- Research Article
- 10.5604/01.3001.0055.4601
Atypical hemolytic- uremic syndrome (aHUS) – a rare disease, new hopes in treatment. Review
- Nov 28, 2025
- Medical Science Pulse
- Marta Turek + 9 more +9
Atypical hemolytic uremic syndrome (aHUS) is a rare disease stemming from complement system dysregulation, manifesting as thrombotic microangiopathy (TMA). It is also described as a heritable, life-threatening disorder characterized by uncontrolled complement activation, leading to systemic TMA and damage to vital organs. aHUS is frequently considered a prototypical form of complement-mediated TMA (CM-TMA), resulting from the dysregulation and overactivation of the alternative complement pathway, culminating in excessive terminal complement activation. The classical clinical triad of aHUS encompasses thrombocytopenia, microangiopathic hemolytic anemia and acute kidney injury.This review paper aims to provide a comprehensive overview of atypical hemolytic uremic syndrome (aHUS). Specifically, this work intends to define aHUS, encompassing its pathomechanism, clinical presentation, and diagnostic criteria. Attention is also directed towards secondary aHUS, which can occur in association with various underlying conditions. Furthermore, this review covers current, available treatment modalities utilizing eculizumab and ravulizumab, providing a comparative analysis of these two agents and highlighting their significance as terminal complement pathway inhibitors. The fundamental mechanism underlying aHUS is the uncontrolled activation of the complement system, particularly its alternative pathway, leading to systemic TMA and life-threatening organ damage. The management of aHUS has been transformed with the introduction of terminal complement inhibitors, such as eculizumab and ravulizumab, which have demonstrated efficacy in improving hematological and renal parameters. Ravulizumab, a long-acting C5 inhibitor, allows for less frequent dosing compared to eculizumab. This paper underscores the importance of early recognition and the implementation of complement inhibitor therapy in aHUS and indicates the necessity for future comparative studies of eculizumab and ravulizumab to optimize treatment strategies.
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