Alternative treatment options for pediatric hemophilia B patients with high‐responding inhibitors: A thrombin generation‐guided study

AA Barg, S Levy‐Mendelovich, E Avishai… - Pediatric Blood & …, 2018 - Wiley Online Library
AA Barg, S Levy‐Mendelovich, E Avishai, R Dardik, M Misgav, G Kenet, T Livnat
Pediatric Blood & Cancer, 2018Wiley Online Library
Little is known about the challenging treatment of pediatric patients with hemophilia B and
inhibitors due to disease rarity. We describe three patients diagnosed in childhood and
followed up to 9 years. All three had allergic reactions to Factor IX, but two were later safely
treated for bleeding episodes with activated prothrombin complex concentrates (APCC=
FEIBA). The third was given only recombinant activated Factor VIIa. Based on ex vivo
thrombin generation analysis, a new alternative treatment of combined bypassing agents …
Abstract
Little is known about the challenging treatment of pediatric patients with hemophilia B and inhibitors due to disease rarity. We describe three patients diagnosed in childhood and followed up to 9 years. All three had allergic reactions to Factor IX, but two were later safely treated for bleeding episodes with activated prothrombin complex concentrates (APCC = FEIBA). The third was given only recombinant activated Factor VIIa. Based on ex vivo thrombin generation analysis, a new alternative treatment of combined bypassing agents was administered for bleeding episodes and several minor surgical procedures with no treatment‐associated adverse events or thrombosis.
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