PK Driven Prophylaxis for Hemophilia A

Assistance Publique - Hôpitaux de Paris12 enrolled

Overview

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

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

Study Type

INTERVENTIONAL

Allocation

Purpose

SUPPORTIVE_CARE

Masking

NONE

Enrollment

Conditions

Hemophilia A

Interventions

MyPKFiTDEVICE

Adjustment of the treatment dose using a decision support software according to the PK values

Eligibility

Sex: MALEMin age: 6 Years

Medical Language ↔ Plain English

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

Locations (1)

AP-HP, Bicêtre Hospital

Le Kremlin-Bicêtre, France

Outcomes

Primary Outcomes

Joint Annual Bleeding Rate

Joint Annual Bleeding Rate assessed by usual patient book

Time frame: during 6 months before (retrospectively) and up to 12 months after using of myPKFiT

Secondary Outcomes

Total annual bleeding rate

Total Annual Bleeding Rate assessed by usual patient book

Time frame: 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

Time frame: up to 12 months

Joint function

Joint function assessed thanks to score of joint state "Haemophilia Joint Health Score" (HJHS)

Time frame: 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

Time frame: 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

Time frame: 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"

Time frame: up to 6 and 12 months

Data from ClinicalTrials.gov

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