Health in Children and Young People with CAH
Research type
Research Study
Full title
Defining the evidence base for health care delivery in congenital adrenal hyperplasia – a multi-centre CAH-UK (Children & Adolescents with CAH in the UK) initiative)
IRAS ID
191301
Contact name
Nils P Krone
Contact email
Sponsor organisation
Sheffield Children's NHS Foundation Trust
Duration of Study in the UK
1 years, 5 months, 1 days
Research summary
Congenital Adrenal Hyperplasia (CAH) is associated with significant morbidity and mortality and occurs in about 1 in 10,000 life-births (about 6,300 individuals in the UK). Recent clinical data suggest impaired heath in young adults with CAH. The onset of such problems during childhood remains unclear. However, it is of paramount importance to define an evidence base to develop strategies for secondary prevention of future health problems. To define novel predictive markers of long-term health problems, we have formed a clinical consortium (CAH-UK) involving 16 tertiary UK centres to explore the current health status in 8 to 18 year olds with CAH. We have designed a cross-sectional study to define the current health in children and young people with CAH in the UK. These patients undergo there to six monthly blood tests as part of the routine clinical care. We have planned to add on additional blood, urine and saliva collections to devise novel test and explore the metabolic status. In addition, we will for the first time define if quality of life is already altered at this young age as it is in young adults. We will establish the presence and onset of potential health problems in children and young persons with CAH in the UK. Study participants will have an immediate benefit from this study as their therapy can be individualised. After validation novel markers of disease control will be implemented into clinical practice within the next 2-3 years improving personalisation of treatment in all patients with CAH. The identification of altered health related quality of life would aid developing care strategies. This combination of investigations represents a major step towards a personalised therapeutic approach in CAH.
REC name
Yorkshire & The Humber - Sheffield Research Ethics Committee
REC reference
15/YH/0537
Date of REC Opinion
27 Jan 2016
REC opinion
Further Information Favourable Opinion