Successful treatment of congenital TTP with a novel approach using plasma-derived factor VIII.

We describe a 19-year-old boy who was diagnosed with congenital thrombotic thrombocytopenic purpura (cTTP) at 7 months of age. He was subsequently treated with fresh frozen plasma infusions every 3 to 4 weeks for the next 15 years at which point he developed significant hypersensitivity reactions to fresh frozen plasma. He required immunosuppressive therapy with systemic desensitization in the intensive care unit but did not tolerate this regimen and suffered debilitating adverse effects. On the basis of the observations from United Kingdom, he was started on a trial with Koate, a plasma-derived factor VIII concentrate with ADAMTS-13 activity that is commercially available in the United States. He tolerated Koate without any complications and attained a target platelet count of>100,000/μL. He has now been in remission for 36 months and responds to exacerbations of cTTP with additional doses of Koate. For patients with cTTP who are intolerant to plasma infusions, therapy with select plasma-derived factor concentrates with ADAMTS-13 activity may represent a reasonable alternative therapy.