Study protocol
Paediatric UK demyelinating disease longitudinal study (PUDDLS)
1 School of Health & Population Sciences, University of Birmingham, Vincent Drive, Birmingham, B15 2TT, UK
2 Department of Neurology, Birmingham Children's Hospital, Steelhouse Lane, Birmingham, B4 6NH, UK
3 Department of Neuroradiology, Great Ormond Street Hospital for Children, Great Ormond Street, London, WC1N 3JH, UK
4 Department of Paediatric Neurology, Royal Preston Hospital, Sharoe Green Lane North, Lancashire PR2 9HT, UK
5 Department of Neuroradiology, Birmingham Children's Hospital, Steelhouse Lane, Birmingham, B4 6NH, UK
6 Department of Neurology, Great Ormond Street Hospital for Children, Great Ormond Street, London, WC1N 3JH, UK
7 Department of Neurology, Bristol Royal Hospital for Children, Upper Maudlin Street, Bristol, BS2 8BJ, UK
8 Department of Neurology, Alder Hey Children's NHS Foundation Trust, Eaton Road, Liverpool, L12 2AP, UK
9 Department of Neuroradiology, Bristol Royal Hospital for Children, Upper Maudlin Street, Bristol, BS2 8BJ, UK
10 Department of Neurology, The Evelina Children's Hospital at Guy's & St Thomas' NHS Trust, Great Maze Pond, London, SE1 9RT, UK
11 Department of Neurology, Oxford Children's Hospital, Headley Way, Oxford, OX3 9DU, UK
12 Department of Neuroradiology, King's College Hospital NHS Trust, Denmark Hill, London, SE5 9RS, UK
13 Academic Division of Child Health, Queen's Medical Centre, Nottingham, NG7 2UH, UK
BMC Pediatrics 2011, 11:68 doi:10.1186/1471-2431-11-68
Published: 28 July 2011Abstract
Background
There is evidence that at least 5% of Multiple sclerosis (MS) cases manifest in childhood. Children with MS present with a demyelinating episode involving single or multiple symptoms prior to developing a second event (usually within two years) to then meet criteria for diagnosis. There is evidence from adult cohorts that the incidence and sex ratios of MS are changing and that children of immigrants have a higher risk for developing MS. A paediatric population should reflect the vanguard of such changes and may reflect trends yet to be observed in adult cohorts. Studying a paediatric population from the first demyelinating event will allow us to test these hypotheses, and may offer further valuable insights into the genetic and environmental interactions in the pathogenesis of MS.
Methods/Design
The Paediatric UK Demyelinating Disease Longitudinal Study (PUDDLS) is a prospective longitudinal observational study which aims to determine the natural history, predictors and outcomes of childhood CNS inflammatory demyelinating diseases. PUDDLS will involve centres in the UK, and will establish a cohort of children affected with a first CNS inflammatory demyelinating event for long-term follow up by recruiting for approximately 5 years. PUDDLS will also establish a biological sample archive (CSF, serum, and DNA), allowing future hypothesis driven research. For example, the future discovery of a biomarker will allow validation within this dataset for the evaluation of novel biomarkers. Patients will also be requested to consent to be contacted in the future. A secondary aim is to collaborate internationally with the International Paediatric Multiple Sclerosis Study Group when future collaborative studies are proposed, whilst sharing a minimal anonymised dataset. PUDDLS is the second of two jointly funded studies. The first (UCID-SS) is an epidemiological surveillance study that already received ethical approvals, and started on the 1st September 2009. There is no direct patient involvement, and UCID-SS aims to determine the UK and Ireland incidence of CNS inflammatory demyelinating disorders in children under 16 years.
Discussion
A paediatric population should reflect the vanguard of MS epidemiological changes and may reflect trends yet to be observed in adult MS cohorts. The restricted window between clinical expression of disease and exposure to environmental factors in children offers a unique research opportunity. Studying a paediatric population from the first demyelinating event will allow us to investigate the changing epidemiology of MS, and may offer further valuable insights into the genetic and environmental interactions in the pathogenesis of MS.
