ReviewJournal of clinical medicine2017
Treatment and Prevention of Bleeds in Haemophilia Patients with Inhibitors to Factor VIII/IX.
Review in Journal of clinical medicine, 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 25 papers.
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
25 citing papers in PubMed.
- Concizumab in hemophilia with inhibitors: longer-term efficacy and safety results from the phase 3 explorer7 study.Blood advances · 2026Trial
- Impact of Inhibitor Development on the Cost Effectiveness of Prophylactic Treatment with Recombinant Factor VIII in Previously Untreated Patients with Severe Hemophilia A.PharmacoEconomics - open · 2026Article
- Association of Patient-Reported Outcomes with Hemophilia A Inhibitor Status and Treatment Product Type.Journal of clinical medicine · 2026Article
- Predicting Inhibitor Development in Hemophilia 'A' using Machine Learning: A Comprehensive Approach to Data Preprocessing, Balancing, and Biomarker Identification Using AI on the CHAMP Dataset.Current pharmaceutical biotechnology · 2025Article
- Concizumab prophylaxis in persons with hemophilia A or B with inhibitors: patient-reported outcome results from the phase 3 explorer7 study.Research and practice in thrombosis and haemostasis · 2024Article
- Platelet-targeted gene therapy induces immune tolerance in hemophilia and beyond.Journal of thrombosis and haemostasis : JTH · 2024Review
- Intraosseous delivery of platelet-targeted factor VIII lentiviral vector in humanized NBSGW mice.Blood advances · 2022Article
- Immune Tolerance Induction (ITI) with a pdFVIII/VWF Concentrate (octanate) in 100 Patients in the Observational ITI (ObsITI) Study.TH open : companion journal to thrombosis and haemostasis · 2022Article
- 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
- Current therapeutic approaches in the management of hemophilia-a consensus view by the Romanian Society of Hematology.Annals of translational medicine · 2021Review
- Article
- Design of an international investigator-initiated study on MOdern Treatment of Inhibitor-positiVe pATiEnts with haemophilia A (MOTIVATE).Therapeutic advances in hematology · 2021Article
- Origins, Development, Current Challenges and Future Directions with Activated Prothrombin Complex Concentrate for the Treatment of Patients with Congenital Haemophilia with Inhibitors.Hamostaseologie · 2020Article
- Enhancing therapeutic efficacy of in vivo platelet-targeted gene therapy in hemophilia A mice.Blood advances · 2020Article
- Article
- Implantable venous access devices in children with severe hemophilia: a tertiary pediatric institutional experience.Pediatric radiology · 2020Article
- Hemophilia therapy: the future has begun.Haematologica · 2020Review
- Editorial: Tolerating Factor VIII: Novel Strategies to Prevent and Reverse Neutralizing Anti-FVIII Antibodies.Frontiers in immunology · 2020Article
- Factor VIII replacement is still the standard of care in haemophilia A.Blood transfusion = Trasfusione del sangue · 2019Review
- Emicizumab: A Review in Haemophilia A.Drugs · 2019Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
3 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
The development of alloantibodies neutralising therapeutically administered factor (F) VIII/IX (inhibitors) is currently the most severe complication of the treatment of haemophilia. When persistent and at a high titre, inhibitors preclude the standard replacement treatment with FVIII/FIX concentrates, making patients' management challenging. Indeed, the efficacy of bypassing agents, i.e., activated prothrombin complex concentrates (aPCC) and recombinant activated factor VII (rFVIIa), needed to overcome the haemostatic interference of the inhibitor, is not comparable to that of factor concentrates. In addition, the therapeutical response is unpredictable, with a relevant inter-individual and even intra-individual variability, and no laboratory assay is validated to monitor the efficacy and safety of the treatment. As a result, inhibitor patients have a worse joint status and quality of life compared to inhibitor-free subjects and the eradication of the inhibitor by immune tolerance induction is the preeminent therapeutic goal, particularly in children. However, over the last decades, treatment with bypassing agents has been optimised, allowing home treatment and the individualisation of regimens aimed at improving clinical outcomes. In this respect, a growing body of evidence supports the efficacy of prophylaxis with both bypassing agents in reducing bleeding rates and improving the quality of life, although the impact on long-term outcomes (in particular on preventing/reducing joint deterioration) is still unknown. This review offers an update on the current knowledge and practice of the use of bypassing agents in haemophiliacs with inhibitors, as well as on debated issues and unmet needs in this challenging setting.
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