Bleeding disorder FVIII deficiency
Conditions
Interventions
The intervention consist of 12 months PK-guided dosing of emicizumab targeted a
a Ctrough of 30 µg/mL (± 15 %).
Based on emicizumab levels during visit 1, there a 3 groups:
Intervention group:
- Sub
Sponsors
Universitair Medisch Centrum Utrecht
Eligibility
Age
No minimum to 99 Years
Inclusion criteria
Inclusion criteria: - Confirmed diagnosis of congenital haemophilia A, with a baseline endogenous FVIII of 1 year at inclusion - Receiving conventional dosing of emicizumab (6 mg/kg/4 weeks with varying intervals) for a duration of at least 12 months prior to inclusion; - Having good bleeding control, defined as: i. No spontaneous joint/muscle bleeds in the previous 6 months AND ii. A maximum of two treated (traumatic) bleeds in the previous 6 months. - Willing and able to provide written informed consent, either by the subject or its parents/legal guardian - Willing to provide bleeding assessment information - Willing to adhere to the medication regimen
Exclusion criteria
Exclusion criteria: Acquired haemophilia A
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| The primary outcome parameter is bleeding, defined as: 1) Proportion of patients without treated bleeds (6 months Bleeding Assessment Phase versus 6 months PK-Guided Dosing Phase). | — |
Secondary
| Measure | Time frame |
|---|---|
| The secondary outcome are defined as: 1. Proportion of patients without treated bleeds (12 months Clinical Phase+Bleeding Assessment Phase versus 12 months PK-guided Dosing Phase+Dose Continuation Phase) 2. Proportion of patients with spontaneous joint- or muscle bleeds (6 months Bleeding Assessment Phase versus 6 months PK-Guided Dosing Phase). 3. Proportion of patients with spontaneous joint- or muscle bleeds (12 months Clinical Phase+Bleeding Assessment Phase versus 12 months PK-guided Dosing Phase+Dose Continuation Phase). 4. Annualized bleeding rate (ABR) of treated bleeds, including joint bleeds and sports induced bleeds ((6 months Bleeding Assessment Phase versus 6 months PK-Guided Dosing Phase). 5. Annualized bleeding rate (ABR) of treated bleeds, including joint bleeds and sports induced bleeds (6 months retrospective + 6 months prospective data (Clinical Phase+Bleeding Assessment Phase) versus 12 months prospective data of PK guided dosing (PK-guided Dosing Phase+Dose Continuation Phase). 6. Cost-effectiveness between 6 months of conventional dosing (Bleeding Assessment Phase) and 6 months of individualized PK-guided dosing of emicizumab (PK-Guided Dosing Phase): 7 Direct and indirect medical costs: 7.1 Direct medical costs are predominantly determined by consumption of emicizumab, additional FVIII, and/or bypassing agents, extracted from the hospital*s pharmacy records. These data are highly reliable, as this medication is exclusively distributed by haemophilia treatment centers. 7.2 Indirect medical costs: are number of (emergency) hospital visits, bleeding related hospital admissions and/or unscheduled surgeries, and days lost from work/school (for patients and/or caregivers). 8. Predictive performance of the MAP Bayesian procedure used for the dose adaptation procedure, defined as % of patients within ±20% of within the target level of 20-39 µg/mL of emicizumab. All emicizumab plasma levels will be determined in the labora | — |
Countries
Netherlands
Outcome results
None listed