ReviewHematology. American Society of Hematology. Education Program2016
Hemophilia and inhibitors: current treatment options and potential new therapeutic approaches.
Review in Hematology. American Society of Hematology. Education Program, 2016. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT07190118 (Effects of Emicizumab Treatment on the Musculoskeletal System in Children With Hemophilia A), which is not on this map. Cited by 46 papers, 2 of them syntheses 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.
Effects of Emicizumab Treatment on the Musculoskeletal System in Children With Hemophilia A: A Prospective Study
Who cites it
46 citing papers in PubMed, 2 syntheses or guidelines pooled it, 105 citations in OpenAlex.
- Factor VIII replacement prophylaxis in patients with hemophilia A transitioning to adults: a systematic literature review.Orphanet journal of rare diseases · 2021Pooled it
- Emicizumab for the treatment of haemophilia A: a narrative review.Blood transfusion = Trasfusione del sangue · 2019Pooled it
- Trial
- Characterization of a factor VIII/immunoglobulin heavy chain μ double-knockout mouse model of hemophilia A for long-term exposure to factor VIII proteins.Research and practice in thrombosis and haemostasis · 2026Article
- The Immunology of Transfusion Medicine: Past, Present, and Future.Methods in molecular biology (Clifton, N.J.) · 2026Review
- Factor X and combined factor VIIa/factor X augment coagulation potential in a plasma model of antithrombin-reduced hemophilia.Research and practice in thrombosis and haemostasis · 2025Article
- 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
- Factor VIII Antibodies Demonstrate Type I or Type II Kinetics in Acquired Haemophilia A.Haemophilia : the official journal of the World Federation of Hemophilia · 2025Article
- Bio-hybrid scaffolds combining polyvinyl alcohol and decellularized articular cartilage for the treatment of focal chondral lesions in hemophilic patients.Frontiers in pharmacology · 2025Article
- Case Report: Severe hemophilia B patient with inhibitor and anaphylaxis reaction to FIX, successfully managed with concizumab prophylaxis therapy.Frontiers in pediatrics · 2025Article
- Advances in Development of Drug Treatment for Hemophilia with Inhibitors.ACS pharmacology & translational science · 2024Review
- Prediction of the chance of successful immune tolerance induction in persons with severe hemophilia A and inhibitors: a clinical prediction model.Research and practice in thrombosis and haemostasis · 2024Article
- Review
- Anesthetic considerations for joint replacement surgery in hemophilic arthropathy: a comprehensive review.Anesthesia and pain medicine · 2024Review
- In vivo LNP-CRISPR Approaches for the Treatment of Hemophilia.Molecular diagnosis & therapy · 2024Review
- Real-world use of recombinant porcine sequence factor VIII in the treatment of acquired hemophilia A: EU PASS.Therapeutic advances in hematology · 2024Article
- Site-directed mutagenesis of tissue factor pathway inhibitor-binding exosite D60A on factor VII results in a new factor VII variant with lower coagulant activity.Research and practice in thrombosis and haemostasis · 2024Article
- Emicizumab as a Promising Form of Therapy for Type A Hemophilia - A Review of Current Knowledge from Clinical Trials.Current protein & peptide science · 2024Review
- Emicizumab prophylaxis for people with hemophilia A: Waste estimation and the Brazilian perspective.Saudi pharmaceutical journal : SPJ : the official publication of the Saudi Pharmaceutical Society · 2023Article
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 2 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
The immune response to infused factor concentrates remains a major source of morbidity and mortality in the treatment of patients with hemophilia A and B. This review focuses on current treatment options and novel therapies currently in clinical trials. After a brief review of immune tolerance regimens, the focus of the discussion is on preventing bleeding in patients with hemophilia and inhibitors. Recombinant factor VIIa and activated prothrombin complex concentrates are the mainstays in treating bleeds in patients with inhibitors. Both agents have been shown to reduce bleeding episodes to a similar degree when infused prophylactically; however, individual patients may respond better to one agent over the other at any given time. The international immune tolerance trial revealed that a high-dose factor VIII regimen provided significantly better bleeding protection than the low-dose regimen. Given the high cost of treatment and the potential for a high-dose immune tolerance regimen to prevent bleeding in some patients, we discuss how we treat patients to maximize the prevention of bleeds while minimizing cost. Novel approaches to treatment of these patients are in development. These include agents that mimic factor VIII or augment thrombin generation by bypassing the inhibitor, as well as agents that inhibit the natural anticoagulants.
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.