High adherence to prophylaxis regimens in haemophilia B patients receiving rIX‐FP: Evidence from clinical trials and real‐world practice. Issue 4 (15th June 2020)

• Record Type: Journal Article
• Authors: Mancuso, Maria Elisa; Oldenburg, Johannes; Boggio, Lisa; Kenet, Gili; Chan, Anthony; Altisent, Carmen; Seifert, Wilfried; Santagostino, Elena
• Is Part Of: Haemophilia. Volume 26:Issue 4(2020)
• Page Start: 637
• Page End: 642
• Publication Date: 2020-06-15
• Subjects: adherence -- albutrepenonacog alfa -- factor IX -- haemophilia B -- rIX‐FP -- treatment; Hemophilia -- Periodicals; 616.1572005
• DOI: 10.1111/hae.14018
• Languages: English
• ISSNs: 1351-8216

Abstract

Abstract: Introduction: Adherence to prophylaxis regimens is essential for bleed prevention in haemophilia but remains a challenge due to the need for frequent infusions. Aim: To evaluate patient adherence to prophylaxis regimens with a long‐acting recombinant factor IX (rIX‐FP; IDELVION ® ) in clinical studies and real‐world practice. Methods: In 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. Results: In 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. Conclusion: In 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.