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Real-world use of complement inhibitors for haemolytic uraemic syndrome: an analysis of the European Rare Kidney Disease Registry cohort
A. Vujović, AL. Sellier-Leclerc, MC. Mancuso, O. Boyer, A. Awan, A. Gargiulo, S. Loos, M. Fila, A. Jankauskiene, G. Ariceta, N. Kanzelmeyer, E. Vidal, M. Van Dyck, TK. Levart, N. Šimánková, S. Decramer, J. Hofstetter, M. Vivarelli, S. Sciascia,...
Status neindexováno Jazyk angličtina Země Anglie, Velká Británie
Typ dokumentu časopisecké články
NLK
Directory of Open Access Journals
od 2018
PubMed Central
od 2018
Europe PubMed Central
od 2018
ROAD: Directory of Open Access Scholarly Resources
od 2018
- Publikační typ
- časopisecké články MeSH
BACKGROUND: Although terminal complement inhibitors transformed the prognosis of atypical haemolytic uraemic syndrome (aHUS) from dismal to favourable, treatment approaches vary due to the intermittent disease nature and high costs. Occasionally, complement inhibition is applied in infectious (i)HUS. We aimed to examine real-world C5 inhibitor use and its impact on patient outcomes. METHODS: This retrospective cohort study used longitudinal data from the European Rare Kidney Disease Registry, collected from 76 nephrology centres across 24 European countries between January 1, 2019 and January 31, 2024. Eligible patients had aHUS or iHUS with onset after January 1, 2011, and/or documented C5 inhibitor use. Exclusions included complement-unrelated HUS, post-transplant HUS, and prophylactic C5 inhibitor use around kidney transplantation. Data, derived from medical records and focused queries, were used to assess C5 inhibitor duration, via time-to-event analysis, and kidney function based on annual creatinine levels. FINDINGS: A total of 238 aHUS and 472 patients with iHUS were included in the analysis. C5 inhibition was applied in 76.5% of aHUS and 18.4% of iHUS, with major utilisation differences between countries (p < 0.0001) and less common use in female patients with aHUS (p = 0.0022). Median (interquartile range) treatment duration was 16.1 (3.6-41.2) months in aHUS and 9 (7-32) days in iHUS. After five years, 56% of genetic, 28% of anti-complement factor H (anti-CFH) antibody-mediated, and 23% of aHUS cases with no identified cause remained on treatment. The long-term (>7 years) risk of treatment resumption was 35% in genetic, 15% in aHUS of no identified cause, and 0% in anti-CFH antibody-mediated aHUS. Post-withdrawal aHUS relapses were mostly mild and did not lead to permanent kidney function impairment, ultimately leading to long-term treatment withdrawal in 92.5% of discontinued cases. INTERPRETATION: Currently, C5 inhibitors are administered in three-quarters of newly diagnosed patients with aHUS in Europe, with varied utilisation and discontinuation practices. Treatment withdrawal is common and safe, although relapses may occur, particularly in genetic aHUS. However, baseline disease severity, selective use in expert centres, and indication bias affect outcome comparability. Findings must be considered in the context of patient-specific factors and disease severity at the time of treatment decisions. FUNDING: This research was supported by the European Reference Network for Rare Kidney Diseases, funded by the European Union within the framework of the "EU4Health Programme 2021-2027".
Division of Nephrology University of Torino Ospedale HUB Torino Nord Turin Italy
Division of Paediatric Nephrology and Dialysis Bambino Gesù Children's Hospital IRCCS Rome Italy
Division of Paediatric Nephrology Children's Health Ireland at Temple Street Temple Street Ireland
Division of Paediatric Nephrology University Hospitals Leuven Leuven Belgium
Division of Paediatric Nephrology University Medical Centre Hamburg Eppendorf Hamburg Germany
Faculty of Medicine University of Ljubljana Ljubljana Slovenia
Paediatric Centre Institute of Clinical Medicine Vilnius University Vilnius Lithuania
Paediatric Nephrology Vall d'Hebron Hospital Autonoma University of Barcelona Barcelona Spain
Citace poskytuje Crossref.org
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