Classical Complement Pathway Inhibition in a Pediatric Patient With Refractory Immune Thrombocytopenia

ABSTRACT Pediatric refractory immune thrombocytopenia (rITP) is generally characterized by persistent thrombocytopenia and failure to achieve a sustained response to multiple standard therapies. We report a 4‐year‐old female with congenital tufting enteropathy, post‐intestinal transplant patient on immunosuppressants, who developed severe rITP unresponsive to intravenous immunoglobulin, corticosteroids, rituximab, thrombopoietin receptor agonists, and vincristine. Complement inhibition with Berinert led to a rapid response, and sutimlimab led to sustained platelet recovery. This case highlights complement inhibition as a potential therapeutic option for rITP, representing the first reported pediatric case utilizing this approach for treatment‐resistant pediatric ITP.