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Updated: May 12, 2026
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Measuring the 50% Haemolytic Complement CH50 Activity of Serum
Published on: March 29, 2010
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Terminal complement inhibition in atypical haemolytic uremic syndrome: a single-centre experience
Valentin D Mocanu1,2, Bogdan M Sorohan1,2, Elena G Micu2
1Nephrology Department, "Carol Davila" University of Medicine and Pharmacy, Bucharest, Romania.
Frontiers in Pharmacology
|December 3, 2025
Summary
Atypical hemolytic uremic syndrome (aHUS) treatment with anti-C5 therapy effectively manages anemia and thrombocytopenia. This study highlights the safety and efficacy of anti-C5 therapy in a Romanian aHUS cohort, with high remission rates and dialysis independence.
Area of Science:
- Nephrology
- Hematology
- Immunology
Background:
- Atypical hemolytic uremic syndrome (aHUS) is a rare thrombotic microangiopathy (TMA) characterized by complement dysregulation.
- It leads to anemia, thrombocytopenia, and acute kidney injury (AKI), often driven by genetic mutations in complement alternative pathway proteins.
- Genetic profiles and clinical outcomes for aHUS vary significantly by region.
Purpose of the Study:
- To evaluate the efficacy and safety of anti-C5 monoclonal antibody therapy in a Romanian cohort of patients with aHUS.
- To analyze the clinical presentation, genetic profile, and treatment outcomes in this specific patient population.
- To investigate the regional genetic variations, particularly the frequency of CFI variants in the Romanian aHUS cohort.
Main Methods:
- A retrospective observational study included 27 patients (12 children, 15 adults) diagnosed with aHUS between January 2017 and January 2025.
- All patients received anti-C5 monoclonal antibodies and were followed for a median of 13 months.
- Data collected included clinical presentation, triggers, diagnostic timelines, genetic testing (CFH/CFHR, CFI variants), and treatment response.
Main Results:
- Infections were the most common trigger (67%); diagnosis and referral were often delayed (median 30 days from event to admission).
- Genetic testing identified CFH/CFHR variants in 39% and CFI variants in 22% of patients, with higher than expected CFI frequency.
- Anti-C5 therapy achieved high remission rates for anemia (approx. 90%), thrombocytopenia (approx. 90%), C3 normalization (90%), and dialysis independence (74%), with no deaths or serious adverse events.
Conclusions:
- Anti-C5 therapy is effective and safe for treating aHUS in the Romanian cohort, leading to significant clinical improvement and renal recovery.
- The higher prevalence of CFI variants suggests a distinct geographical distribution of genetic factors in Romanian aHUS patients.
- Study limitations include a small patient cohort and observational design, necessitating further research.
