Congenital Thrombotic Thrombocytopenic Purpura (CTTP): Updates from the American Society of Hematology (ASH) 2019 annual meeting

Details

Thrombotic thrombocytopenic purpura (TTP) is a rare blood disorder that impacts three in a million adults per year, with congenital or hereditary TTP (cTTP, also known as Upshaw-Schulman syndrome) accounting for a third of the overall incidence. The incidence of TTP rises with increasing age and the mortality rate of untreated TTP may be as high as 90%.

In recent years molecular mechanisms contributing to TTP have been identified: patients diagnosed with TTP have larger von Willebrand factor molecules (vWF) and a defective protease enzyme of A Disintegrinlike And Metalloprotease with ThromboSpondin type 1 motif 13 (ADAMTS13), which cleaves larger vWF molecules and inhibits platelet adhesion. In congenital or hereditary TTP (cTTP, also known as Upshaw-Schulman syndrome), the gene that codes of ADAMTS13 is defective and cannot properly produce the enzyme, whereas in acquired TTP, antibody production leads to downstream enzymatic deactivation.

Target Audience:
The following healthcare professionals: hematologists; physician assistants, nurse practitioners, nurses, and pharmacists who practice in hematology; and any other healthcare professionals with an interest in or who clinically encounter patients with cTTP.

By the end of the session the participant will be able to:

• Summarize the most impactful findings presented at the ASH 2019 annual meeting relating to congenital TTP
• Recall the pathophysiology of congenital TTP
• Describe the clinical manifestations and methods of establishing a diagnosis of cTTP, and apply that information to a patient case
• List treatment strategies for cTTP, and apply that information to a patient case