A5008161447- Hemophilia Treatment and Research
- Chronic Myeloid Leukemia Treatments
- Antiplatelet Therapy and Cardiovascular Diseases
- Heparin-Induced Thrombocytopenia and Thrombosis
- COVID-19 Clinical Research Studies
- Virus-based gene therapy research
- CAR-T cell therapy research
- Coagulation, Bradykinin, Polyphosphates, and Angioedema
- Diabetes and associated disorders
- SARS-CoV-2 and COVID-19 Research
University College London
2021-2022
The Royal Free Hospital
2021-2022
Royal Free London NHS Foundation Trust
2021
Abstract Introduction Gene therapy for haemophilia is in late-stage clinical development and has the potential to become a therapeutic option practice. Aims To enhance understanding of perspectives people with around gene therapy, highlight their concerns about motivations having therapy. Method Structured, qualitative interviews were conducted recorded six who had received an investigational product. The recordings transcribed thematically analysed. Results Most those interviewed under age...
Abstract Introduction Cardiovascular events in patients with inherited bleeding disorders are challenging to manage. The risk of secondary antithrombotic treatment must be balanced against the thrombosis haemostatic therapy. Methods Patients coronary artery bypass grafting (CABG), percutaneous intervention (PCI) or atrial fibrillation (AF) from a single centre (2010–2018) included. Results A total 11 undergoing CABG ( n = 3), PCI 5) AF 3) and diagnosis haemophilia 8), B 1), factor XI...
Model-informed personalized prophylaxis with factor VIII (FVIII) replacement therapy aimed at higher trough levels is becoming indispensable for patients severe hemophilia A. This study to identify the most suitable population pharmacokinetic (PK) models using various FVIII products and 2 clinical assays implement one in open-access software.Twelve published PK were systematically compared predict time above target (TaT) a reference dosing occasion. External validation was performed 5-point...