ReviewTherapeutic advances in hematology2018
Novel therapies and current clinical progress in hemophilia A.
Review in Therapeutic advances in hematology, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 32 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.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
32 citing papers in PubMed, 2 syntheses or guidelines pooled it, 57 citations in OpenAlex.
- Efanesoctocog Alfa Versus Emicizumab in Adolescent and Adult Patients With Haemophilia A Without Inhibitors.Advances in therapy · 2025Pooled it
- Efanesoctocog Alfa versus Standard and Extended Half-Life Factor VIII Prophylaxis in Adolescent and Adult Patients with Haemophilia A without Inhibitors.Advances in therapy · 2025Pooled it
- Trial
- Long-term safety and efficacy of the anti-tissue factor pathway inhibitor marstacimab in participants with severe haemophilia: Phase II study results.British journal of haematology · 2023Trial
- Social vulnerability, access to care, and outcomes in hemophilia.Blood vessels, thrombosis & hemostasis · 2026Article
- Evolving landscape of haemophilia care in India: Balancing innovation, access, and sustainability.The Indian journal of medical research · 2026Article
- Rebalancing Hemostasis: Fitusiran as a First-in-Class RNAi Therapy in Hemophilia A and B.Health science reports · 2026Article
- Oral health and quality of life in children with blood coagulation disorders and hemoglobinopathies. A cross-sectional study.Journal of family medicine and primary care · 2025Article
- Real-World Amount of Clotting Factor Products and Non-Factor Products Dispensed and Annual Medical Expenditures for Japanese Patients with Haemophilia A.Drugs - real world outcomes · 2024Article
- The impact of bleeding event frequency on health-related quality of life and work productivity outcomes in a European cohort of adults with haemophilia A: insights from the CHESS II study.Orphanet journal of rare diseases · 2023Article
- Psychometrics of the patient-reported outcomes measurement information system measures in hemophilia: the applicability of the pediatric item banks.Research and practice in thrombosis and haemostasis · 2023Article
- In Vitro FVIII-Encoding Transgenic Mesenchymal Stem Cells Maintain Successful Coagulation in FVIII-Deficient Plasma Mimicking Hemophilia ATurkish journal of haematology : official journal of Turkish Society of Haematology · 2023Article
- Article
- Novel Gene-Correction-Based Therapeutic Modalities for Monogenic Liver Disorders.Bioengineering (Basel, Switzerland) · 2022Review
- A qualitative study on the experiences of haemophilia carriers before, during and after pregnancy.Haemophilia : the official journal of the World Federation of Hemophilia · 2021Article
- Advances in the management of haemophilia: emerging treatments and their mechanisms.Journal of biomedical science · 2021Review
- Tailoring the effect of antithrombin-targeting therapy in haemophilia A using in silico thrombin generation.Scientific reports · 2021Article
- Continuous infusion with octocog alfa during cardiac surgery for tetralogy of Fallot in a haemophilic child.Blood transfusion = Trasfusione del sangue · 2021Article
- Patient-relevant health outcomes for hemophilia care: Development of an international standard outcomes set.Research and practice in thrombosis and haemostasis · 2021Article
- PROTECT VIII kids extension study: Long-term safety and efficacy of BAY 94-9027 (damoctocog alfa pegol) in children with severe haemophilia A.Haemophilia : the official journal of the World Federation of Hemophilia · 2021Article
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
The evolution of hemophilia treatment and care is a fascinating one but has been fraught with many challenges at every turn. Over the last 50 years or so patients with hemophilia and providers have witnessed great advances in the treatment of this disease. With these advances, there has been a dramatic decrease in the mortality and morbidity associated with hemophilia. Even with the remarkable advancements in treatment, however, new and old challenges continue to plague the hemophilia community. The cost of factor replacement and the frequency of infusions, especially in patients with severe hemophilia on prophylaxis, remains a significant challenge for this population. Other challenges include obtaining reliable venous access, especially in younger patients, and the development of neutralizing alloantibodies (inhibitors). The development of extended half-life products, a bispecific antibody which mimics the coagulation function of factor VIII (FVIII) and inhibition of anticoagulation proteins such as antithrombin with antibodies, aptamers or RNA interference technology have offered novel therapeutic approaches to overcome some of these existing challenges. Additionally, ongoing gene therapy research offers a way to possibly cure hemophilia. These novel treatment tools in conjunction with the establishment of an increasing number of comprehensive hemophilia centers and worldwide advocacy efforts have continued to push the progress of hemophilia care to new frontiers. This review highlights and summarizes these novel therapeutic approaches and the current clinical progress of hemophilia A.
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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.