Markers of hypothalamic dysfunction in children
Research type
Research Study
Full title
Markers of Hypothalamic Dysfunction in Children with Hypothalamo-Pituitary lesions
IRAS ID
239254
Contact name
Mehul T Dattani
Contact email
Sponsor organisation
University College London
Clinicaltrials.gov Identifier
Z6364106/2018/08/23 , UCL Data Registration
Duration of Study in the UK
2 years, 7 months, 1 days
Research summary
The hypothalamus is a small but vital brain structure regulating essential body functions and it is tightly connected with the pituitary gland, the master regulatory gland of the endocrine system. Hypothalamic dysfunction (HD) is a rare, complex and poorly understood disorder associated with significant morbidity, compromising quality of life, increasing disability and causing premature death. Current clinical and biochemical tools and conventional qualitative magnetic resonance imaging (MRI) techniques do not allow clear differentiation between hypothalamic and more manageable pituitary disorders. As a result, the endocrine phenotypes of these children are highly unpredictable and targeted interventions are lacking.
The proposed prospective cross-sectional study aims to: i) assess the role of advanced quantitative MRI techniques in identifying structural and functional hypothalamic abnormalities; and ii) correlate the imaging findings with specific hormonal patterns and clinical symptoms in order to develop prediction models directly translatable into clinical practice. The secondary aim is to potentially develop a clinical questionnaire to help clinicians to accurately diagnose HD in children with hypopituitarism.
The study will take place at Great Ormond Street (GOS) Hospital and University College London (UCL) GOS Institute of Child Health (ICH). It will last 3 years and it will be funded by the “Children with Cancer UK Doctoral Fellowship”.
Sixty patients with hypothalamo-pituitary lesions will be enrolled and they will undergo: clinical examination and questionnaires, hormonal and imaging assessment.
The proposed study is directly relevant to any children with acquired or congenital lesions of the hypothalamo-pituitary axis, for whom current therapies are inadequate to ameliorate life-limiting obesity, risk of secondary type 2 diabetes mellitus and neuro-behavioural disorders, or improve health-related quality of life. In particular these issues are relevant to the large cohort of survivors of suprasellar brain tumours and hypothalamo-pituitary maldevelopmental lesions who are significantly affected by the burden of hypothalamic damage.REC name
London - Bloomsbury Research Ethics Committee
REC reference
18/LO/1552
Date of REC Opinion
9 Oct 2018
REC opinion
Favourable Opinion