ReviewTherapeutic advances in hematology2018
Practical aspects of extended half-life products for the treatment of haemophilia.
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 59 papers, 2 of them syntheses that pooled it.
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
59 citing papers in PubMed, 2 syntheses or guidelines pooled it, 112 citations in OpenAlex.
- Matching-Adjusted Indirect Comparison of Efficacy and Consumption of rVIII-SingleChain Versus Two Recombinant FVIII Products Used for Prophylactic Treatment of Adults/Adolescents with Severe Haemophilia A.Advances in therapy · 2021Pooled it
- Assessment Tools for Adherence to Prophylactic Haemophilia Treatment in Adult and Adolescent Patients: A Systematic Review.Haemophilia : the official journal of the World Federation of HemophiliaPooled it
- Confirmed long-term safety and efficacy of prophylactic treatment with BAY 94-9027 in severe haemophilia A: final results of the PROTECT VIII extension study.Haemophilia : the official journal of the World Federation of Hemophilia · 2021Trial
- 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
- Pharmacokinetics, immunogenicity, safety, and preliminary efficacy of subcutaneous turoctocog alfa pegol in previously treated patients with severe hemophilia A (alleviate 1).Journal of thrombosis and haemostasis : JTH · 2020Trial
- Factor IX Padua AAV gene therapy in adolescents with hemophilia B: a phase 1 trial.Nature medicine · 2026Article
- Enhancing Hemophilia Care: Real-World Outcomes Following Switching to Extended Half-Life Factor VIII in Greece-The TOOL Study.Life (Basel, Switzerland) · 2026Article
- Gene Therapy in Hemophilia: Clinical Advances, Immunological Challenges, and Emerging Therapeutic Perspectives.International journal of molecular sciences · 2026Review
- Nine areas with outstanding challenges for hemophilia B research.Therapeutic advances in hematology · 2026Review
- Switching From Standard to Extended Half-Life Coagulation Factor Replacement in Haemophilia: Clinical Outcomes and Costs of Care in Finland.Haemophilia : the official journal of the World Federation of Hemophilia · 2025Article
- Therapeutic advances in hemophilia: from molecular innovation to patient-centered global care.Frontiers in medicine · 2025Review
- Intracranial hemorrhage in an infant leads to the diagnosis and treatment of severe hemophilia B: a case report.Italian journal of pediatrics · 2024Article
- Recent Advances in Gene Therapy for Hemophilia: Projecting the Perspectives.Biomolecules · 2024Review
- Real-world usage and effectiveness of recombinant factor VIII/factor IX Fc in hemophilia A/B: final data from the 24-month, prospective, noninterventional PREVENT study in Germany.Research and practice in thrombosis and haemostasis · 2024Article
- [Treatment of haemophilia in Austria].Wiener klinische Wochenschrift · 2024Article
- Cost-Effectiveness of Recombinant Factor IX Fc Prophylaxis and Recombinant Factor IX On-Demand Treatment in Patients with Haemophilia B Without Inhibitors.Advances in therapy · 2024Article
- Six-Year, Real-World Use of Prophylaxis with Recombinant Factor IX-Albumin Fusion Protein (rIX-FP) in Persons with Hemophilia B: A Single-Center Retrospective-Prospective Study.Journal of clinical medicine · 2024Article
- Protein S antibody as an adjunct therapy for hemophilia B.Blood advances · 2024Article
- Hemophilia Healing with AAV: Navigating the Frontier of Gene Therapy.Current gene therapy · 2024Review
- Use of nonacog beta pegol during surgery in persons with hemophilia B: a case series.Research and practice in thrombosis and haemostasis · 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
9 authors at 9 institutions in 7 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Haemophilia A and haemophilia B are congenital X-linked bleeding disorders caused by deficiency of coagulation factor VIII (FVIII) and IX (FIX), respectively. The preferred treatment option for patients with haemophilia is replacement therapy. For patients with severe disease, prophylactic replacement of coagulation factor is the treatment of choice; this has been shown to reduce arthropathy significantly, reduce the frequency of bleeds and improve patients' quality of life. Prophylaxis with standard recombinant factor requires regular intravenous infusion at least two (FIX) to three (FVIII) times a week. Recombinant FVIII and FIX products with an extended half-life are in development, or have been recently licensed. With reported mean half-life extensions of 1.5-1.8 times that of standard products for FVIII and 3-5 times that of standard products for FIX, these products have the potential to address many of the unmet needs of patients currently treated with standard factor concentrates. For example, they may encourage patients to switch from on-demand treatment to prophylaxis and improve the quality of life of patients receiving prophylaxis. Indeed, extended half-life products have the potential to reduce the burden of frequent intravenous injections, reducing the need for central venous lines in children, promote adherence, improve outcomes, potentially allow for more active lifestyles and, depending on the dosing regimen, increase factor trough levels. Members of the Zürich Haemophilia Forum convened for their 19th meeting to discuss the practicalities of incorporating new treatments into the management of people with haemophilia. This review of extended half-life products considers their introduction in haemophilia treatment, including the appropriate dose and schedule of infusions, laboratory monitoring, patient selection, safety considerations, and the economic aspects of care.
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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.