View clinical trials related to Hemophilia B.
Filter by:Performing an individual pharmacokinetic (PK) estimate is only the first step in implementing tailored prophylaxis, which requires using the PK profile information to design a personalized treatment regimen matching the treatment needs of individual patients. The overarching goal of WAPPS-Hemo is to provide an easy-to-use web application supporting all the steps needed to accomplish tailoring care of individual patients by matching their unique characteristics to the most appropriate treatment regimen, realizing the promise of personalized medicine. This study will assess the impact of adopting population PK (popPK) based tailored prophylaxis in clinical practice, including proportion of patients eligible for tailoring, and encountered barriers. The impact on patient important outcomes and on societal outcomes, particularly financial impact, vs. current standardized regimens will be measured. It is hypothesized that WAPPS-Hemo, via estimation of precise individual PK profiles and by supporting the simulation of treatment regimens will: 1. improve or maintain patient important outcomes, while reducing wastage of factor concentrates; and 2. establish best practices and effective knowledge translation strategies for the implementation of personalized medicine. Additionally, a solid base of data will be generated to model the bleeding risk of severe hemophilia A/B patients undergoing tailored prophylaxis which will enable evaluation of a combination of patient and treatment characteristics predictive of individual bleeding risk.
The WBDR is an international observational disease registry of patients with hemophilia. It will provide a platform for a network of hemophilia treatment centres (HTCs) around the world to collect uniform and standardized patient data and guide clinical practice. With informed consent from the patient, the WBDR stores anonymous data about the person's disease, such as hemophilia type and severity, symptoms, and treatment.
Rationale: Haemophilia is a rare disease; to improve knowledge international collaboration is needed. Well-defined clinical data will be collected from complete cohorts in order to prevent selection bias. Objective: To collect data on bleeding during neonatal period, endogenous (genetic) and exogenous (treatment-related) determinants of inhibitor development and long term outcome.
Severe haemophilia A and B (SHA, SHB) are inherited bleeding disorders affecting male patients and are characterised by low levels of circulating clotting factors VIII and IX respectively. Clinically low levels present with multiple recurrent bleeds into joints and muscle from the first couple of years of life. In addition patients may present with spontaneous and potentially fatal bleeding into any organ. The mainstay of treatment is replacement with the missing factor in the form of intravenous injections of factor VIII and IX. Clotting factors can be given to treat a bleed or can be given to prevent a bleed, and the latter is termed prophylaxis. Regular prophylaxis is the current standard of care and aims to decrease spontaneous bleeding events and resulting joint damage, and this requires patients to self-infuse factor into their veins two to four times week. Patient's compliance with prescribed regimen and recommendations has a significant influence on outcomes. Advances in biomolecular and protein engineering have extended the duration of the effect of clotting factor VIII and IX through multiple mechanisms. This extension of the duration of the effect presents the clinician and patients with opportunities to tailor the treatment to their particular needs, circumstances and body other characteristics. It has been suggested that decreasing the frequency of infusions will improve adherence and thus contribute to improved outcomes. In rare disorders, it is an accepted fact that post-marketing studies are crucial to understand the generalisability of the efficacy and safety outcomes and identify any new safety and efficacy concerns in relation to specific population group. The investigators propose the development of a registry for systematic collection of information with the dual aim of analysing the relationship between patient and treatment characteristics, and outcomes, and simultaneously identify areas for practice development that can improve the overall quality of life experienced by the haemophilia patient community.
The German Pediatric Hemophilia Research Database will collect data on the prophylactic and therapeutic use of factor concentrates, complications, outcome measures (joint scores, QoL) and living circumstances in newly diagnosed children with hemophilia.
Patients with hemophilia who have the same level of deficient factor(s) may express different severity of clinical presentation and bleeding tendency. Therefore a test which could determine overall hemostasis rather than simple concentration of a single deficient factor may correlate better with clinical phenotype in these patients. The investigators will therefore study the usefulness of global hemostatic methods (endogenous thrombin potential (ETP), overall hemostatic potential (OHP), fibrin clot structure) and microparticles in the prediction of severity of bleeding and estimation of response to the treatment in patients with hemophilia. Since hemophilia patients on prophylactic treatment virtually do not bleed, additional patients who are treated on demand only will be included enabling to study possible modulatory effects of different hemostatic factors (particularly prothrombotic and thrombin activatable fibrinolysis inhibitor (TAFI)) on clinical presentation. The investigators will correlate both those factors and clinical severity with global hemostatic methods. The investigators expect to prove that individual tailoring of the treatment, which may enable lowering the prophylactic dose of factor concentrate without increasing the risk of bleeding, is justified in some hemophilia patients. This approach would reduce the amount of necessary factor concentrate in certain patients and decrease the cost (which represents extensive burden for health care systems) of treatment without potential risk for the patients.