Alternative treatment options for pediatric hemophilia B patients with high‐responding inhibitors: A thrombin generation‐guided study. Issue 12 (19th September 2018)

• Record Type: Journal Article
• Authors: Barg, Assaf Arie; Levy‐Mendelovich, Sarina; Avishai, Einat; Dardik, Rima; Misgav, Mudi; Kenet, Gili; Livnat, Tami
• Is Part Of: Pediatric blood & cancer. Volume 65:Issue 12(2018)
• Page Start: n/a
• Page End: n/a
• Publication Date: 2018-09-19
• Subjects: FEIBA -- hemophilia B -- inhibitor -- rFVIIa -- thrombin generation; Tumors in children -- Periodicals; Blood -- Diseases -- Periodicals; Cancer in children -- Periodicals; 618.92
• DOI: 10.1002/pbc.27381
• Languages: English
• ISSNs: 1545-5009

Abstract

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.