Hemophilia A
Conditions
Keywords
Hemophilia A, Prophylaxy, Pharmacokinetic, Bayesian
Brief summary
The purpose of this study is to evaluate the implementation of pharmacokinetic (PK) driven prophylaxis in current clinical practice
Detailed description
Starting from 3 pharmacokinetic (PK) points, data are entered in the PK calculator device, and treatment dose is adjusted following the results of PK values obtained and clinical bleeding data
Interventions
DEVICEMyPKFiT
Adjustment of the treatment dose using a decision support software according to the PK values
Sponsors
Baxter Healthcare Corporation
Assistance Publique - Hôpitaux de Paris
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
SUPPORTIVE_CARE
Masking
NONE
Eligibility
Sex/Gender
MALE
Age
6 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Male Patient * Severe or moderately severe haemophilia A (FVIII = 2 %), preventively treated (processing) by ADVATE for at least 6 months, * At last 6 years old, * An informed consent must be signed by the patient or his legal representative for the patients minor. * Affiliated to a national insurance scheme
Exclusion criteria
* Previous or actual treatment with FVIII inhibitors \> 0,6 UB at the selection * Induction of immune tolerance * Planned orthopedic surgery for the 18 next months * Any other haemostatic pathology * Any treatment interacting on the haemostasis * Patient under guardianship * Patient participating in another biomedical research
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Joint Annual Bleeding Rate | during 6 months before (retrospectively) and up to 12 months after using of myPKFiT | Joint Annual Bleeding Rate assessed by usual patient book |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Quality of life | up to 12 months | Quality of life assessed by QoL-A-Haemo questionnaire for Adult and Haemo-QoL for children aged 6-17 years |
| Joint function | up to 12 months | Joint function assessed thanks to score of joint state Haemophilia Joint Health Score (HJHS) |
| Adherence to treatment estimated as the quantity of Facteur VIII consumed reported to the prescribed quantity. | up to 6 and 12 months | Quantity of Facteur VIII consumed will be assessed by usual patient book |
| Total annual bleeding rate | during 6 months before (retrospectively) and up to 12 months after using of myPKFiT | Total Annual Bleeding Rate assessed by usual patient book |
| Hemorrhagic Risk Sport | up to 6 and 12 months | Hemorrhagic Risk Sport assessed by the scale Hemophilia and sport |
| Evolution between the annualized incidence of intercurrent events appeared or worsened under each treatment period (with and without myPKFit), globally, by System Organ ( SO) and Preferred Term ( PT) of the code MedDRA | during 6 months before (retrospectively) and up to 12 months after using of myPKFiT | — |
| Consumption of Factor VIII estimated by the Quantity of Facteur VIII consumed by thr patient. | during 6 months before (retrospectively) and up to 12 months after using of myPKFiT | Consumption of Factor VIII assessed by usual patient book |
Countries
France
Outcome results
None listed