Brief Title
Does the Thrombin Generation Test Performed During the Pharmacokinetic Profile of the Substitutive Factor VIII Bring Benefits to the Personalized Treatment of Pediatric Patients and Adult Hemophilia A Patients Under Prophylaxis ?
Official Title
Does the Thrombin Generation Test Performed During the Pharmacokinetic Profile of the Substitutive Factor VIII Bring Benefits to the Personalized Treatment of Pediatric Patients and Adult Hemophilia A Patients Under Prophylaxis ?
Brief Summary
In the context of hemophilia, it is well know that the level of factor VIII alone does not reflect the clinical phenotype of the patients in an accurate way. At equal factor VIII levels, certain patients will bleed more than others. The thrombin generation test (TGT) is a test that seems to provide a better prediction of the overall hemostatic status of an individual patient. In a previous study, the investigators have established normal reference values of the thrombin generation curve in children aged 6 months to 16 years and adults. The goal was to evaluate the use of this test in different clinical contexts and in severe hemophilia patients in particular. A pilot study showed that the patients having a thrombin generation <150 had a severe phenotype, whether those who received an appropriate prophylaxy had a thrombin generation superior to 150. Moreover, the investigators now have access to a software tool that allows them to individually determine the pharmacokinetic profile of the factor VIII injected to each patient. The factor VIII concentration is measured at injection and 30 minutes, 1 hour, 2 hours and 24 hours afterwards. The introduction of these concentrations in the software allows to obtain the half-life of factor for a given patient, the maximum peak, and the minimum factor level (though level). The injected dosis might be sufficient (disappearance of substantial diminution of the bleedings) or unsufficient (persisting bleeding) for a given patient. This study aims: - to measure the pharmacokinetic profile of factor VIII by two different methods, the time-based method and the chromogenic method - to correlate the results with the TGT results obtained at the same time points and determine which method gives the best correlation - to link the clinical symptomatology (improved symptomatology or not) with the TGT results - to determine which minimal TGT result is linked to a minimal bleeding rate - to adapt the prophylactic dosis of the patient in a personalized way.
Study Type
Interventional
Primary Outcome
Factor VIII blood concentration - chronometric method
Condition
Hemophilia
Intervention
Chronometric method
Study Arms / Comparison Groups
hemophilia
Description: Patients with severe hemophilia (or moderate hemophilia if presence of hemorrhages) under prophylaxy and subjected to a pharmacokinetic profile of factor VIII
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Device
Estimated Enrollment
50
Start Date
January 2017
Completion Date
July 2020
Primary Completion Date
July 2020
Eligibility Criteria
Inclusion Criteria: - Patients with severe or moderate hemophilia, on prophylaxis, and suffering from bleedings. Exclusion Criteria: - Patients with difficult venous access - Patients who have had surgery or trauma in the month before, patients with acute disease (infection, inflammation)
Gender
All
Ages
N/A - N/A
Accepts Healthy Volunteers
No
Contacts
Anne Demulder, MD, 024773990, [email protected]
Location Countries
Belgium
Location Countries
Belgium
Administrative Informations
NCT ID
NCT02803502
Organization ID
CHUB-PK TGT
Responsible Party
Principal Investigator
Study Sponsor
Brugmann University Hospital
Study Sponsor
Anne Demulder, MD, Principal Investigator, CHU Brugmann
Verification Date
November 2017