ReviewResearch and practice in thrombosis and haemostasis2018
Performing and interpreting individual pharmacokinetic profiles in patients with Hemophilia A or B: Rationale and general considerations.
Review in Research and practice in thrombosis and haemostasis, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 25 papers, 1 of them a synthesis that pooled it.
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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
25 citing papers in PubMed, 1 synthesis or guideline pooled it.
- A Systematic Review of Modelling Approaches in Economic Evaluations of Treatments for Inherited Bleeding Disorders.Applied health economics and health policy · 2026Pooled it
- Does difference between label and actual potency of factor VIII concentrate affect pharmacokinetic-guided dosing of replacement therapy in haemophilia A?Haemophilia : the official journal of the World Federation of Hemophilia · 2022Trial
- Direct comparison of two extended half-life PEGylated recombinant FVIII products: a randomized, crossover pharmacokinetic study in patients with severe hemophilia A.Annals of hematology · 2020Trial
- 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
- Plasma emicizumab concentrations and bleeding rates in children and adults with severe hemophilia A.Research and practice in thrombosis and haemostasis · 2026Observational
- Quality-of-Life Assessment and Pharmacokinetic Study in Hemophilia A Patients Undergoing Prophylactic Treatment.Pharmacy (Basel, Switzerland) · 2025Article
- 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
- Clinical Predictors and Prediction Models for rFVIII-Fc Half Life in Real-World People with Severe Hemophilia A.Journal of clinical medicine · 2023Article
- Article
- Managing surgery in hemophilia with recombinant factor VIII Fc and factor IX Fc: Data on safety and effectiveness from phase 3 pivotal studies.Research and practice in thrombosis and haemostasis · 2022Article
- IDELVION: A Comprehensive Review of Clinical Trial and Real-World Data.Journal of clinical medicine · 2022Review
- Current Choices and Management of Treatment in Persons with Severe Hemophilia A without Inhibitors: A Mini-Delphi Consensus.Journal of clinical medicine · 2022Article
- First experience of a hemophilia monitoring platform: florio HAEMO.Research and practice in thrombosis and haemostasis · 2022Article
- Pharmacokinetics of perioperative FVIII in adult patients with haemophilia A: An external validation and development of an alternative population pharmacokinetic model.Haemophilia : the official journal of the World Federation of Hemophilia · 2021Article
- Mobile Health Technology for the Personalized Therapy of Hemophilia.Frontiers in medicine · 2021Review
- Relationship between factor VIII activity, bleeds and individual characteristics in severe hemophilia A patients.Haematologica · 2020Article
- Favorable Pharmacokinetic Characteristics of Extended-Half-Life Recombinant Factor VIII BAY 94-9027 Enable Robust Individual Profiling Using a Population Pharmacokinetic Approach.Clinical pharmacokinetics · 2020Article
- Comparing Factor Use and Bleed Rates in U.S. Hemophilia A Patients Receiving Prophylaxis with 3 Different Long-Acting Recombinant Factor VIII Products.Journal of managed care & specialty pharmacy · 2020Article
- Development and Validation of a Population-Pharmacokinetic Model for Rurioctacog Alfa Pegol (AdynovateClinical pharmacokinetics · 2020Article
- Pharmacokinetics in routine haemophilia clinical practice: rationale and modalities-a practical review.Therapeutic advances in hematology · 2020Review
Corrections and comments
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Authors and funding
19 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
objectivesIn a separate document, we have provided specific guidance on performing individual pharmacokinetic (PK) studies using limited samples in persons with hemophilia with the goal to optimize prophylaxis with clotting factor concentrates. This paper, intended for clinicians, aims to describe how to interpret and apply PK properties obtained in persons with hemophilia.
methodsThe members of the Working Party on population PK (PopPK) of the ISTH SSC Subcommittee on Factor VIII and IX and rare bleeding disorders, together with additional hemophilia and PK experts, completed a survey and ranking exercise whereby key areas of interest in the field were identified. The group had regular web conferences to refine the manuscript's scope and structure, taking into account comments from the external feedback to the earlier document.
resultsMany clinical decisions in hemophilia are based on some form of explicit or implicit PK assessment. Individual patient PK profiles can be analyzed through traditional or PopPK methods, with the latter providing the advantage of fewer samples needing to be collected on any prophylaxis regimen, and without the need the for a washout period. The most useful presentation of PK results for clinical decision making are a curve of the factor activity level over time, the time to achieve a certain activity level, or related parameters like half-life or exposure (AUC). Software platforms have been developed to deliver this information to clinicians at the point of care. Key characteristics of studies measuring average PK parameters were reviewed, outlining what makes a credible head-to-head comparison among different concentrates. Large data collections of PK and treatment outcomes currently ongoing will advance care in the future.
conclusionsTraditionally used to compare different concentrates, PK can support tailoring of hemophilia treatment by individual profiling, which is greatly simplified by adopting a PopPK/Bayesian method and limited sampling protocol.
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