ArticleResearch and practice in thrombosis and haemostasis2022
Impact of novel hemophilia therapies around the world.
Article in Research and practice in thrombosis and haemostasis, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 28 papers, 3 of them syntheses 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.
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Who cites it
28 citing papers in PubMed, 3 syntheses or guidelines pooled it, 51 citations in OpenAlex.
- A Systematic Review of Modelling Approaches in Economic Evaluations of Treatments for Inherited Bleeding Disorders.Applied health economics and health policy · 2026Pooled it
- Systematic Literature Review of Outcomes Associated With Adherence to Haemophilia Drug Therapy.Haemophilia : the official journal of the World Federation of Hemophilia · 2025Pooled it
- 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
- Public health assistance for people with haemophilia in Brazil (PATCH study): a cross-sectional study protocol.BMJ open · 2025Article
- 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
- Emicizumab in Severe Hemophilia A: Clinical and Patient Determinants of Transition Within a Standardized Program.Journal of hematology · 2025Article
- Gene therapy as an innovative approach to the treatment of hemophilia B-a review.Journal of applied genetics · 2025Review
- Activated prothrombin complex concentrate in patients receiving emicizumab prophylaxis: from evidence to clinical practice.Research and practice in thrombosis and haemostasis · 2025Review
- Concizumab, a Non-Replacement Therapy for Persons with Hemophilia with Inhibitors.Journal of clinical medicine · 2025Review
- Clinical trials to gene therapy development and production in Brazil: a review.Lancet regional health. Americas · 2025Review
- Economic Burden of Haemophilia from a Societal Perspective: A Scoping Review.PharmacoEconomics - open · 2025Review
- MHC class II presentation of FVIII-AnnexinA5 fusion proteins internalized by antigen presenting cells.Frontiers in immunology · 2025Article
- Emerging Therapies in Hemophilia: Improving Equitable Access to Care.Journal of blood medicine · 2025Review
- What is a cure through gene therapy? An analysis and evaluation of the use of "cure".Medicine, health care, and philosophy · 2024Article
- Debates Surrounding the Use of Antithrombotic Therapy in Hemophilic Patients with Cardiovascular Disease: Best Strategies to Minimize Severe Bleeding Risk.International journal of molecular sciences · 2024Review
- Ethical aspects of hemophilia gene therapy: a qualitative interview study with stakeholders.Research and practice in thrombosis and haemostasis · 2023Article
- Mim8, a novel factor VIIIa mimetic bispecific antibody, shows favorable safety and pharmacokinetics in healthy adults.Research and practice in thrombosis and haemostasis · 2023Article
- Practices and challenges for hemophilia management under resource constraints in Thailand.Orphanet journal of rare diseases · 2023Article
- Bispecific antibodies mimicking factor VIII in hemophilia A: converting innovation to an essential medicine.Research and practice in thrombosis and haemostasis · 2023Review
Corrections and comments
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Authors and funding
2 authors at 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Hemophilia A and B are hereditary bleeding disorders, characterized by factor VIII or IX deficiencies, respectively. For many decades, prophylaxis with coagulation factor concentrates (replacement therapy) was the standard-of-care approach in hemophilia. Since the 1950s, when prophylaxis started, factor concentrates have been improved with virus inactivation and molecule modification to extend its half-life. The past years have brought an intense revolution in hemophilia care, with the development of nonfactor therapy and gene therapy. Emicizumab is the first and only nonreplacement agent to be licensed for prophylaxis in people with hemophilia A, and real-world data show similar efficacy and safety from the pivotal studies. Other nonreplacement agents and gene therapy have ongoing studies with promising results. Innovative approaches, like subcutaneous factor VIII and lipid nanoparticles, are in the preclinical phase. These novel agents, such as extended half-life concentrates and emicizumab, have been available in resource-constrained countries through the constant efforts of the World Federation of Haemophilia Humanitarian Aid Program. Despite the wide range of new approaches and therapies, the main challenge remains the same: to guarantee treatment for all. In this article, we discuss the evolution of hemophilia care, global access to hemophilia treatment, and the current and future strategies that are now under development. Finally, we summarize relevant new data on this topic presented at the ISTH 2021 virtual congress.
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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.