Classical Complement Pathway Inhibition in a Pediatric Patient With Refractory Immune Thrombocytopenia. (PubMed, Pediatr Blood Cancer) - Oct 6, 2026 - "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."
Journal • Hematological Disorders • Immune Thrombocytopenic Purpura • Pediatrics • Thrombocytopenia • Thrombocytopenic Purpura • Transplantation
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Caileigh Pudela; Catherine M Broome; Sarah Hsieh; Susmita Sarangi; Tara K Suntum
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