Long-term follow-up of patients with congenital thrombotic thrombocytopenia purpura receiving a plasma-derived factor VIII (Koate) that contains ADAMTS13.
Tammuella Chrisentery-SingletonLisa N BoggioManuel D CarcaoSami IbrahimiOsman KhanArash MahajerinAnita RajasekharVivek SharmaMacGregor SteeleMarcela TorresFrank J RodinoShannon L CarpenterPublished in: Haemophilia : the official journal of the World Federation of Hemophilia (2023)
is a safe and well-tolerated source of the missing ADAMTS13 enzyme in patients with hTTP, producing a marked reduction in thrombocytopenia prevalence, low frequency of TTP episodes, and with the added benefit of self- or caregiver-administration.
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