High adherence to prophylaxis regimens in haemophilia B patients receiving rIX‐FP: Evidence from clinical trials and real‐world practice Journal Articles uri icon

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  • AbstractIntroductionAdherence to prophylaxis regimens is essential for bleed prevention in haemophilia but remains a challenge due to the need for frequent infusions.AimTo evaluate patient adherence to prophylaxis regimens with a long‐acting recombinant factor IX (rIX‐FP; IDELVION®) in clinical studies and real‐world practice.MethodsIn two phase 3 clinical studies, patients with haemophilia B (FIX ≤2%) recorded their dose, dosing frequency and rIX‐FP consumption in an e‐diary. Adherence to prescribed prophylaxis regimens was assessed in all patients and to prescribed dose in patients ≥12 years only. Additionally, adherence to rIX‐FP prophylaxis regimens in real‐world practice was captured.ResultsIn clinical studies, 94.9% (n = 56/59) of patients ≥12 years and 100% (n = 27) of paediatric patients received ≥80% of the expected number of infusions for their assigned prophylaxis schedule. Overall, mean adherence rate was 95.5% across all prophylaxis regimens in patients ≥12 years and 97.9% with a 7‐day regimen in paediatric patients. In patients ≥12 years, 85.7% (n = 54/63) were dose adherent, defined as receiving within 10% of their prescribed dose ≥80% of the time. In real‐world practice, adherence was observed in 100% (n = 14 and n = 15, respectively) of patients in two haemophilia treatment centres and 57.1% (n = 4/7) of patients in a third centre; non‐adherence (n = 3/7) was linked to insurance‐related and parental issues.ConclusionIn clinical studies, patients with haemophilia B had high adherence rates to rIX‐FP prophylaxis regimens with a variety of dosing intervals, enabling them to achieve very low bleeding rates. High adherence may also be achievable in real‐world practice.


  • Mancuso, Maria Elisa
  • Oldenburg, Johannes
  • Boggio, Lisa
  • Kenet, Gili
  • Chan, Anthony
  • Altisent, Carmen
  • Seifert, Wilfried
  • Santagostino, Elena

publication date

  • July 2020