VoT2: Investigations of Healthcare Delivery to Patients with PKU
Research type
Research Study
Full title
VoT2: Investigations of Healthcare Delivery to Patients with Phenylketonuria (PKU)
IRAS ID
284593
Contact name
Darragh Nerney
Contact email
Sponsor organisation
European Brain Council (EBC)
Duration of Study in the UK
0 years, 10 months, 0 days
Research summary
The VoT2 – PKU Study will aim to identify best practice in the management of adolescents and adults with Phenylketonuria (PKU) across three participating centres (Eire (Ireland), UK, and Spain), and potentially additional European centres identified through MetabERN. The proposed research will encompass both pediatric and adult services.
Data collection will be comprised of three components:
1) Centre-based unit
- Characterization of staffing, clinic resources and workload, and model of care across the three centres
- Identification of the number of PKU patients cared for, guidelines followed and adherence to targets
- Survey of Dieticians working with children or adults to explore their treatment of PKU patients.2) Centre-based audit of medical records
- Demographic data: Age- and gender- distribution, nationality, ethnicity, relationship status, highest educational achievement, living status, employment status, for females of reproductive age: gravida/parity
- Medical data: Genotype, HbA1c (Glycated Haemoglobin), BMI, height, weight, waist circumference, lipid profile, blood pressure, phenylalanine level, clinic attendance, resource utilization, co-morbidities and concomitant medications.
*Medical data collected retrospectively will vary between sites (Eire/UK/Spain) according to routine standard of care.3) Prospective patient data collection
- A survey to evaluate patient satisfaction with current delivery of care and challenges in their management
- Questionnaires to assess overall well-being and quality of life (i.e. HADS, EQ-5D-5L, and the FNS).
*Patients to be targeted for prospective data collection are those included in the centre-based audit of medical records.It is hoped, the planned observations and subsequent analysis will help to advance care pathways by examining the services’ current practice arrangements combined with analysis of patient characteristics and resource utilization; aiming to identify gaps in delivery of care so as to improve patient satisfaction and overall clinical outcome, health status and well-being.
REC name
East Midlands - Nottingham 1 Research Ethics Committee
REC reference
20/EM/0269
Date of REC Opinion
19 Nov 2020
REC opinion
Unfavourable Opinion