Pediatric atypical hemolytic-uremic syndrome due to auto-antibodies against factor H: is there an interest to combine eculizumab and mycophenolate mofetil?

BACKGROUND: Atypical hemolytic and uremic syndrome (aHUS), a thrombotic micro-angiopathy (TMA) caused by deregulation in the complement pathway, is sometimes due to the presence of anti-complement factor H (CFH) auto-antibodies. The "standard" treatment for such aHUS combines plasma exchange therapy and immunosuppressive drugs. Eculizumab, a monoclonal antibody that blocks the terminal pathway of the complement cascade, could be an interesting alternative in association with an immunosuppressive treatment for maintenance regimen. CASE-DIAGNOSIS/TREATMENT: We report on two children, diagnosed with mildly severe aHUS due to anti-CFH antibodies, who were treated with the association eculizumab-mycophenolate mofetil (MMF). Neither side effects nor relapses were observed during the 3 years of follow-up; MMF was even progressively tapered and withdrawn successfully in one patient.
CONCLUSIONS: The association of eculizumab and MMF appears to be an effective and safe option in pediatric cases of aHUS due to anti-CFH antibodies of mild severity.