Back to Search
Start Over
High adherence to prophylaxis regimens in haemophilia B patients receiving rIX-FP: Evidence from clinical trials and real-world practice
- Source :
- Haemophilia
- Publication Year :
- 2020
-
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.
- Subjects :
- Adult
medicine.medical_specialty
albutrepenonacog alfa
Adolescent
Recombinant Fusion Proteins
Hemorrhage
haemophilia B
030204 cardiovascular system & hematology
Haemophilia
Hemophilia B
Factor IX
03 medical and health sciences
Young Adult
0302 clinical medicine
Internal medicine
medicine
Humans
Haemophilia B
adherence
Dosing
Practice Patterns, Physicians'
Clinical Haemophilia
Child
Infusions, Intravenous
Genetics (clinical)
Serum Albumin
Aged
treatment
business.industry
rIX‐FP
Original Articles
Hematology
General Medicine
Bleed
Middle Aged
medicine.disease
Clinical trial
Treatment Adherence and Compliance
Regimen
Original Article
business
Dosing Frequency
030215 immunology
medicine.drug
Subjects
Details
- ISSN :
- 13652516
- Volume :
- 26
- Issue :
- 4
- Database :
- OpenAIRE
- Journal :
- Haemophilia : the official journal of the World Federation of Hemophilia
- Accession number :
- edsair.doi.dedup.....91323be54a182e26beef77f95d568c88