The goal of this multicentre, prospective, open-label, cross-over clinical study is to determine whether individualized PK-guided dosing of emicizumab is non-inferior to conventional dosing of emicizumab in the prevention of bleeding in congenital haemophilia A patients.
Confirmed diagnosis of congenital haemophilia A, with a baseline endogenous FVIII of <6 IU/ml
Aged > 1 year at inclusion (inclusion of children 1-16 years after favourable interim-analysis see protocol)
Receiving conventional dosing of emicizumab (6 mg/kg/4 weeks with varying intervals) for a duration of at least 12 months prior to inclusion;
Willing and able to provide written informed consent, either by the subject or its parents/legal guardian
Acquired haemophilia A
Crossover
Emicizumab - PK-guided dose reduction
Other interventionPK-guided dose reduction emicizumab targeted at a Ctrough of 30μg/mL.
Emicizumab - Dosis continuation group
Other interventionContinue on their current dose regimen
Emicizumab - Dose adjustment group
Other interventionAdjusted in dosing regimen according to local protocol
Proportion of patients without treated bleeds
Comparison proportion treated bleeds 6 months before (conventional) and after intervention (PK-guided dosing)
Proportion of patients without treated bleeds
Comparison proportion without treated bleeds 12 months before (conventional) and after intervention (PK-guided dosing)
Proportion of patients without spontaneous joint- or muscle bleeds
Comparison proportion without spontaneous joint- or muscle bleeds 6 and 12 months before and after intervention.
Annualized bleeding rate (ABR) of treated bleeds, including joint bleeds and sport-induced bleeds
Comparison annualized bleeding rate 6 and 12 months before and after intervention.
To compare cost-effectiveness between conventional dosing and individualized PK-guided dosing of emicizumab
Direct and indirect medical costs: 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. 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).
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Kathelijn Fischer
Lead sponsor
UMC Utrecht
Sponsor institution
Erasmus Medical Center
Collaborator
Amsterdam University Medical Center
Collaborator
Leiden University Medical Center
Collaborator
Radboud University Medical Center
Collaborator
Maastricht University Medical Center
Collaborator
University Medical Center Groningen
Collaborator
HagaZiekenhuis
Collaborator
Dutch Society of Haemophilia Patients
Collaborator
