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

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.

• Authors: Manuel Rodríguez López, Juan Eduardo Megías Vericat, Carmen Albo López, Santiago Bonanad
• Journal: BMJ case reports (BMJ Case Rep) Vol. 13 Issue 10 (Oct 13 2020) ISSN: 1757-790X [Electronic] England
• PMID: 33051199 (Publication Type: Case Reports, Journal Article)
• Copyright: © BMJ Publishing Group Limited 2020. No commercial re-use. See rights and permissions. Published by BMJ.

Chemical References

• Recombinant Fusion Proteins
• Serum Albumin
• Factor IX
• albutrepenonacog alfa

Topics

• Blood Coagulation Tests
• Child
• Drug Costs
• Drug Substitution (economics)
• Factor IX (administration & dosage, economics, pharmacokinetics)
• Half-Life
• Hemophilia B (complications, diagnosis, drug therapy, economics)
• Hemorrhage (economics, etiology, prevention & control)
• Humans
• Male
• Recombinant Fusion Proteins (administration & dosage, economics, pharmacokinetics)
• Serum Albumin (administration & dosage, economics, pharmacokinetics)
• Severity of Illness Index