Developmental medicine and child neurology
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Dev Med Child Neurol · Nov 2013
Detecting meaningful change using the North Star Ambulatory Assessment in Duchenne muscular dystrophy.
Clinician-reported outcome instruments such as the North Star Ambulatory Assessment (NSAA) need to be able to detect clinically important change to be suitable for clinical trials. However, in Duchenne muscular dystrophy (DMD), identifying changes in function is not straightforward. In this study, we use Rasch-transformed data to examine the responsiveness and minimal important difference (MID) of the NSAA in males with DMD receiving different corticosteroid regimes. ⋯ This study, based on Rasch-transformed NSAA data, provides an initial basis for the interpretation of clinical change in DMD over time and between corticosteroid regimes. Our proposed MIDs can be mapped back to differences in specific item content across the range of the NSAA.
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Dev Med Child Neurol · Nov 2013
Comparative StudyComparison of Bayley-2 and Bayley-3 scores at 18 months in term infants following neonatal encephalopathy and therapeutic hypothermia.
Neuroprotection trials for neonatal encephalopathy use moderate or severe disability as an outcome, with the Bayley Scales of Infant Development, Second Edition (Bayley-2) Index scores of <70 as part of the criteria. The Bayley Scales of Infant and Toddler, 3rd Development, Third Edition (Bayley-3) have superseded Bayley-2 and yield higher than expected scores in typically developing and high-risk infants. The aim of this study, therefore, was to compare Bayley-2 scores and Bayley-3 scores in term-born infants surviving neonatal encephalopathy treated with hypothermia. ⋯ Fewer children were classified with severe delay using the Bayley-3 than the Bayley-2, which prohibits direct comparison of scores. Increased Bayley-3 cut-off thresholds for classifying severe disability are recommended when comparing studies in this clinical group using Bayley-2 scores.
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Dev Med Child Neurol · Jul 2013
Diffusion-weighted imaging changes in cerebral watershed distribution following neonatal encephalopathy are not invariably associated with an adverse outcome.
Patterns of injury in term-born infants with neonatal encephalopathy following hypoxia-ischaemia are seen earlier and are more conspicuous on diffusion-weighted magnetic resonance imaging (DW-MRI) than on conventional imaging. Although the prognostic value of DW-MRI in infants with basal ganglia and thalamic damage has been established, data in infants in whom there is extensive injury in a watershed distribution are limited. The aim of this study was to assess cognitive and functional motor outcome in a cohort of infants with changes in a predominantly watershed distribution injury on neonatal cerebral MRI, including DWI. ⋯ Extensive DWI changes in a watershed distribution in term-born neonates are not invariably associated with adverse sequelae, even in the presence of cystic evolution. Associated lesions of the basal ganglia and thalami are a better predictor of adverse sequelae than the extent and severity of the watershed abnormalities seen on DW-MRI.
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Dev Med Child Neurol · Jul 2013
Characteristics of post-traumatic headaches in children following mild traumatic brain injury and their response to treatment: a prospective cohort.
Post-traumatic headaches (PTHs) following mild traumatic brain injury (mTBI) are common; however, few studies have examined the characteristics of PTHs or their response to treatment. The aims of this study were (1) to describe the clinical characteristics of PTH in a prospective cohort of children presenting to a paediatric emergency department with mTBI, and (2) to evaluate the response of PTH to treatment. ⋯ This is the first prospective cohort study to describe the clinical characteristics of PTHs following mTBI in children. Migraine was the most common headache type seen; other headaches included tension-type, cervicogenic, and occipital neuralgias, and 64% responded to treatment. Referral to a headache specialist should be considered, especially when the features are not typical of one of the primary headache disorders.