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

Pediatr Blood Cancer. 2018 Dec;65(12):e27381. doi: 10.1002/pbc.27381. Epub 2018 Sep 19.

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.

Keywords: FEIBA; hemophilia B; inhibitor; rFVIIa; thrombin generation.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adolescent
  • Blood Coagulation Factors / therapeutic use*
  • Blood Coagulation Tests / methods
  • Child, Preschool
  • Factor VIIa / therapeutic use*
  • Hemophilia B / drug therapy*
  • Humans
  • Male
  • Recombinant Proteins / therapeutic use
  • Thrombin / biosynthesis*
  • Thrombin / drug effects

Substances

  • Blood Coagulation Factors
  • Recombinant Proteins
  • recombinant FVIIa
  • anti-inhibitor coagulant complex
  • Factor VIIa
  • Thrombin

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