Childhood central nervous system acquired demyelinating disorders: incidence, clinical features, MRI characteristics and prognostic features

[摘要] Objectives: Acquired demyelinating syndromes (ADS) are rare childhood central nervous system disorders. In this thesis I aimed to describe the UK incidence, clinical features, MRI characteristics and prognostic features of childhood ADS.Methods: I describe the first UK population active surveillance and one year follow up study which features multiple source ascertainment, MRI review, and blinded clinical expert panel diagnostic review. I also describe: 4 longitudinal retrospective case series delineating prognostic risk factors; outline the setup of a longitudinal cohort; and describe methodological concepts important for the design of the future clinical trials in ADS.Results: The incidence of first onset ADS in children aged 1-15 years old was 9.83 per million children per year; the highest surveillance rate reported to date. The female-to-male ratio in children older than 10 years was 1.52:1. A trend towards higher incidence rates of ADS in children of South Asian and Black ethnicity was observed compared with White children. A number of MRI characteristics distinguished acute disseminated encephalomyelitis cases (1/3 of cohort) from clinically isolated syndrome (CIS) cases (2/3 of cohort). Of CIS cases with contrast imaging, 26% fulfilled McDonald 2010 MS diagnostic criteria. Predictive risk factors for MS diagnosis included: CIS presentation; presence of periventricular lesions on MRI brain scan; and age> 8 years.Conclusions: The detailed study of ADS presented in this work will help to inform future clinical service delivery and clinical trial design.

[发布机构] University:University of Birmingham;Department:School of Health and Population Sciences

[关键词] R Medicine;R Medicine (General)