ReviewHaematologica2025
Rebalancing agents in hemophilia: knowns, unknowns, and uncertainties.
Review in Haematologica, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 9 papers.
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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
9 citing papers in PubMed.
- Personalized Treatment of Hemophilia: Matching Therapies to Patient Needs in a Rapidly Evolving Landscape.Drugs · 2026Review
- Non-Factor Therapies in Haemophilia: The Era of Factor VIII Mimetics and Targeted Rebalancing Agents.Transfusion medicine and hemotherapy : offizielles Organ der Deutschen Gesellschaft fur Transfusionsmedizin und Immunhamatologie · 2026Review
- Long-term outcomes with emicizumab prophylaxis for haemophilia A in China: A multicentre, large-cohort retrospective study.British journal of haematology · 2026Observational
- Laboratory Challenges in the Era of Novel Haemophilia Therapies.Transfusion medicine and hemotherapy : offizielles Organ der Deutschen Gesellschaft fur Transfusionsmedizin und Immunhamatologie · 2026Review
- Challenges in Balancing Hemostasis and Thrombosis in Therapy Tailoring for Hemophilia: A Narrative Review.International journal of molecular sciences · 2026Review
- A Case Report of Non-Neutralizing Acquired Factor V Inhibitor Mimicking Deficiency: Diagnostic Challenges and Therapeutic Implications.Case reports in hematology · 2026Article
- Transforming Hemophilia Treatment With Novel Rebalancing Agents: Clinical Studies and Practical Perspectives.Clinical and applied thrombosis/hemostasis : official journal of the International Academy of Clinical and Applied Thrombosis/HemostasisReview
- Forgotten Steps in the Coagulation Cascade.Haemophilia : the official journal of the World Federation of HemophiliaArticle
- Real-World Effectiveness and Safety of Concizumab Prophylaxis in Hemophilia: Results From a National French Cohort.Haemophilia : the official journal of the World Federation of HemophiliaObservational
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.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Treatment options for patients with hemophilia and other bleeding disorders have advanced dramatically over the last few years, not only with the availability of safer factor concentrates, but also with the introduction of factor VIII-mimicking agents. Until recently, there were still areas of hemophilia care that required attention and optimization, including the need for repeated venipuncture, often requiring a central venous access device, and the possible development of inhibitors that limit the efficacy of factor replacement, thereby increasing the complexity and burden of therapy. A new class of rebalancing agents aims to address these remaining issues by inhibiting various natural anticoagulants. Fitusiran is a small interfering RNA agent that reduces antithrombin synthesis in hepatocytes, favoring a procoagulant state. Other promising rebalancing agents are concizumab and marstacimab, which selectively bind to the K2 domain of the tissue factor pathway inhibitor, thus restoring thrombin generation. SerpinPC is a subcutaneous biological inhibitor that blocks the anticoagulant activated protein C pathway, while VGA039 is a monoclonal antibody that targets its cofactor protein S. Although the available clinical data are promising, several important challenges remain. These include the thrombotic risk of rebalancing agents, perioperative and bleeding management, availability in low-income countries, efficacy and factor VIII equivalence compared to existing treatments, ideal target populations, and potential application in other hemostatic disorders. The primary aim of this review is to summarize the best available evidence on these novel rebalancing agents, while highlighting the unknowns, and emphasizing the uncertainties that lie ahead.
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