ReviewHematology. American Society of Hematology. Education Program2021
Factor-mimetic and rebalancing therapies in hemophilia A and B: the end of factor concentrates?
Review in Hematology. American Society of Hematology. Education Program, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 16 papers, 1 of them a synthesis 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.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
16 citing papers in PubMed, 1 synthesis or guideline pooled it, 26 citations in OpenAlex.
- Non-clotting factor therapies for preventing bleeds in people with congenital hemophilia A or B.The Cochrane database of systematic reviews · 2024Pooled it
- Marstacimab prophylaxis in hemophilia A/B without inhibitors: results from the phase 3 BASIS trial.Blood · 2025Trial
- Long-term safety and efficacy of fitusiran prophylaxis, and perioperative management, in people with hemophilia A or B.Blood advances · 2025Trial
- Comprehensive care for hereditary angioedema: lessons learned from HAEmophilia Treatment Centers.Allergy, asthma, and clinical immunology : official journal of the Canadian Society of Allergy and Clinical Immunology · 2026Article
- Nine areas with outstanding challenges for hemophilia B research.Therapeutic advances in hematology · 2026Review
- Structure and function of therapeutic antibodies approved by the US FDA in 2024.Antibody therapeutics · 2025Review
- Thrombin Generation Assay to Support Hematologists in the Era of New Hemophilia Therapies.International journal of laboratory hematology · 2025Review
- Economic Burden of Haemophilia from a Societal Perspective: A Scoping Review.PharmacoEconomics - open · 2025Review
- Concizumab improves clot formation in hemophilia A under flow.Journal of thrombosis and haemostasis : JTH · 2024Article
- In vivo LNP-CRISPR Approaches for the Treatment of Hemophilia.Molecular diagnosis & therapy · 2024Review
- Article
- From a bispecific monoclonal antibody to gene therapy: A new era in the treatment of hemophilia A.Biomedical papers of the Medical Faculty of the University Palacky, Olomouc, Czechoslovakia · 2023Review
- Article
- Expert opinion on current and future prophylaxis therapies aimed at improving protection for people with hemophilia A.Journal of medicine and life · 2022Article
- Albumin-Fusion Recombinant FIX in the Management of People with Hemophilia B: An Evidence-Based Review.Drug design, development and therapy · 2022Review
- Preoperative diagnosis and management of inherited bleeding disorders in female adolescents and adults.Canadian journal of surgery. Journal canadien de chirurgieReview
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
2 authors at 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Hemophilia A (HA) and B are inherited bleeding disorders caused by a deficiency of factor VIII or factor IX, respectively. The current standard of care is the administration of recombinant or purified factor. However, this treatment strategy still results in a high economic and personal burden to patients, which is further exacerbated by the development of inhibitors-alloantibodies to factor. The treatment landscape is changing, with nonfactor therapeutics playing an increasing role in what we consider to be the standard of care. Emicizumab, a bispecific antibody that mimics the function of factor VIIIa, is the first such nonfactor therapy to gain US Food and Drug Administration approval and is rapidly changing the paradigm for HA treatment. Other therapies on the horizon seek to target anticoagulant proteins in the coagulation cascade, thus "rebalancing" a hemorrhagic tendency by introducing a thrombotic tendency. This intricate hemostatic balancing act promises great things for patients in need of more treatment options, but are these other therapies going to replace factor therapy? In light of the many challenges facing these therapies, should they be viewed as a replacement of our current standard of care? This review discusses the background, rationale, and potential of nonfactor therapies as well as the anticipated pitfalls and limitations. This is done in the context of a review of our current understanding of the many aspects of the coagulation system.
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.