en fin de page sous « Comment cette classification a été obtenue ».