Characterization of patients with aHUS and associated triggers or clinical conditions: A Global aHUS Registry analysis.

Autor: Licht C; Division of Nephrology, The Hospital for Sick Children, Toronto, Ontario, Canada., Al-Dakkak I; Alexion, AstraZeneca Rare Disease, Boston, Massachusetts, USA., Anokhina K; Alexion, AstraZeneca Rare Disease, Boston, Massachusetts, USA., Isbel N; Department of Nephrology, Princess Alexandra Hospital, Brisbane, Queensland, Australia., Frémeaux-Bacchi V; Laboratoire d'Immunologie, Hôpital Européen Georges Pompidou, Paris, France., Gilbert RD; Regional Paediatric Nephro-Urology Unit, Southampton Children's Hospital, Southampton, UK., Greenbaum LA; Division of Pediatric Nephrology, Emory University School of Medicine and Children's Healthcare of Atlanta, Atlanta, Georgia, USA., Ariceta G; Department of Pediatric Nephrology, Vall d'Hebron Hospital, Autonomous University of Barcelona, Barcelona, Spain., Ardissino G; Centro per la Cura e lo Studio della Sindrome Emolitico-Uremica, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan, Italy., Schaefer F; Division of Pediatric Nephrology, Heidelberg University Hospital, Heidelberg, Germany., Rondeau E; Urgences Néphrologiques et Transplantation Rénale, Hôpital Tenon, Paris, France.
Jazyk: angličtina
Zdroj: Nephrology (Carlton, Vic.) [Nephrology (Carlton)] 2024 Aug; Vol. 29 (8), pp. 519-527. Date of Electronic Publication: 2024 Apr 11.
DOI: 10.1111/nep.14304
Abstrakt: Introduction: Atypical haemolytic uremic syndrome (aHUS) is a rare form of thrombotic microangiopathy (TMA) associated with complement dysregulation; aHUS may be associated with other 'triggers' or 'clinical conditions'. This study aimed to characterize this patient population using data from the Global aHUS Registry, the largest collection of real-world data on patients with aHUS.
Methods: Patients enrolled in the Global aHUS Registry between April 2012 and June 2021 and with recorded aHUS-associated triggers or clinical conditions prior/up to aHUS onset were analysed. aHUS was diagnosed by the treating physician. Data were classified by age at onset of aHUS (< or ≥18 years) and additionally by the presence/absence of identified pathogenic complement genetic variant(s) and/or anti-complement factor H (CFH) antibodies. Genetically/immunologically untested patients were excluded.
Results: 1947 patients were enrolled in the Global aHUS Registry by June 2021, and 349 (17.9%) met inclusion criteria. 307/349 patients (88.0%) had a single associated trigger or clinical condition and were included in the primary analysis. Malignancy was most common (58/307, 18.9%), followed by pregnancy and acute infections (both 53/307, 17.3%). Patients with an associated trigger or clinical condition were generally more likely to be adults at aHUS onset.
Conclusion: Our analysis suggests that aHUS-associated triggers or clinical conditions may be organized into clinically relevant categories, and their presence does not exclude the concurrent presence of pathogenic complement genetic variants and/or anti-CFH antibodies. Considering a diagnosis of aHUS with associated triggers or clinical conditions in patients presenting with TMA may allow faster and more appropriate treatment.
(© 2024 The Authors. Nephrology published by John Wiley & Sons Australia, Ltd on behalf of Asian Pacific Society of Nephrology.)
Databáze: MEDLINE
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