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Therapeutic advances in eculizumab for atypical hemolytic uremic syndrome: a narrative review

Dandan Zhang1, Yichen Xiao2, Hualin Ma3,4,5

  • 1Department of Nephrology, Shenzhen People's Hospital, The Second Clinical Medical College, Jinan University, Shenzhen, China.

Frontiers in Pharmacology
|June 15, 2026
PubMed

Atypical hemolytic uremic syndrome (aHUS) represents an uncommon, life-threatening complement-mediated thrombotic microangiopathy that is linked to both significant renal and extrarenal complications. Eculizumab, a monoclonal antibody targeting complement component C5, has dramatically improved patient outcomes and is widely recognized as a first-line therapy. However, uncertainty remains regarding the optimal dosing regimen, therapeutic monitoring, feasibility of treatment discontinuation, and appropriate duration of therapy in patients with aHUS. This narrative review is based on a PubMed literature search conducted through 2025, with search terms encompassing aHUS, eculizumab, complement inhibitor, treatment discontinuation, and dosing strategies. By integrating evidence from key clinical trials, observational cohorts, and registry-based analyses, a comprehensive evaluation of eculizumab's efficacy and safety profiles is presented. We discuss individualized management strategies, with particular emphasis on dose optimization. Importantly, we focus on the available evidence regarding eculizumab discontinuation, addressing its feasibility, relapse risk, and post-discontinuation monitoring strategies, and suggest that cautious treatment withdrawal can be considered in selected patients under close surveillance. In addition, tailored management approaches for specific aHUS subtypes are discussed. In conclusion, we outline future directions for complement C5 inhibition, emphasizing that the advancement and clinical implementation of novel C5 inhibitors may offer new opportunities for the long-term management of aHUS.

Keywords:
atypical hemolytic uremic syndromecomplement inhibitordose individualizationeculizumabthrombotic microangiopathytreatment discontinuation

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