Factor VIII Deficiency, Hemophilia, Hemophilia A
Conditions
Keywords
Hemophilia, Hemophilia A, Factor VIII, Advate, Antithrombotic factor, Pharmacokinetic dosing, Hemarthrosis, myPKFiT
Brief summary
The purpose of this study is to describe the pharmacokinetic profile of patients with hemophilia A in prophylaxis in Spain using myPKFit®
Detailed description
This pharmacokinetic profile will be obtained in only in patient using Advate® (INN-octocog alfa) or Adynovi® (INN- rurioctocog alfa pegol), both recombinant intravenous FVIII products, with myPKFiT®. This patients will be followed during the period of 12 months, and the pharmacokinetic will be measured collecting at least 2 blood samples, in which the presence of factor VIII will be determined by the usual clinical methods: chromogenic method or coagulative method.
Interventions
DRUGoctocog alfa
Pharmacokinetic Characterization of the Hemophilia A Population in Spain using an standalone web-based software
Pharmacokinetic Characterization of the Hemophilia A Population in Spain using an standalone web-based software
Sponsors
Takeda
Instituto de Investigación Hospital Universitario La Paz
Spanish Society of Thrombosis and Haemostasis
Study design
Observational model
COHORT
Time perspective
OTHER
Eligibility
Sex/Gender
MALE
Age
1 Years to 65 Years
Healthy volunteers
No
Inclusion criteria
* Male patients. * Age between 1 and 65 years old. * Patients diagnosed with Hemophilia A who are on prophylactic treatment with Advate® or Adynovi® or adjusted with myPKFIT. * Patients older than 18 who have signed the informed consent form. * In the case of mature minors, in addition to the consent signed by the legal guardian, an assent of the minor must be obtain. * In the case of patients legally incapable for giving their consent, their primary caregiver will consent as the patient's legal guardian.
Exclusion criteria
* Withdrawal of informed consent. * Patients with any medical or psychological condition that according to the researcher's criteria prevents them from following the usual clinical practice procedures. * Patients with concomitant diagnosis of other haemostasis disorders. * Patients being treated for induction of immunologic tolerance at the time of inclusion.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Pharmacokinetic profile: FVIII half-life measured with MyPKFiT | 3-4 hours postdose +/- 30 minutes | Change from Baseline to 12 months |
| Pharmacokinetic profile: Clearance measured with MyPKFiT | 3-4 hours postdose +/- 30 minutes | Change from Baseline to 12 months |
| Pharmacokinetic profile: Volume in steady state measured with MyPKFiT | 3-4 hours postdose +/- 30 minutes | Change from Baseline to 12 months |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Annual Joint Bleeding Record | baseline | Hemophilia Joint Health Score |
| Hemorrhagic risk of physical activity | baseline | Measured with Broderick scale |
| Adverse Events | baseline | Collect and identify adverse events |
| Self-adherence to treatment evaluation | month 12 | Measured by VERITAS- PRO scale |
| Joint condition | baseline | Hemophilia Joint Health Score |
| Number of total bleeding episodes in a year | baseline | Measured by annual bleeding record |
Countries
Spain
Outcome results
None listed