ReviewBlood transfusion = Trasfusione del sangue2018
Non-factor replacement therapy for haemophilia: a current update.
Review in Blood transfusion = Trasfusione del sangue, 2018. 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 34 papers, 4 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
34 citing papers in PubMed, 4 syntheses or guidelines pooled it, 63 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
- 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
- Evaluating the Safety and Efficacy of Concizumab in Hemophilia A/B Patients: A Systematic Review.Clinical and applied thrombosis/hemostasis : official journal of the International Academy of Clinical and Applied Thrombosis/HemostasisPooled it
- 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
- Optimising prophylaxis outcomes and costs in haemophilia patients switching to recombinant FVIII-Fc: a single-centre real-world experience.Blood transfusion = Trasfusione del sangue · 2020Trial
- Screening chemical libraries for the development of oral treatments for bleeding disorders.Blood vessels, thrombosis & hemostasis · 2026Article
- 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
- Case Report: Severe hemophilia B patient with inhibitor and anaphylaxis reaction to FIX, successfully managed with concizumab prophylaxis therapy.Frontiers in pediatrics · 2025Article
- Review
- Thrombotic Disease in Hemophilic Patients: Is This a Paradox in a State of Hypocoagulability?Diagnostics (Basel, Switzerland) · 2024Review
- Emicizumab as a Promising Form of Therapy for Type A Hemophilia - A Review of Current Knowledge from Clinical Trials.Current protein & peptide science · 2024Review
- Toward non-factor therapy in hemophilia: an antithrombin insensitive Gla-domainless factor Xa as tissue factor pathway inhibitor bait.Research and practice in thrombosis and haemostasis · 2023Article
- Antithrombin lowering in hemophilia: a closer look at fitusiran.Research and practice in thrombosis and haemostasis · 2023Review
- Thrombin generation and implications for hemophilia therapies: A narrative review.Research and practice in thrombosis and haemostasis · 2023Review
- How to translate and implement the current science of gene therapy into haemophilia care?Therapeutic advances in hematology · 2023Review
- Quantification of emicizumab by mass spectrometry in plasma of people with hemophilia A: A method validation study.Research and practice in thrombosis and haemostasis · 2022Article
- In vivo delivery of CRISPR-Cas9 using lipid nanoparticles enables antithrombin gene editing for sustainable hemophilia A and B therapy.Science advances · 2022Article
- International consensus recommendations on the management of people with haemophilia B.Therapeutic advances in hematology · 2022Review
- Zero incidence of factor VIII inhibitors and successful haemostatic response in previously factor VIII-treated patients with haemophilia A switching to turoctocog alfa in a noninterventional study.Haemophilia : the official journal of the World Federation of Hemophilia · 2022Article
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
One of the most challenging issues facing us in the treatment of haemophilia is the development of alloantibodies against infused factor VIII (FVIII) or factor IX (FIX). Inhibitors render factor replacement therapy ineffective, exposing patients to an unacceptably high risk of morbidity and mortality. Besides the well-known bypassing agents (i.e. activated prothrombin complex concentrate and recombinant activated factor VII) used to treat or prevent bleeding in haemophilia patients with inhibitors, there is growing interest in a new class of therapeutic agents which act by enhancing coagulation (i.e. emicizumab) or inhibiting anticoagulant pathways (i.e. fitusiran and concizumab). This review will focus on these innovative therapies, providing an update on their current stage of clinical development.
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.