Recommendations for the individualised management of atypical hemolytic uremic syndrome in adults

Ana Ávila; Mercedes Cao; Mario Espinosa; Joaquín Manrique; Enrique Morales

doi:10.3389/fmed.2023.1264310

Recommendations for the individualised management of atypical hemolytic uremic syndrome in adults

Front Med (Lausanne). 2023 Dec 1:10:1264310. doi: 10.3389/fmed.2023.1264310. eCollection 2023.

Authors

Ana Ávila^#¹, Mercedes Cao^#², Mario Espinosa^#³, Joaquín Manrique^#⁴, Enrique Morales^#⁵

Affiliations

¹ Department of Nephrology, Hospital Universitario Dr. Peset, Valencia, Spain.
² Department of Nephrology, Hospital Universitario A Coruña, A Coruña, Spain.
³ Department of Nephrology, Hospital Universitario Reina Sofía, Córdoba, Spain.
⁴ Department of Nephrology, Complejo Hospitalario de Navarra, Pamplona, Spain.
⁵ Department of Nephrology, Hospital Universitario 12 de Octubre, Madrid, Spain.

^# Contributed equally.

Abstract

Background: Despite significant advances in therapeutic management of atypical hemolytic uremic syndrome (aHUS), guidelines are not timely updated and achieving a consensus on management recommendations remains a topic of ongoing discussion.

Methods: A Scientific Committee with five experts was set up. A literature review was conducted and publications addressing the classification of aHUS, patient profiles and therapeutic approach were selected. Recommendations were proposed at an initial meeting, evaluated through an online questionnaire and validated during a second meeting.

Results: Patients with confirmed or clear suspicion of aHUS should be treated with C5 inhibitors within 24 h of the diagnosis or suspicion of aHUS. Treatment monitoring and the decision to interrupt treatment should be individualised according to the risk of relapse and each patient's evolution. aHUS with a genetic variant or associated with pregnancy should be treated for at least 6-12 months; de novo aHUS associated with kidney transplant until renal function is recovered and genetic variants are ruled out; aHUS associated with malignant hypertension until genetic variants are ruled out; aHUS associated with non-kidney transplant, autoimmune diseases, infection-or drug-induced until the thrombotic microangiopathy is resolved. Patients with a high risk of relapse should be treated for longer than 6-12 months.

Conclusion: These recommendations provides physicians who are not familiar with the disease with recommendations for the management of aHUS in adults. The experts who participated advocate early treatment, maintenance for at least 6-12 months and treatment interruption guided by genetic background, trigger factors, risk of relapse and evolution.

Keywords: C5 inhibitor; atypical hemolytic uremic syndrome; early treatment initiation; thrombotic microangiopathy; treatment interruption.

Grants and funding

The author(s) declare financial support was received for the research, authorship, and/or publication of this article. This project resulted from an advisory board funded by Alexion, AstraZeneca Rare Disease, Boston, MA, USA. Following the advisory board meeting, the attendees independently decided to draft a manuscript based on their discussion; Alexion had no role in this decision yet did sponsor the medical writing for the manuscript, which was performed by Cristina Calle from Adelphi Targis S.L. Alexion provided a courtesy review of the manuscript just prior to submission; however, the authors maintained complete control over the manuscript content, and it reflects their opinions. The authors did not receive any specific grant from funding agencies in the public, commercial or not-for-profit sectors.