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Alternative treatment options for pediatric hemophilia B patients with high‐responding inhibitors: A thrombin generation‐guided study
Author(s) -
Barg Assaf Arie,
LevyMendelovich Sarina,
Avishai Einat,
Dardik Rima,
Misgav Mudi,
Kenet Gili,
Livnat Tami
Publication year - 2018
Publication title -
pediatric blood and cancer
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.116
H-Index - 105
eISSN - 1545-5017
pISSN - 1545-5009
DOI - 10.1002/pbc.27381
Subject(s) - medicine , prothrombin complex concentrate , adverse effect , recombinant factor viia , thrombosis , prothrombin complex , thrombin generation , factor ix , factor viia , surgery , coagulation , thrombin , intensive care medicine , pediatrics , warfarin , platelet , tissue factor , atrial fibrillation
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.