New research from The IMPACT Study. This summary is based on a paper published in the journal Research and Practice in Thrombosis and Haemostasis on January 20, 2026, titled "Optimizing the management of congenital thrombotic thrombocytopenic purpura."

Read the paper here. 

Learn more about IMPACT. 

Transcript: 

New research from The IMPACT Study, a research group of the Rare Diseases Clinical Research Network.

Advancing Treatment in Congenital Thrombotic Thrombocytopenic Purpura.

This summary is based on a paper published in the journal Research and Practice in Thrombosis and Haemostasis on January 20, 2026. 

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.