Implementation of pharmacokinetic-guided dosing of prophylaxis in hemophilia patients.
- Conditions
- bleeding disorderhemophilia10005330
Recruitment & Eligibility
- Status
- Pending
- Sex
- Not specified
- Target Recruitment
- 50
Inclusion Criteria
- Hemophilia A and B patients of all ages on prophylaxis;
- Prophylaxis with SHL factor concentrates or EHL factor concentrates;
- Written (parental) informed consent, according to local law and regulations
Exclusion Criteria
- Patients with other congenital or acquired hemostatic abnormalities;
- General medical conditions which may interfere with participation in the
study;
- Inability to adhere to prophylaxis and/ or inability to keep detailed logs on
infusion and bleeding episodes;
- Withdrawal of (parental) informed consent.
,
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method <p>1. Observed FVIII and FIX levels in comparison to prospectively predicted FVIII<br /><br>and FIX levels by MAP Bayesian analysis (predictive performance). </p><br>
- Secondary Outcome Measures
Name Time Method <p>1. Association of both prospectively and retrospectively collected real life-<br /><br>and simulated FVIII/FIX levels with bleeding episodes, daily activities and<br /><br>joint status.<br /><br>2. Expectations, feasibility and experience with PK-guided dosing with the<br /><br>different factor concentrates (standard half-life versus extended half-life) as<br /><br>reported by patient/ caretakers and physician will be measured using a Visual<br /><br>Analogue Scale (VAS) at the start and end of the study.<br /><br>3. Economic analysis in which costs and benefits of standard prophylactic<br /><br>treatment and PK-guided prophylaxis are compared.<br /><br>4. Analysis of modifiers effecting PK parameters of FVIII/FIX concentrate in<br /><br>order to further optimize population PK models. </p><br>