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Published on: December 27, 2017

Update in treatment options for congenital and immune thrombotic thrombocytopenic purpura

George M Rodgers1

  • 1Division of Hematology and Hematologic Malignancies, Huntsman Cancer Institute, University of Utah Health Sciences Center, Salt Lake City, UT, United States.

Frontiers in Pharmacology
|July 9, 2026
PubMed

Thrombotic thrombocytopenic purpura (TTP) is a thrombotic microangiopathy resulting either from congenital deficiency (cTTP) or acquired (immune) deficiency (iTTP) of A Disintegrin and Metalloprotease with ThromboSpondin-type 1 motif, member 13 (ADAMTS13). Deficiency of ADAMTS13 leads to disseminated platelet thrombosis and organ dysfunction. High mortality of cTTP is prevented by plasma infusion to replace the deficient protease, or more recently by infusion of recombinant ADAMTS13. Standard treatment of iTTP includes steroids, plasma exchange, and rituximab, with or without caplacizumab. Although standard treatment of iTTP improves mortality, refractory cases persist, indicating the need for additional treatment options. This review summarizes the status of novel treatment options for cTTP and iTTP, including additional recombinant ADAMTS13 products, ADAMTS13 gene therapies, plasma cell-directed therapies (bortezomib, daratumumab) as well as novel inhibitors of von Willebrand factor activity.

Keywords:
ADAMTS13Upshaw-Schulman syndromecaplacizumabplasma exchangerituximabthrombotic thrombocytopenic purpura

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