Patient reported outcomes in rare diseases
Research type
Research Study
Full title
Patient Reported OutComEs in rarE Disease (PROCEED)
IRAS ID
201876
Contact name
Fatima Isa
Contact email
Sponsor organisation
University of Birmingham
Duration of Study in the UK
0 years, 5 months, 31 days
Research summary
The Institute of Transitional Medicine(ITM) Birmingham have recently opened a new Centre for Rare Disease (CfRD), which brings together multi-speciality and multi-disciplinary teams to deliver care of patients with rare diseases, based within the Institute of Translational Medicine(ITM), Birmingham.
PRO measures can capture a variety of concepts that are relevant to the patients, such as symptom frequency, severity or change; the level of physiological functioning; health-related quality of life (HRQOL); treatment satisfaction; treatment side effects and treatment adherence.
The aim of this research is to develop a pathway for the optimal selection of PROs for inclusion within the CfRD clinical information platform. Initial work in this area will focus on two diverse patient populations that receive clinical care within the new centre – (i) patients with Primary Sclerosing Cholangitis, and (ii) patients transitioning from child to adult renal transplantation services.
PSC is slow progressive disease and can lead to cirrhosis, portal hypertension, the tumour of the liver and liver failure.The exact cause and mechanism of PSC are unknown.
One of the major challenges for renal transplantation in adolescence is high rate graft failures and acute or chronic rejections due to non-adherence and non-compliance. Rejection episodes can be life threatening to patients and is a significant burden on the health system.
This study aims:
• To explore patients experiences of living with Primary Sclerosing Cholangitis and the impact of their symptoms/care on their quality of life (QOL)
• To explore the experiences of patients transitioning from child to adult renal transplantation services and the impact of their symptoms/transition care on their quality of life
• To explore clinician’s experience of caring for patients with PSC and renal transplant
• To explore the viewpoints of patients and clinicians on the use of PROMs in the NHS with the aim of supporting patient care
The study will use face to face or telephone interviews to explore the experience of patients living with PSC and renal transplant and the impact of the symptoms/care on their quality of life. In the long term, the data of this study will add to the body of evidence on the burden of PSC and challenges of renal transplant, to identify potential areas for new intervention and audit and benchmark purposes.REC name
West Midlands - Edgbaston Research Ethics Committee
REC reference
16/WM/0198
Date of REC Opinion
20 May 2016
REC opinion
Further Information Favourable Opinion