PK Driven Prophylaxis for Hemophilia A

Study Overview
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Brief Summary
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The purpose of this study is to evaluate the implementation of pharmacokinetic (PK) driven prophylaxis in current clinical practice

Detailed Description
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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

Official Title
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Interest of Preventive Personalized Treatment in Hemophilia A

Conditions
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Hemophilia A

Intervention / Treatment
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* Device: MyPKFiT


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Minimum Age: 6 Years

Sexes Eligible for Study
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Male

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Supportive Care
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: MyPKFiT<br>Personalized prophylaxis : Treatment is adjustment according to PK modeling | Device: MyPKFiT<br>* Adjustment of the treatment dose using a decision support software according to the PK values<br>* Other names: Personalized prophylaxis by PK modeling;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Joint Annual Bleeding Rate | Joint Annual Bleeding Rate assessed by usual patient book | during 6 months before (retrospectively) and up to 12 months after using of myPKFiT | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Total annual bleeding rate | Total Annual Bleeding Rate assessed by usual patient book | during 6 months before (retrospectively) and up to 12 months after using of myPKFiT | 
| Quality of life | Quality of life assessed by QoL-A-Haemo questionnaire for Adult and Haemo-QoL for children aged 6-17 years | up to 12 months | 
| Joint function | Joint function assessed thanks to score of joint state Haemophilia Joint Health Score (HJHS) | up to 12 months | 
| Adherence to treatment estimated as the quantity of Facteur VIII consumed reported to the prescribed quantity. | Quantity of Facteur VIII consumed will be assessed by usual patient book | up to 6 and 12 months | 
| Consumption of Factor VIII estimated by the Quantity of Facteur VIII consumed by thr patient. | Consumption of Factor VIII assessed by usual patient book | during 6 months before (retrospectively) and up to 12 months after using of myPKFiT | 
| Hemorrhagic Risk Sport | Hemorrhagic Risk Sport assessed by the scale Hemophilia and sport | up to 6 and 12 months | 
| 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 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Hemophilia A, Prophylaxy, Pharmacokinetic, Bayesian