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Preliminary reliability and validity of a battery for assessing functional skills in children with Sturge-Weber syndrome.

ABSTRACT: The purpose of this study was to evaluate inter-rater reliability and validity of a proposed functional outcome battery for clinical trials in children with Sturge-Weber syndrome (SWS).Ten children were evaluated twice on the same day using a series of functional outcome measures selected for sensitivity to the range of age and function of children with SWS: Modified Rankin Scale, Pediatric Evaluation of Disability Index, Modified House Functional Classification, and a modified version of the Erhardt Developmental Prehension Assessment. Inter-rater reliability was calculated, and criterion validity was explored through correlations with the Sturge-Weber Syndrome-Neurological Rating Score (SWS-NRS).Inter-rater reliability was high across all measures. Correlations were identified between the SWS-NRS and the study measures.The proposed battery of functional outcome measures captures child's functioning at the levels of impairment, activity and participation and is robust to evaluation by different raters and across sessions on the same day. This battery is expected to be sensitive to treatment-related changes in qualitative patterns of hand use, functional skills, and/or change in independence in daily living.

SUBMITTER: Reidy TG

PROVIDER: S-EPMC4276129 | biostudies-literature | 2014 Dec

REPOSITORIES: biostudies-literature

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Preliminary reliability and validity of a battery for assessing functional skills in children with Sturge-Weber syndrome.

Reidy Teressa Garcia TG Suskauer Stacy J SJ Bachur Cathy D CD McCulloch Charles E CE Comi Anne M AM

Child's nervous system : ChNS : official journal of the International Society for Pediatric Neurosurgery 20141026 12

<h4>Purpose</h4>The purpose of this study was to evaluate inter-rater reliability and validity of a proposed functional outcome battery for clinical trials in children with Sturge-Weber syndrome (SWS).<h4>Methods</h4>Ten children were evaluated twice on the same day using a series of functional outcome measures selected for sensitivity to the range of age and function of children with SWS: Modified Rankin Scale, Pediatric Evaluation of Disability Index, Modified House Functional Classification, ...[more]

PMID: 25344741

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Project description:AIM:We assessed the utilization of the National Institutes of Health Quality of Life in Neurological Disorders (Neuro-QoL) in pediatric patients with Sturge-Weber syndrome, a rare neurovascular disorder which frequently results in seizures, brain atrophy, calcification, and a range of neurological impairments. METHODS:Subjects were seen clinically and consented for research. All 22 patients filled out the Pediatric Neuro-QoL. The Neuro-QoL subscores were converted to T-scores to compare with the referenced control population. Twenty-one participants also filled out the Brain Vascular Malformation Consortium Database Questionnaire containing data pertaining to Sturge-Weber syndrome-related medical history, medications, comorbidities, and family history. All data were analyzed with a significance threshold of P < 0.05. RESULTS:Cognitive function quality of life was significantly lower (P < 0.001) in pediatric patients with Sturge-Weber syndrome compared with referenced control subjects. Male gender (P = 0.02) was associated with lower cognitive function Neuro-QoL. The extent of skin (R = -0.46, P = 0.04), total eyelid port-wine birthmark (R = -0.56, P = 0.007), eye (R = -0.58, P = 0.005), and total Sturge-Weber syndrome involvement (R = -0.63, P = 0.002) were negatively correlated with cognitive function Neuro-QoL. A younger age at seizure onset was associated with lower cognitive function Neuro-QoL (hazard ratio = 0.90, P = 0.004) even after controlling for extent of brain, skin, or eye involvement. Antidepressant use was associated with lower cognitive function Neuro-QoL (P = 0.005), and cognitive function Neuro-QoL was negatively correlated with depression Neuro-QoL; however, after adjusting for depression this relationship was no longer significant. CONCLUSIONS:The results suggest targeting cognitive function Neuro-QoL in treatment trials and reiterate the prognostic value of early seizure onset. In addition, sex-related differences were noted, which should be further studied.

Project description:BackgroundEnlarged deep medullary veins (EDMVs) in patients with Sturge-Weber syndrome (SWS) may channel venous blood from the surface to the deep vein system in brain regions affected by the leptomeningeal venous malformation. Thus, the quantification of EDMV volume may provide an objective imaging marker for this vascular compensatory process. The present study proposes a novel analytical method to quantify enlarged EDMV volumes in the affected hemisphere of patients with unilateral SWS.MethodsTwenty young subjects, including 10 patients with unilateral SWS and 10 healthy siblings (age 14.5±6.7 and 16.0±7.0 years, respectively) underwent 3T brain MRI scanning using susceptibility-weighted imaging (SWI) and volumetric T1-weighted sequences. The proposed image analytic steps segmented EDMVs in white matter regions, defined on the volumetric T1-weighted images, by statistically associating the likelihood of intensity, location, and tubular shape on SWI. The volumes of the segmented EDMVs, calculated in each hemisphere, were compared between affected and unaffected hemispheres. EDMV volumes were also correlated with visually assessed EDMV scores, hemispheric white matter volumes, and cortical surface areas. Parametric tests including Pearson's correlation, unpaired and paired t-tests, were used. A P value <0.05 was considered statistically significant.ResultsIt was found that EDMVs were identified well in SWS-affected hemispheres while calcified regions were excluded. Mean EDMV volumes in the SWS-affected hemispheres were 10-12-fold greater than in the unaffected or healthy control hemispheres; while white matter volumes and cortical surface areas were lower. EDMV volumes in the SWS-affected hemispheres showed a strong positive correlation with the visual EDMV scores (r=0.88, P=0.001) and an inverse correlation with cortical surface area ratios (r=-0.65, P=0.04) but no correlation with white matter volume ratios.ConclusionsEDMVs were detected in the SWS-affected atrophic hemispheres reliably while avoiding calcified regions. The approach can be used to quantify enlarged deep cerebral veins in the human brain, which may provide a potential marker of cerebral venous remodeling.

Dataset Information

Preliminary reliability and validity of a battery for assessing functional skills in children with Sturge-Weber syndrome.

Publications

Preliminary reliability and validity of a battery for assessing functional skills in children with Sturge-Weber syndrome.

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