ArticleJournal of clinical medicine2021
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.
Article in Journal of clinical medicine, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 13 papers.
What it found
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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.
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Who cites it
13 citing papers in PubMed.
- Prospective collection of bleeding rate and factor usage in participants with hemophilia A and B in a noninvestigational product study.Research and practice in thrombosis and haemostasis · 2026Trial
- Durability of efficacy, safety, and quality of life 5 years after valoctocogene roxaparvovec gene transfer for severe hemophilia A: final phase 3 GENEr8-1 trial results.Research and practice in thrombosis and haemostasis · 2026Trial
- Comparative Effectiveness of Valoctocogene Roxaparvovec and Prophylactic Factor VIII Replacement in Severe Hemophilia A.Advances in therapy · 2024Trial
- Factors contributing to the development of hemophilic arthropathy: A real-world study.Annals of hematology · 2026Article
- Prophylactic Weekly Efanesoctocog Alfa Versus Standard-Care Factor VIII in People Living With Severe Hemophilia A : A Cost-Effectiveness Analysis.Annals of internal medicine · 2025Article
- A Comparative Review on the Production of Factor VIII in Human and Non-human Hosts.Current pharmaceutical design · 2025Review
- Activated factor X delivered by adeno-associated virus significantly inhibited bleeding and alleviated hemophilic synovitis in hemophilic mice.Gene therapy · 2024Article
- Efficacy, safety, and quality of life 4 years after valoctocogene roxaparvovec gene transfer for severe hemophilia A in the phase 3 GENEr8-1 trial.Research and practice in thrombosis and haemostasis · 2024Article
- Outcomes and management of invasive procedures in participants with hemophilia A post gene therapy: a post hoc analysis of the GENEr8-1 phase III trial.Therapeutic advances in hematology · 2024Article
- Valoctocogene roxaparvovec gene transfer in participants with HIV.Blood advances · 2023Article
- The potential role of protease systems in hemophilic arthropathy.Blood advances · 2022Article
- Epidemiology, patient journey and unmet needs related to hemophilia in Brazil: a scoping review with evidence map.Hematology, transfusion and cell therapyArticle
- Preoperative diagnosis and management of inherited bleeding disorders in female adolescents and adults.Canadian journal of surgery. Journal canadien de chirurgieReview
Corrections and comments
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Authors and funding
18 authors.
Funding
Abstract
Regular prophylaxis with exogenous factor VIII (FVIII) is recommended for individuals with severe haemophilia A (HA), but standardised data are scarce. Here, we report real-world data from a global cohort. Participants were men ≥18 years old with severe HA (FVIII ≤ 1 IU/dL) receiving regular prophylaxis with FVIII. Participants provided 6 months of retrospective data and were prospectively followed for up to 12 months. Annualised bleeding rate (ABR) and FVIII utilisation and infusion rates were calculated. Differences between geographic regions were explored. Of 294 enrolled participants, 225 (76.5%) completed ≥6 months of prospective follow-up. Pre-baseline and on-study, the median (range) ABR values for treated bleeds were 2.00 (0-86.0) and 1.85 (0-37.8), respectively; the median (range) annualised FVIII utilisation rates were 3629.0 (1008.5-13541.7) and 3708.0 (1311.0-14633.4) IU/kg/year, respectively; and the median (range) annualised FVIII infusion rates were 120.0 (52.0-364.0) and 122.4 (38.0-363.8) infusions/year, respectively. The median (range) Haemo-QoL-A Total Score was 76.3 (9.4-100.0) (
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