ReviewF1000Research2018
Emerging therapies for hemophilia: controversies and unanswered questions.
Review in F1000Research, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 25 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
25 citing papers in PubMed, 1 synthesis or guideline pooled it.
- Clotting factor concentrates for preventing bleeding and bleeding-related complications in previously untreated or minimally treated children with hemophilia A or B.The Cochrane database of systematic reviews · 2025Pooled it
- Long-term safety and efficacy of fitusiran prophylaxis, and perioperative management, in people with hemophilia A or B.Blood advances · 2025Trial
- Befovacimab, an anti-tissue factor pathway inhibitor antibody: Early termination of the multiple-dose, dose-escalating Phase 2 study due to thrombosis.Haemophilia : the official journal of the World Federation of Hemophilia · 2022Trial
- Trial
- A Psychometric Analysis of the Haemophilia Quality of Life Questionnaire for Adults Physical Health Subscale and Total Scores: Data From the Phase 3 XTEND-1 Trial in Patients With Severe Haemophilia A.Haemophilia : the official journal of the World Federation of HemophiliaTrial
- Clinical immunogenicity in rAAV gene therapy: Insights and implications.Molecular therapy. Advances · 2026Review
- Long-Term Dystrophin Replacement Therapy in Duchenne Muscular Dystrophy Causes Cardiac Inflammation.JACC. Basic to translational science · 2025Article
- Exploring nonreplacement therapies' impact on hemophilia and other rare bleeding disorders.Research and practice in thrombosis and haemostasis · 2024Article
- Stable and durable factor IX levels in patients with hemophilia B over 3 years after etranacogene dezaparvovec gene therapy.Blood advances · 2023Article
- Antithrombin lowering in hemophilia: a closer look at fitusiran.Research and practice in thrombosis and haemostasis · 2023Review
- Review
- Understanding the evolution of coverage policies for prophylaxis treatments of hemophilia A without inhibitors: a payer Delphi panel.Journal of managed care & specialty pharmacy · 2021Article
- Blood coagulation factor X: molecular biology, inherited disease, and engineered therapeutics.Journal of thrombosis and thrombolysis · 2021Review
- Musculoskeletal ultrasound in hemophilia: Results and recommendations from a global survey and consensus meeting.Research and practice in thrombosis and haemostasis · 2021Article
- The Role of Physiotherapy in the New Treatment Landscape for Haemophilia.Journal of clinical medicine · 2021Review
- Timing of Intensive Immunosuppression Impacts Risk of Transgene Antibodies after AAV Gene Therapy in Nonhuman Primates.Molecular therapy. Methods & clinical development · 2020Article
- B Cell Depletion Eliminates FVIII Memory B Cells and Enhances AAV8-coF8 Immune Tolerance Induction When Combined With Rapamycin.Frontiers in immunology · 2020Article
- Safety and Efficacy of Turoctocog Alfa in the Prevention and Treatment of Bleeding Episodes in Previously Treated Patients from China with Severe Hemophilia A: Results from the Guardian 7 Trial.Therapeutics and clinical risk management · 2020Article
- Gene Therapy for Hemophilia: Facts and Quandaries in the 21st Century.Mediterranean journal of hematology and infectious diseases · 2020Review
- Advances of adeno-associated virus applied in gene therapy to hemophilia from bench work to the clinical use.Blood science (Baltimore, Md.) · 2019Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
3 authors.
Funding
Abstract
Several new therapies for hemophilia have emerged in recent years. These strategies range from extended half-life factor replacement products and non-factor options with improved pharmacokinetic profiles to gene therapy aiming for phenotypic cure. While these products have the potential to change hemophilia care dramatically, several challenges and questions remain regarding broader applicability, long-term safety, and which option to pursue for each patient. Here, we review these emerging therapies with a focus on controversies and unanswered questions in each category.
Indexed as
Identifiers
What OpenQuestion holds
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.