Multidisciplinary consensus on the diagnosis and management of patients with atypical Hemolytic Uremic Syndrome (complement-mediated TMA): Recommendations from Italian scientific societies, patient associations and regulators
- PMID: 40204022
- DOI: 10.1016/j.phrs.2025.107714
Multidisciplinary consensus on the diagnosis and management of patients with atypical Hemolytic Uremic Syndrome (complement-mediated TMA): Recommendations from Italian scientific societies, patient associations and regulators
Abstract
Atypical Hemolytic Uremic Syndrome (aHUS) is a severe, systemic, rare disease (RD) that can occur in people of all ages, and is associated with high rates of morbidity and mortality. Because the management of patients with aHUS can be difficult, more effective strategies should be implemented. Faculty members from several Italian Scientific Societies, Patient Associations and Regional Institutional Experts on RDs met to discuss aHUS management within a multidisciplinary team (MDT), using a Delphi process to develop consensus recommendations. Consensus (≥70 % agreement by faculty members) was reached on 51 statements with the aim of improving patient management and outcomes. These statements provide a unified framework for the differential diagnosis of aHUS, prompt recognition of the pathology, referral to RD reference centers, selecting between treatment relapse prevention measures options, patient management by a MDT and improving the overall awareness of aHUS. Despite the broad scope of the consensus statements, several unmet needs in the management of patients with aHUS were identified, including diagnostic suspicion, rapid genetic investigations, regular review of the centers of expertise (considering the number of treated patients), permanent clinical referral in treatment centers and widespread expertise among adult and pediatric specialists. We hope that this standardized framework will form the basis of the "digital ecosystem" concept and development of possible information technology solutions to assist the MDT involved in the management of patients with aHUS.
Keywords: Atypical hemolytic uremic syndrome; Delphi consensus; Multidisciplinary care; Patient-centered approach.
Copyright © 2025 The Authors. Published by Elsevier Ltd.. All rights reserved.
Conflict of interest statement
Declaration of Competing Interest EDS: was an invited speakers at meetings supported by Alexion and AstraZeneca. MP was a speaker at symposia sponsored by Alexion Pharmaceuticals. RG is a member of the advisory board of Bayer and Roche, and a speaker at bureau/educational meetings of Pfizer, SOBI, Takeda and Novo Nordisk. M Man has received research grants, reimbursement for travel, and consulting payments from Stealth BT, Takeda, Sanofi Genzyme, Khondrion, Abliva, Reneo, Zogenix and Precision Biosciences; is supported by the Telethon (GSP16001), the E-Rare project GENOMIT (01GM1920B) and the Italian Ministry of Health (2022B9WY4A); and is part of the European Reference Networks EURO NMD and RND. GS has received speaker/consulting honoraria from Sanofi, TEVA, Daiichi-Sankyo, Eli Lilly and MSD. AMS was a speaker at symposia sponsored by Alexion, Kyowa Kirin and others; fees were received on behalf of the Association. GS: has received speaker/consulting honoraria from Sanofi, TEVA, Daiichi-Sankyo, Eli Lilly, and MSD. LG: has received research grants from Abionix and Sanofi; has received speaker honoraria from Bayer and Werfen; served as consultant for Baxter, Tavere, Astrazeneca, GSK, Novartis, Chinook, Roche, Reate, Nestle, Otsuka, Gilead Science, Bayer, and Vifor Fresenius. ADL has been member of the advisory board or scientific consultancy of Sanofi and Agomab, and a speaker/chairperson at sponsored symposia by Alexion, Biogen and Roche. M Mazzucato, LS, PC, GC, FDI, SDR, FD, AG, GA, AB, SB, LB, PF, EGF, FF, CL, FM, A Pad, A Pas, GDS, BS, AT, CV and LZ have no interests to declare.
