Fatal Bilateral Strokes in a Child With Hemolytic Uremic Syndrome- A Potential Therapeutic Role of Eculizumab

Background: HUS is a life-threatening multisystem disease caused by uncontrolled complement activation. About 25 % of patients have CNS (Central nervous system) involvement, often leading to serious long-term disabilities in young children. Eculizumab, a humanized monoclonal antibody that targets the complement protein C5, has shown to improve the disease course in children with better neurological outcomes. Early use in disease course showed much better results and its potential use as a prophylactic therapy has been indicated. Scoring systems have been developed for prediction of CNS complications, which may help identify potential candidates for prophylactic Eculizumab therapy. Presentation: We present a 3 year old child who presented with Shiga-Toxin Producing E coli Hemolytic-Uremic Syndrome (STEC-HUS) and seizures. One week into hospitalization he suddenly became unresponsive besides withdrawal to pain. Brain MRI revealed multifocal infarcts involving bilateral basal ganglia, thalami, dorsal brainstem, and cerebellar white matter with microhemorrhages. He was started on Eculizumab in an attempt to halt neurological decline. In spite of starting Eculizumab therapy, our patient succumbed to a fatal cardiac arrest. Based on the SCWP ( sodium, CSR, white count and protein) scoring system our patient was at very high risk for developing neurological sequelae even at initial presentation. His therapy was not started until very late in the course of the disease which may have led to the unfavorable outcome Conclusion: Ecluzimab therapy should be initiated early in HUS patients with CNS involvement. Scoring systems may help identify at risk patients and potentially start prophylactic Eculizumab therapy to achieve improved neurological outcomes in children.