Hemophilia A Clinical Trial
Official title:
UK-PK Study: National Study of the Implementation of a Pharmacokinetic-Focused Educational Package for Patients Living With Severe Haemophilia A
Prospective, interventional study of pharmacokinetic (PK)-focused FVIII dosing discussions
delivered as part of a patient education package.
The study will capture severe haemophilia A patient reported outcome measures before and
after PK-focused dosing discussions, including a standardised patient education package, that
include personalised PK-guided dosing suggestions from a computational predictive device
(myPKFiT®). The pragmatic study design recognises the CE marked myPKFiT® device is being
implemented into routine care nationally and consequently only requires a single additional
clinic visit for the purpose of consenting.
There is a paucity of patient reported outcome data documenting the impact of PK focused
prophylaxis discussions. This prospective, multicentre UK-PK trial will coincide with the
initiation of the myPKFiT® device in the context of an enhanced clinical review. This will
include a standardised patient education package using a short animated film explaining PK
and personalised prophylaxis, followed by modelling of the patient's own PK using the
myPKFiT® device and discussing the dosing options with the patient. The aim of the PK
educational package is to educate and engage patients and ensure that their treatment
regimens meet their individual needs. In addition, the study will capture the prophylaxis
dosing strategy agreed upon by patients and the clinical team where treatment decisions are
made jointly between patients and the clinical team (concordance). Looking forward,
haemophilia clinical care team members need a marker to judge or predict patient engagement
with this personalising medicine process. Successful consultation strategies may differ if
the team can anticipate how an individual person with haemophilia (PWH) might respond to
discussions about potential change. The primary outcome measure is the Patient Activation
Measure (PAM) which gauges the knowledge, skills and confidence essential to managing one's
own health and healthcare. A PAM score can predict healthcare outcomes including medication
adherence, healthcare utilisation and hospital visits. It is hypothesized that a more
activated patient will be more receptive to changes in their treatment regimen, education and
information about their health condition.
In addition to patient activation, the study will assess a range of patient reported outcomes
and psychological factors using validated tools. These will include patients'
Haemophilia-related quality of life (HAEMO-QoL-A), beliefs about medicines (BMQ),
self-reported adherence (VERITAS-Pro) and activity levels. These tools will provide clear
patient reported outcomes for the haemophilia community, with accompanying EQ5D data,
enabling robust health economic evaluation.
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