Research Publications
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Glasner MF, Sukumar S, Cataland SR, Scully M, Chaturvedi S. Optimizing the management of congenital thrombotic thrombocytopenic purpura. Res Pract Thromb Haemost. 2026 Jan 20;9(Suppl 4):103270. doi: 10.1016/j.rpth.2025.103270. PMID: 41641186; PMCID: PMC12866082.
Congenital thrombotic thrombocytopenic purpura (cTTP) is a disorder in which blood clots form in the small blood vessels throughout the body. cTTP is caused by mutations in the ADAMTS-13 gene, leading to microvascular thrombosis (abnormal clotting), hemolytic anemia (destruction of red blood cells), and organ damage.
In this review, researchers summarize the clinical course, complications, and advances in treatment of cTTP. The team reviewed registry data, clinical studies, expert guidelines, and real-world patient experiences, including pregnancy management.
Results suggest that recombinant ADAMTS-13, a therapy that helps restore the ADAMTS13 enzyme, is more effective, safe, and convenient than the standard plasma therapy. Authors note that although recombinant ADAMTS-13 represents a major advance in cTTP management, more studies are needed to evaluate its long-term safety, particularly during pregnancy, and to optimize treatment strategies.