ArticleBlood advances2023
Minimum factor VIII levels to prevent joint bleeding in mild hemophilia A.
Article in Blood advances, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 13 papers.
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Who cites it
13 citing papers in PubMed, 15 citations in OpenAlex.
- Article
- Focus on Women with Hemophilia and Carriers: How to Address All the Existing Gaps in Diagnosis and Care.Journal of clinical medicine · 2026Review
- Factor VIIIResearch and practice in thrombosis and haemostasis · 2026Article
- [Clinical efficacy and safety of Octocog alfa in Chinese patients with hemophilia A: One-year follow-up results from the Antihemophilic Factor Hemophilia A Outcome Database (AHEAD) study].Zhonghua xue ye xue za zhi = Zhonghua xueyexue zazhi · 2025Article
- Why is the uptake of gene therapy in hemophilia less than expected?Research and practice in thrombosis and haemostasis · 2025Article
- Current clinical applications of AAV-mediated gene therapy.Molecular therapy : the journal of the American Society of Gene Therapy · 2025Review
- Gene therapy for hemophilia - From basic science to first approvals of "one-and-done" therapies.Molecular therapy : the journal of the American Society of Gene Therapy · 2025Review
- Mild or moderate hemophilia is not always a mild or moderate bleeding disorder: Back to the clinical phenotype.HemaSphere · 2025Article
- Gene therapy in hemophilia: the dawn of a new era.Research and practice in thrombosis and haemostasis · 2025Review
- Real-World Evidence on Joint Condition in Non-Severe Hemophilia A Patients: A Multicenter Study.Journal of blood medicine · 2025Article
- Roctavian gene therapy for hemophilia A.Blood advances · 2024Review
- Pre-clinical evaluation of an enhanced-function factor VIII variant for durable hemophilia A gene therapy in male mice.Nature communications · 2024Article
- Gender equity in hemophilia: need for healthcare, familial, and societal advocacy.Frontiers in medicine · 2024Review
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Authors and funding
6 authors at 2 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
The severity of the bleeding phenotype in patients with hemophilia A (HA) broadly correlates with the degree of coagulation factor VIII (FVIII) deficiency in plasma. However, the FVIII level necessary to achieve the goal of zero joint bleeds remains unclear. This study aimed to identify the minimum FVIII level necessary to prevent joint bleeds in patients with HA. In this retrospective study, patients with congenital mild HA treated on demand, aged ≥16 years, with no history of FVIII inhibitors, followed at the Angelo Bianchi Bonomi Hemophilia and Thrombosis Center in Milan, were enrolled. We investigated 270 male patients with a median age of 45 years (16-88) and median lifelong FVIII of 21 IU/dL. One hundred patients (37%) had a lifelong history of at least 1 joint bleed. The mean annualized joint bleeding rate (AJBR) and spontaneous AJBR were 0.016 (standard deviation [SD], 0.032) and 0.001 (SD, 0.010), respectively. After adjusting for age, for each IU/dL increase in FVIII, there was a 6% reduction in AJBR and an 11% reduction in spontaneous AJBR. The minimum FVIII levels needed to prevent lifelong any joint bleeds and spontaneous joint bleeds resulted to be 19.2 IU/dL and 17.7 IU/dL, respectively. In this large cohort of persons with mild HA, we identified the minimum FVIII levels needed to prevent total and spontaneous joint bleeds (19.2 IU/dL and 17.7 IU/dL, respectively). These findings could suggest important implications for the accurate design of prophylactic therapies for persons with moderate and severe HA, including gene therapy.
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