Paediatric UK demyelinating disease longitudinal study (PUDDLS)

<p>Abstract</p> <p>Background</p> <p>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...

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Main Authors: Likeman Marcus, Kneen Rachel, Jardine Philip, Hemingway Cheryl, Gunny Roxanna, Foster Katharine, De Goede Christian, Chong Wui K, Cummins Carole, Absoud Michael, Lim Ming J, Pike Mike, Sibtain Naomi, Whitehouse William P, Wassmer Evangeline
Format: Article
Language:English
Published: BMC 2011-07-01
Series:BMC Pediatrics
Online Access:http://www.biomedcentral.com/1471-2431/11/68
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spelling doaj-d9ce238d0fc5485494278cd3244a3a432020-11-25T01:03:49ZengBMCBMC Pediatrics1471-24312011-07-011116810.1186/1471-2431-11-68Paediatric UK demyelinating disease longitudinal study (PUDDLS)Likeman MarcusKneen RachelJardine PhilipHemingway CherylGunny RoxannaFoster KatharineDe Goede ChristianChong Wui KCummins CaroleAbsoud MichaelLim Ming JPike MikeSibtain NaomiWhitehouse William PWassmer Evangeline<p>Abstract</p> <p>Background</p> <p>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.</p> <p>Methods/Design</p> <p>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.</p> <p>Discussion</p> <p>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.</p> http://www.biomedcentral.com/1471-2431/11/68
collection DOAJ
language English
format Article
sources DOAJ
author Likeman Marcus
Kneen Rachel
Jardine Philip
Hemingway Cheryl
Gunny Roxanna
Foster Katharine
De Goede Christian
Chong Wui K
Cummins Carole
Absoud Michael
Lim Ming J
Pike Mike
Sibtain Naomi
Whitehouse William P
Wassmer Evangeline
spellingShingle Likeman Marcus
Kneen Rachel
Jardine Philip
Hemingway Cheryl
Gunny Roxanna
Foster Katharine
De Goede Christian
Chong Wui K
Cummins Carole
Absoud Michael
Lim Ming J
Pike Mike
Sibtain Naomi
Whitehouse William P
Wassmer Evangeline
Paediatric UK demyelinating disease longitudinal study (PUDDLS)
BMC Pediatrics
author_facet Likeman Marcus
Kneen Rachel
Jardine Philip
Hemingway Cheryl
Gunny Roxanna
Foster Katharine
De Goede Christian
Chong Wui K
Cummins Carole
Absoud Michael
Lim Ming J
Pike Mike
Sibtain Naomi
Whitehouse William P
Wassmer Evangeline
author_sort Likeman Marcus
title Paediatric UK demyelinating disease longitudinal study (PUDDLS)
title_short Paediatric UK demyelinating disease longitudinal study (PUDDLS)
title_full Paediatric UK demyelinating disease longitudinal study (PUDDLS)
title_fullStr Paediatric UK demyelinating disease longitudinal study (PUDDLS)
title_full_unstemmed Paediatric UK demyelinating disease longitudinal study (PUDDLS)
title_sort paediatric uk demyelinating disease longitudinal study (puddls)
publisher BMC
series BMC Pediatrics
issn 1471-2431
publishDate 2011-07-01
description <p>Abstract</p> <p>Background</p> <p>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.</p> <p>Methods/Design</p> <p>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.</p> <p>Discussion</p> <p>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.</p>
url http://www.biomedcentral.com/1471-2431/11/68
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