Atypical hemolytic uremic syndrome and complement blockade: established and emerging uses of complement inhibition

Area of Science:

• Nephrology
• Hematology
• Immunology

Background:

• Atypical hemolytic uremic syndrome (aHUS) is characterized by microangiopathic hemolysis and thrombocytopenia.
• These hallmark features present a diagnostic dilemma due to their presence in various clinical disorders.
• aHUS is often a diagnosis of exclusion, ruling out conditions like thrombotic thrombocytopenic purpura and vitamin B12 deficiency.

Purpose of the Study:

• To review the literature on the treatment of hemolysis syndromes with complement inhibition.
• To discuss the overlap between various hemolysis syndromes and aHUS.
• To highlight new reports on treating C3 glomerulopathy, Shiga toxin-related HUS, and medication-related TMA.

Main Methods:

• Literature review of complement inhibition therapies.
• Analysis of clinical presentations of aHUS and related disorders.
• Synthesis of recent findings on treating specific hemolysis syndromes.

Main Results:

• Complement activation is implicated in many diseases beyond aHUS.
• Genetic variants in complement regulatory genes predispose individuals to microangiopathic hemolysis.
• Complement inhibition shows promise in treating diverse hemolysis syndromes.

Conclusions:

• Understanding complement activation is crucial for diagnosing and managing aHUS and related conditions.
• Complement inhibition represents a significant therapeutic advance for these disorders.
• Further research into complement-mediated diseases will refine treatment strategies.