Study Assessing an Algorithm-based Strategy of Eculizumab Discontinuation in Children and Adults With aHUS
Status:
Completed
Trial end date:
2019-12-03
Target enrollment:
Participant gender:
Summary
Atypical hemolytic syndrome (aHUS) is a severe renal disease affecting children and adults.
It is characterized by the occlusion of intrarenal vessels due to the presence of
platelet/fibrin thrombi, and leads to end-stage renal disease in up to 2/3 of patients. The
discovery of complement alternative pathway as a major risk factor for aHUS has led to the
design of a disease-specific treatment, the anti-C5 monoclonal antibody, eculizumab.
Complement inhibition using eculizumab has clearly improved the renal outcome of aHUS
patients with a dramatic decrease in the risk of end-stage renal disease. However, the
optimal duration of eculizumab therapy is still debated. The present study aims to assess the
feasibility and safety of the discontinuation of eculizumab treatment in children and adults
with aHUS.