In typical
hemolytic uremic syndrome (HUS) approximately 25% of patients show central nervous system (CNS) involvement often leading to serious long-term disabilities. We used the C5-complement inhibitor
Eculizumab as rescue
therapy. From 2011 to 2014, 11 children (median age 22 months, range 11-175) with enterohemorrhagic Escherichia coli-positive HUS requiring dialysis who had
seizures (11/11) and/or were in a stupor or
coma (10/11) were treated with
Eculizumab. Two patients enrolled on the Safety and Efficacy Study of
Eculizumab in
Shiga-Toxin Producing E coli
Hemolytic-Uremic Syndrome (STEC-HUS) each received 6 doses of
Eculizumab, 3 patients 2 doses, and 6 patients 1 dose. Laboratory diagnostics of blood samples and magnetic resonance imaging (MRI) were performed as per center practice. Data were analyzed retrospectively. Cranial MRI was abnormal in 8 of 10 patients with findings in the basal ganglia and/or white matter. A 2-year-old boy with severe cardiac involvement and
status epilepticus needed repeated
cardio-pulmonary resuscitation and
extracorporeal membrane oxygenation. He died 8 days after start of
Eculizumab treatment. Two patients with hemorrhagic
colitis and repeated
seizures required artificial ventilation for 6 and 16 days, respectively. At the time of discharge, 1 patient showed severe neurological impairment and 1 mild neurological impairment. The 8 surviving patients experienced no further
seizures after the first dose of
Eculizumab. Three patients showed mild neurological impairment at discharge, whilst the remaining 5 showed no impairment. The platelets normalized 4 days (median) after the first dose of
Eculizumab (range 0-20 days). The mean duration of dialysis after the first dose of
Eculizumab was 14.1 ± 6.1 days. In children with typical HUS and CNS involvement early use of
Eculizumab appears to improve neurological outcome. In severe HUS cases which progress rapidly with multiple organ involvement, late treatment with
Eculizumab seems to show less benefit. We speculate that prophylactic
Eculizumab therapy before development of neurological symptoms could be advantageous.