Clinical, pharmacokinetic and economic analysis of the first switch to an extended half-life factor IX (albutrepenonacog alfa, rFIX-FP) in Spain

Autores de IIS La Fe

• Juan Eduardo Megías Vericat

  Autor

• Santiago Bonanad Boix

  Autor

Participantes ajenos a IIS La Fe

• Rodríguez López M.
• Albo López C.

Grupos

• Farmacogenética

• Hemostasia, Trombosis, Arteriosclerosis y Biología vascular

Abstract

Extended half-life of factor IX (FIX) demonstrated clinical benefit and lower treatment burden than standard half-life FIX products in clinical trials. We analysed the impact in efficacy, pharmacokinetics (PKs) and costs of the switch from nonacog alfa (rFIX) to albutrepenonacog alfa (rFIX-FP) in the first patient with haemophilia B (HB) treated in Spain outside clinical trials. A 7-year-old boy presented with HB with poor venous access and repetition infections using rFIX, which was switched to rFIX-FP. Prophylaxis was adjusted by PKs using WAPPS-Hemo tailoring from 100 IU/kg/week of rFIX to 80 IU/kg/3 weeks of rFIX-FP. Comparing 6 months before, rFIX-FP reduced 68.5% FIX consumption/kg and 58.3% infusion frequency, but total costs/weight showed a slight increase. Ratio of half-life between rFIX and rFIX-FP was 3.4-3.7. This case report revealed that switch to rFIX-FP decreased frequency and FIX consumption, without adverse events and bleeds. Copyright 2020 BMJ Publishing Group. All rights reserved

Keywords

• Blood Coagulation Tests; Child; Drug Costs; Drug Substitution; Factor IX; Half-Life; Hemophilia B; Hemorrhage; Humans; Male; Recombinant Fusion Proteins; Serum Albumin; Severity of Illness Index; antibiotic agent; blood clotting factor 9; recombinant blood clotting factor 9; blood clotting factor 9; fusion protein; recombinant blood clotting factor 9; serum albumin; Article; bleeding; case report; catheter infection; child; clinical article; drug cost; drug half life; extended half life; F9 gene; granuloma; hemarthrosis; hemophilia B; human; male; muscle hematoma; nonsense mutation; priority journal; school child; Spain; Staphylococcus epidermidis; vascular access; bleeding; blood clotting test; complication; drug substitution; economics; half life time; hemophilia B; severity of illness index