Long-term follow-up of patients with congenital thrombotic thrombocytopenia purpura receiving a plasma-derived factor VIII (Koate) that contains ADAMTS13.

Tammuella Chrisentery-Singleton,Lisa N Boggio,Manuel D Carcao,Sami Ibrahimi,Osman Khan,Arash Mahajerin,Anita Rajasekhar,Vivek Sharma,MacGregor Steele,Marcela Torres,Frank J Rodino,Shannon L Carpenter

Haemophilia : the official journal of the World Federation of Hemophilia（2023）

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摘要

These data suggest that FVIII/VWF 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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关键词

ADAMTS13 protein, congenital thrombotic thrombocytopenic, factor VIII/administration and dosage, microangiopathic haemolytic anaemia, paediatric, prophylaxis, purpura, Upshaw-Schulman syndrome, von Willebrand factor-cleaving protease

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