Baseline Demographics and Characteristics of 466 Patients with Atypical Hemolytic Uremic Syndrome in the Global aHUS Registry
Notice bibliographique
Résumé
Abstract Introduction:Atypical hemolytic uremic syndrome (aHUS) is a rare, genetic, life-threatening disease of chronic, uncontrolled complement activation leading to thrombotic microangiopathy, renal, and other end-organ damage. Started in April 2012, the aHUS Registry is an observational, non-interventional, multicenter, global registry designed to collect information on patients (pts) with aHUS. By making follow-up data on the aHUS indication for eculizumab (ECU) available, the Registry fulfills postmarketing regulatory requirements while also highlighting the need for and benefit of a sponsor/academia partnership. An independent Scientific Advisory Board ensures data are made accessible for publication. Herein, we report baseline demographics and clinical characteristics of pts enrolled in the aHUS Registry. Methods:Pts of all ages with a clinical diagnosis of aHUS (irrespective of treatment) are eligible for enrollment into the Registry. An identified complement abnormality is not required. Demographic, medical and disease history, treatment, and efficacy and safety outcomes data are collected at Registry enrollment and prospectively thereafter. Results:By July 1, 2014, 466 pts (from Australia, Austria, Belgium, Canada, France, Germany, Israel, Italy, Russia, Spain, Sweden, Switzerland, the UK, and USA) were enrolled in the aHUS Registry. Of these, 261 (56.0%) were treated with ECU and 286 (61.4%) were ≥18 years of age. Family history of aHUS, prior kidney grafts, dialysis, plasma exchange/plasma infusion (PE/PI), and renal impairment were assessed at baseline in ECU- and non–ECU-treated pts (Table). Mean time from aHUS diagnosis to ECU initiation was 2.5 years. Sixty-five pts (24.9%) discontinued ECU; of these, 8 (12.3%) restarted. Conclusions:Since the public disclosure of data from the last aHUS Registry update, pt enrollment has continued to increase. Ongoing and future analyses will further clinicians’ understanding of the history and progression of aHUS. Additional clinical sites are encouraged to enroll pts into the aHUS Registry to facilitate knowledge acquisition and optimization of pt care and quality of life. Disclosures Licht: Achillon Pharmaceuticals: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees, Research Funding, Speakers Bureau; Alexion Pharmaceuticals: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees, Research Funding, Speakers Bureau. Ardissino:Alexion Pharmaceuticals: Consultancy, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau. Ariceta:Alexion Pharmaceuticals: Consultancy, Membership on an entity's Board of Directors or advisory committees. Cohen:Alexion Pharmaceuticals: Consultancy. Gasteyger:Alexion Pharma International Sàrl: Employment, Equity Ownership. Greenbaum:Alexion Pharmaceuticals: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees, Research Funding. Ogawa:Alexion Pharmaceuticals: Employment. Schaefer:Alexion Pharmaceuticals: Consultancy, Membership on an entity's Board of Directors or advisory committees, Research Funding. Vande Walle:Alexion Pharmaceuticals: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees, Research Funding, Speakers Bureau. Fremeaux-Bacchi:Alexion Pharmaceuticals: Consultancy, Membership on an entity's Board of Directors or advisory committees, Research Funding; CLS Behring: Honoraria.
Récupéré en direct depuis OpenAlex et désinversé. Les résumés ne sont pas conservés dans cette base de données : les index inversés représentent 8,6 Go des 9,3 Go de texte de la base, et le serveur dispose de 13 Go libres.
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) | 0,000 | 0,000 |
| Bibliométrie | 0,000 | 0,000 |
| Études des sciences et des technologies | 0,000 | 0,000 |
| Communication savante | 0,000 | 0,000 |
| Science ouverte | 0,000 | 0,000 |
| Intégrité de la recherche | 0,000 | 0,000 |
| Charge utile insuffisante (le modèle a refusé de juger) | 0,000 | 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 ».