Trial reportHaemophilia : the official journal of the World Federation of Hemophilia2020
High adherence to prophylaxis regimens in haemophilia B patients receiving rIX-FP: Evidence from clinical trials and real-world practice.
Trial report in Haemophilia : the official journal of the World Federation of Hemophilia, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 11 papers.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
11 citing papers in PubMed.
- High adherence to prophylaxis regimens in haemophilia B patients receiving rIX-FP: Evidence from clinical trials and real-world practice.Haemophilia : the official journal of the World Federation of Hemophilia · 2020Trial
- Integrating Clinical, Functional, and Patient-Reported Outcomes in Haemophilia Care: A Delphi-Based Consensus on a New Monitoring Tool.Journal of clinical medicine · 2026Article
- Real-world experience of rIX-FP prophylaxis at dosing intervals of up to 14 days in a pediatric patient with hemophilia B during the COVID-19 pandemic.Clinical case reports · 2023Article
- IDEAL study: A real-world assessment of pattern of use and clinical outcomes with recombinant coagulation factor IX albumin fusion protein (rIX-FP) in patients with haemophilia B in Italy.Haemophilia : the official journal of the World Federation of Hemophilia · 2023Observational
- Switching from Sucrose-Formulated rFVIII to Octocog Alfa (BAY 81-8973) Prophylaxis Improves Bleed Outcomes in the LEOPOLD Clinical Trials.Journal of blood medicine · 2023Article
- Long-term prophylaxis: what are our options and how to define success?Hematology. American Society of Hematology. Education Program · 2022Review
- IDELVION: A Comprehensive Review of Clinical Trial and Real-World Data.Journal of clinical medicine · 2022Review
- Investigation of the Bleeding Tendency in Sudanese Female Carriers of Hemophilia B.BioMed research international · 2022Article
- Albumin-Fusion Recombinant FIX in the Management of People with Hemophilia B: An Evidence-Based Review.Drug design, development and therapy · 2022Review
- How do we optimally utilize factor concentrates in persons with hemophilia?Hematology. American Society of Hematology. Education Program · 2021Review
- HAEMFIX: Impact of Switching From SHL-FIX to EHL-FIX in Patients With Haemophilia B.Haemophilia : the official journal of the World Federation of HemophiliaArticle
Corrections and comments
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Authors and funding
8 authors.
Funding
Abstract
introductionAdherence 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
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.
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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.