Trial reportAnnals of hematology2020
Treatment patterns and bleeding outcomes in persons with severe hemophilia A and B in a real-world setting.
Trial report in Annals of hematology, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 18 papers, 1 of them a synthesis that pooled it.
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
18 citing papers in PubMed, 1 synthesis or guideline pooled it, 34 citations in OpenAlex.
- Pharmacokinetics and Associated Efficacy of Emicizumab in Humans: A Systematic Review.Clinical pharmacokinetics · 2021Pooled it
- A New Risk-Based Scoring Approach to Individualize Prophylaxis in Patients with Hemophilia A: Results from the PREDICT Study.Advances in therapy · 2026Trial
- Enhancing Hemophilia A Care Through Home-Based Prophylaxis: Real-World Outcomes of a National Patient Support Program in Mexico.Journal of clinical medicine · 2026Article
- Health care resources and costs associated with delivering gene therapy for hemophilia in clinical practice.Research and practice in thrombosis and haemostasis · 2026Article
- Hemophilia is associated with accelerated biological aging.Haematologica · 2025Article
- The association of factor VIII activity levels with bleeding and quality of life in haemophilia a: findings from the European CHESS II study.Orphanet journal of rare diseases · 2025Article
- Clinical data on treatment regimen and use of medication among patients with hemophilia B in Korea.Blood research · 2024Article
- Review
- Bleeding pattern and consumption of factor VIII concentrate in adult patients with haemophilia A without inhibitors in the Czech Republic between 2013 and 2021 (Czech National Haemophilia Programme registry data).Annals of hematology · 2023Article
- Article
- Article
- What have we learned about the patient's experience of von Willebrand disease? A focus on women.Hematology. American Society of Hematology. Education Program · 2022Review
- Clinical profile and demographic characteristics of moderate and severe hemophilia patients in a tertiary care hospital of Bangladesh.Orphanet journal of rare diseases · 2022Article
- Medical costs and hospital utilization for hemophilia A and B urban inpatients in China: a national cross-sectional study.BMC health services research · 2022Article
- Effectiveness of Prophylactic Coagulation Factor Replacement Therapy in Patients with Severe Hemophilia A in Taiwan - A Population-Based Study.Clinical epidemiology · 2022Article
- The lived experience of women with a bleeding disorder: A systematic review.Research and practice in thrombosis and haemostasis · 2022Review
- Real-World Rates of Bleeding, Factor VIII Use, and Quality of Life in Individuals with Severe Haemophilia A Receiving Prophylaxis in a Prospective, Noninterventional Study.Journal of clinical medicine · 2021Article
- Bleeding outcomes and factor utilization after switching to an extended half-life product for prophylaxis in haemophilia A in Austria.Scientific reports · 2021Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
5 authors at 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
The current standard of care treatment for severe hemophilia A and B (SHA and SHB) is the prophylactic intravenous replacement of coagulation factor VIII or IX (FVIII/FIX) to prevent spontaneous bleeding. Persons with hemophilia without prophylactic treatment receive therapy in case of bleeding, i.e., on demand. To assess treatment patterns, utilization of products, and bleeding outcomes in a real-world cohort of persons with SHA and SHB, defined as FVIII or FIX activity < 1%, data was retrospectively collected from hemophilia-specific patient diaries used for home treatment, medical records, and entries into the Austrian Hemophilia Registry from the year 2012 to 2017. Fifty-three male persons with SHA (n = 47) and SHB (n = 6) were included; 26 with SHA and 5 with SHB were on prophylaxis, 8 and 1 switched therapy regimen, and 13 and 0 received on-demand therapy. Persons on prophylaxis used a mean factor FVIII or FIX dose of 71.7 and 40.1 IU/kg/week. Median (IQR) annualized bleeding rates (ABR) in SHA were 28.0 (23.4-31.3) in the on-demand, 4.9 (1.6-13.5) in the prophylaxis group, and 3.0 (2.0-6.8) in the prophylactic group of SHB. Three persons with SHA had zero bleeds during the observation period. On-demand therapy and hepatitis B and C were associated with higher ABR but not age, weight, and HIV positivity. Bleeding rates and the proportion of on-demand therapy in persons with hemophilia were high in our real-world cohort. Further improvement is needed, which might be facilitated with the advent of factor products with extended half-life or non-factor therapies.
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