ArticleBlood coagulation & fibrinolysis : an international journal in haemostasis and thrombosis2020
Real-world outcomes associated with standard half-life and extended half-life factor replacement products for treatment of haemophilia A and B.
Article in Blood coagulation & fibrinolysis : an international journal in haemostasis and thrombosis, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 17 papers, 1 of them a synthesis that pooled it.
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Who cites it
17 citing papers in PubMed, 1 synthesis or guideline pooled it, 41 citations in OpenAlex.
- Systematic Literature Review of Outcomes Associated With Adherence to Haemophilia Drug Therapy.Haemophilia : the official journal of the World Federation of Hemophilia · 2025Pooled it
- Laboratory Challenges in the Era of Novel Haemophilia Therapies.Transfusion medicine and hemotherapy : offizielles Organ der Deutschen Gesellschaft fur Transfusionsmedizin und Immunhamatologie · 2026Review
- Integrating Clinical, Functional, and Patient-Reported Outcomes in Haemophilia Care: A Delphi-Based Consensus on a New Monitoring Tool.Journal of clinical medicine · 2026Article
- A precision gene-engineered B cell medicine producing sustained levels of active factor IX for hemophilia B therapy.Molecular therapy : the journal of the American Society of Gene Therapy · 2026Article
- Public health assistance for people with haemophilia in Brazil (PATCH study): a cross-sectional study protocol.BMJ open · 2025Article
- Switching From Standard to Extended Half-Life Coagulation Factor Replacement in Haemophilia: Clinical Outcomes and Costs of Care in Finland.Haemophilia : the official journal of the World Federation of Hemophilia · 2025Article
- Assessment of Clinical Characteristics and Sociocontextual Factors on Medication Adherence in Children and Adolescents With Hemophilia.Pediatric blood & cancer · 2025Article
- Human plasma cells engineered to secrete bispecifics drive effective in vivo leukemia killing.Molecular therapy : the journal of the American Society of Gene Therapy · 2024Article
- Cost-Effectiveness of Recombinant Factor IX Fc Prophylaxis and Recombinant Factor IX On-Demand Treatment in Patients with Haemophilia B Without Inhibitors.Advances in therapy · 2024Article
- Cost-Effectiveness Analysis of Etranacogene Dezaparvovec Versus Extended Half-Life Prophylaxis for Moderate-to-Severe Haemophilia B in Germany.PharmacoEconomics - open · 2024Review
- Estimating the impact of improved management of haemophilia a on clinical outcomes and healthcare utilisation and costs.BMC research notes · 2023Article
- A Review of Factor VIII and Factor IX Assay Methods for Monitoring Extended Half-Life Products in Hemophilia A and B.Methods in molecular biology (Clifton, N.J.) · 2023Review
- Comparison of Real-World Dose and Consumption for Two Extended Half-Life Recombinant Factor VIII Products for the Treatment of Hemophilia A in the United States.Journal of blood medicine · 2022Article
- Clinical, humanistic, and economic burden of severe hemophilia B in the United States: Results from the CHESS US and CHESS US+ population surveys.Orphanet journal of rare diseases · 2021Article
- The Clinical Genetics of Hemophilia B (Factor IX Deficiency).The application of clinical genetics · 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
- Treatment of Haemophilia A Without Inhibitors: Real-World Treatment Patterns and Clinical Outcomes in the US.Haemophilia : the official journal of the World Federation of HemophiliaObservational
Corrections and comments
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Authors and funding
10 authors at 3 institutions in 2 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
: Standard-of-care treatment for haemophilia A or B is to maintain adequate coagulation factor levels through clotting factor administration. The current study aimed to evaluate annualised bleeding rates (ABR) and treatment adherence for haemophilia A or B patients receiving standard half-life (SHL) vs. extended half-life (EHL) factor replacement products. We analysed data from the Adelphi Disease-Specific Programmes, a health record-based survey of United States and European haematologists. Analysis included 651 males with moderate-to-severe haemophilia A or B (the United States, n = 132; Europe, n = 519). The haemophilia A analysis included 501 patients (SHL, n = 435; EHL, n = 66). In the combined United States/European population, mean (SD) ABR was 1.7 (1.69) for the SHL group and 1.8 (2.00) for the EHL group. A total of 72% of patients receiving SHL factor VIII and 75% of patients receiving EHL factor VIII in the combined population were fully adherent (no doses missed of the last 10 doses), as reported by physicians. The haemophilia B analysis included 150 patients (SHL, n = 114; EHL, n = 36). The mean (SD) ABR in the combined population was 2.1 (2.16) for patients receiving SHL factor IX (FIX) and 1.4 (1.48) for patients receiving EHL FIX. The percentage of fully adherent patients (physician-reported) was similar in both treatment groups (SHL FIX, 68%; EHL FIX, 73%). In this preliminary real-world survey in a relatively small sample of patients, measures of ABR and adherence between SHL and EHL products were evaluated. Additional real-world research on prescribing patterns, SHL vs. EHL effectiveness, and adherence is warranted.
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