ReviewJournal of biomedical science2021
Advances in the management of haemophilia: emerging treatments and their mechanisms.
Review in Journal of biomedical science, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 41 papers, 1 of them a synthesis that pooled it.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
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
41 citing papers in PubMed, 1 synthesis or guideline pooled it, 62 citations in OpenAlex.
- Assessment Tools for Adherence to Prophylactic Haemophilia Treatment in Adult and Adolescent Patients: A Systematic Review.Haemophilia : the official journal of the World Federation of HemophiliaPooled it
- Long-term safety and efficacy of fitusiran prophylaxis, and perioperative management, in people with hemophilia A or B.Blood advances · 2025Trial
- Experiences with Efanesoctocog Alfa: Exit Interviews with Caregivers of Previously Treated XTEND-Kids Phase III Trial Patients with Haemophilia A.The patient · 2026Article
- Laboratory Challenges in the Era of Novel Haemophilia Therapies.Transfusion medicine and hemotherapy : offizielles Organ der Deutschen Gesellschaft fur Transfusionsmedizin und Immunhamatologie · 2026Review
- Evolution of Hemophilia Management in Italy: Results from a Delphi Consensus Study.Journal of clinical medicine · 2026Article
- Social vulnerability, access to care, and outcomes in hemophilia.Blood vessels, thrombosis & hemostasis · 2026Article
- Integrating Clinical, Functional, and Patient-Reported Outcomes in Haemophilia Care: A Delphi-Based Consensus on a New Monitoring Tool.Journal of clinical medicine · 2026Article
- QFitlia (Fitusiran): redefining hemophilia treatment with RNAi therapy. A correspondence.Annals of medicine and surgery (2012) · 2026Article
- Article
- A Paradigm Shift in Hemophilia Care: The Promise of Gene Therapy.Current gene therapy · 2026Review
- Health care resources and costs associated with delivering gene therapy for hemophilia in clinical practice.Research and practice in thrombosis and haemostasis · 2026Article
- Haemophilia B: an illustrative review of current challenges and opportunities.Research and practice in thrombosis and haemostasis · 2025Article
- Emicizumab in Children with Severe Hemophilia A.Indian journal of pediatrics · 2025Observational
- Estimated Long-Term Durability of Valoctocogene Roxaparvovec Treatment in Male patients with Severe Hemophilia A: An Extrapolation of Clinical Data.Advances in therapy · 2025Article
- FRONTIER1 multiple ascending dose extension: a safety, tolerability, pharmacokinetics, and pharmacodynamics study of Mim8 in people with hemophilia A.Research and practice in thrombosis and haemostasis · 2025Article
- Real-World Unmet Needs of Patients With Haemophilia A and Haemophilia B With or Without Inhibitors: End-of-Study Results From the explorer6 Non-Interventional Study.Haemophilia : the official journal of the World Federation of Hemophilia · 2025Observational
- Gene therapy as an innovative approach to the treatment of hemophilia B-a review.Journal of applied genetics · 2025Review
- Article
- Artificial Intelligence in the Management of Hereditary and Acquired Hemophilia: From Genomics to Treatment Optimization.International journal of molecular sciences · 2025Review
- Thrombin Generation Assay to Support Hematologists in the Era of New Hemophilia Therapies.International journal of laboratory hematology · 2025Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
4 authors at 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Mainstay haemophilia treatment, namely intravenous factor replacement, poses several clinical challenges including frequent injections due to the short half-life of recombinant factors, intravenous administration (which is particularly challenging in those with difficult venous access), and the risk of inhibitor development. These impact negatively upon quality of life and treatment compliance, highlighting the need for improved therapies. Several novel pharmacological therapies developed for haemophilia aim to rebalance the clotting cascade and potentially circumvent the aforementioned challenges. These therapies utilise a range of different mechanisms, namely: the extension of the circulating half-life of standard recombinant factors; the mimicking of factor VIII cofactor activity; rebalancing of coagulation through targeting of natural anticoagulants such as antithrombin and tissue factor pathway inhibitor; and inducing the production of endogenous factors with gene therapy. These therapies carry the potential of revolutionising haemophilia treatment by alleviating the current challenges presented by mainstay factor replacement. This review will provide an overview of the key trial findings related to novel therapies based on the mechanisms described above.
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Registered trials
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.