ReviewTherapeutic advances in hematology2020
Pharmacokinetics in routine haemophilia clinical practice: rationale and modalities-a practical review.
Review in Therapeutic advances in hematology, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 21 papers.
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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.
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Who cites it
21 citing papers in PubMed, 25 citations in OpenAlex.
- Efficacy and safety in patients with haemophilia A switching to octocog alfa (BAY 81-8973): Final results of the global real-world study, TAURUS.European journal of haematology · 2023Trial
- Personalized prophylactic therapy optimization in hemophilia A using a hybrid PK-PD-TTE model and deep RL.Journal of pharmacokinetics and pharmacodynamics · 2026Article
- A revised classification of FVIII concentrates: rationale and novel metrics.Blood advances · 2026Article
- Integrating Clinical, Functional, and Patient-Reported Outcomes in Haemophilia Care: A Delphi-Based Consensus on a New Monitoring Tool.Journal of clinical medicine · 2026Article
- Final data from the German AHEAD study: effectiveness and safety of octocog alfa and rurioctocog alfa pegol for hemophilia A in a real-world setting.Therapeutic advances in hematology · 2026Article
- Haemophilia B: an illustrative review of current challenges and opportunities.Research and practice in thrombosis and haemostasis · 2025Article
- Relationship between Dose, Factor IX Activity Levels and Bleeding Probability for rIX-FP Prophylaxis in Hemophilia B: A Repeated Time-to-Event Analysis.Clinical pharmacology and therapeutics · 2025Article
- Evaluation of pharmacokinetics of intravenous protein C concentrate in protein C deficiency: implications for treatment initiation and maintenance.Research and practice in thrombosis and haemostasis · 2025Article
- Global patterns of hemophilia drug trials, hemophilia care, and health care measures.Research and practice in thrombosis and haemostasis · 2025Article
- Performance of instrumental activities of daily living in patients with haemophilic arthropathy. A cross-sectional cohort study.Haemophilia : the official journal of the World Federation of Hemophilia · 2024Article
- Pharmacokinetic model-based assessment of factor IX prophylaxis treatment regimens in severe hemophilia B.Scientific reports · 2024Article
- Comparison Pharmacokinetic Dosing Tools in Hemophilia A Children.Indian journal of hematology & blood transfusion : an official journal of Indian Society of Hematology and Blood Transfusion · 2024Article
- The Effects of Coronavirus Disease 2019 Pandemic on Patients with Hemophilia and Inherited Bleeding Disorders: Results from 2 Centers in Turkey.Turkish archives of pediatrics · 2023Article
- Area under the curve: Comparing the value of factor VIII replacement therapies in haemophilia A.Haemophilia : the official journal of the World Federation of Hemophilia · 2023Article
- Individualised prophylaxis based on personalised target trough FVIII level optimised clinical outcomes in paediatric patients with severe haemophilia A.Haemophilia : the official journal of the World Federation of Hemophilia · 2022Article
- Review
- First experience of a hemophilia monitoring platform: florio HAEMO.Research and practice in thrombosis and haemostasis · 2022Article
- The Impact of Pharmacokinetic-Guided Prophylaxis on Clinical Outcomes and Healthcare Resource Utilization in Hemophilia A Patients: Real-World Evidence from the CHESS II Study.Journal of blood medicine · 2022Article
- How do we optimally utilize factor concentrates in persons with hemophilia?Hematology. American Society of Hematology. Education Program · 2021Review
- Pharmacokinetic Variability in Anti-TFPI Therapies: Why Absolute Metrics Matter for Plasma Monitoring.Haemophilia : the official journal of the World Federation of HemophiliaArticle
Corrections and comments
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Authors and funding
2 authors at 2 institutions in 2 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Prophylactic therapy with exogenous clotting factor concentrates in haemophilia A and B aims to achieve levels of circulating FVIII or FIX that are adequate for the prevention or reduction of spontaneous joint bleeding. Historically, a minimum trough level of at least 1% of the normal levels of circulating clotting factor has been targeted using standardised protocols. However, clearance of clotting factor varies between products and patients, and other pharmacokinetic (PK) parameters such as the frequency and magnitude of peaks may be important for ensuring optimal coverage. Thus, it is increasingly recognised that an individualised, PK-based approach to prophylaxis is necessary to achieve optimal protection. This review focuses on the clinical implications of using PK-guided, individualised prophylaxis in haemophilia to improve patient outcomes and considers practical methods of establishing patients' PK parameters. The most useful PK parameters will depend on the aim of the specific treatment (e.g. preventing activity-related and traumatic bleeds or addressing subclinical bleeding). In clinical practice, lengthy and frequent post-infusion sampling for PK analysis is costly and a significant burden for patients. However, a Bayesian analysis allows for the estimation of different PK parameters (e.g. half-life, factor concentrations over time, etc.) with only a minimum number of samples (e.g. 4, 24 and 48 h for haemophilia A), by using the patient's data to adjust a relevant population PK value towards the actual value. Numerous tools are available to aid in the practical use of Bayesian PK-guided dosing in the clinic, including the Web-based Application for the Population Pharmacokinetic Service hosted by McMaster University, Canada. The PK data can be used to determine the appropriate prophylaxis regimen for the individual patient, which can be monitored by assessment of the trough level at each clinic visit. Collection of PK data and subsequent PK-guided dosing should become standard practice when determining treatment strategies for people with haemophilia.
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Registered trials
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