The global aHUS registry: methodology and initial patient characteristics

Area of Science:

• Rare diseases
• Complement genetics
• Thrombotic microangiopathy

Background:

• Atypical hemolytic uremic syndrome (aHUS) is a rare, genetic systemic disease.
• It is characterized by uncontrolled complement activation, leading to thrombotic microangiopathy (TMA) and organ damage.

Purpose of the Study:

• To establish a global registry for collecting comprehensive data on aHUS patients.
• To gather information on demographics, disease history, and treatment outcomes.

Main Methods:

• An observational, noninterventional, multicenter registry initiated in April 2012.
• Enrollment is open to all patients with a clinical diagnosis of aHUS.
• Minimum of 5 years of follow-up planned.

Main Results:

• As of September 30, 2014, 516 patients from 16 countries were enrolled.
• 61.0% were adults, 39.0% were pediatric patients.
• 59.1% of patients received eculizumab treatment.

Conclusions:

• The global aHUS Registry is expected to provide valuable baseline and natural history data.
• It will yield insights into medical outcomes, treatment effectiveness, and safety in a diverse aHUS patient population.