Cannabidiol Treatment for Neurological, Cognitive, and Psychiatric Symptoms in Sturge-Weber Syndrome.

BACKGROUND: A prior drug trial of cannabidiol for treatment-resistant epilepsy in patients with Sturge-Weber syndrome (SWS), a rare neurovascular condition, implicated improvements in neurological, quality of life (QOL), neuropsychologic, psychiatric, and motor outcomes. METHODS: Ten subjects with SWS brain involvement, controlled seizures, and cognitive impairments received study drug in this Johns Hopkins institutional review board-approved, open-label, prospective drug trial. Oral cannabidiol was taken for six months (dose ranged from 5 to 20 mg/kg/day). SWS neuroscore, port-wine birthmark score, QOL, and adverse events were recorded every four to 12 weeks. Neuropsychologic, psychiatric, and motor assessments were administered at baseline and six months' follow-up. Most evaluations were conducted virtually due to the coronavirus disease 2019 pandemic. RESULTS: Cannabidiol was generally well tolerated. Six subjects reported mild to moderate side effects related to study drug and continued on drug; one subject withdrew early due to moderate side effects. No seizures were reported. Significant improvements in SWS neuroscore, patient-reported QOL, anxiety and emotional regulation, and report of bimanual ability use were noted. Migraine QOL scores were high at baseline in these subjects, and remained high. Neuropsychologic and other QOL and motor outcomes remained stable, with some within-subject improvements noted. CONCLUSIONS: Further studies are needed to determine whether Epidiolex can improve quality of life and be beneficial for neurological, anxiety, and motor impairments in SWS independent of seizure control. Large multicentered studies are needed to extend these preliminary findings.

Hybrid Telehealth Pediatric Constraint Induced Movement Therapy Compared to In-person Intervention: A Case Series.

Constraint induced movement therapy is an established, evidence-based intervention for children with hemiplegia. This case series describes the feasibility and clinical opportunities of using a hybrid telehealth and onsite model to deliver pediatric constraint induced movement therapy during the COVID-19 pandemic. These cases support that a hybrid model had a similar effect on upper extremity improvement compared to a traditional, in-person model and may be an option when access to in-person care is not available.

Speech production gains following constraint-induced movement therapy in children with hemiparesis.

PURPOSE: The purpose of this study was to investigate changes in speech skills of children who have hemiparesis and speech impairment after participation in a constraint-induced movement therapy (CIMT) program. While case studies have reported collateral speech gains following CIMT, the effect of CIMT on speech production has not previously been directly investigated to the knowledge of these investigators. METHODS: Eighteen children with hemiparesis and co-occurring speech impairment participated in a 21-day clinical CIMT program. The Goldman-Fristoe Test of Articulation-2 (GFTA-2) was used to assess children's articulation of speech sounds before and after the intervention. Changes in percent of consonants correct (PCC) on the GFTA-2 were used as a measure of change in speech production. RESULTS: Children made significant gains in PCC following CIMT. Gains were similar in children with left and right-sided hemiparesis, and across age groups. CONCLUSION: This study reports significant collateral gains in speech production following CIMT and suggests benefits of CIMT may also spread to speech motor domains.

Infant constraint induced movement therapy: Lessons learned from clinical implementation.

Current evidence supports the efficacy of pediatric constraint induced movement therapy (CIMT) for toddlers and children but little has been published about its use in early intervention with children eighteen months and younger. This paper and case report describes the clinical experience of developing and executing a modified pediatric CIMT intervention with infants under 18 months old. This is an emerging area of practice that is showing positive trends with initial cases.

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

PURPOSE: 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). METHODS: 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). RESULTS: Inter-rater reliability was high across all measures. Correlations were identified between the SWS-NRS and the study measures. CONCLUSIONS: 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.

Outcomes of a clinic-based pediatric constraint-induced movement therapy program.

A single-group pre- and post-test design was used to evaluate functional outcomes of a constraint-induced movement therapy (CIMT) protocol implemented in an outpatient therapy center. The participants were 29 children with hemiplegia, ages 1.6-19.1 years old. The less-involved upper limb was placed in a cast that was worn 24 hr a day, 7 days a week. Individual therapy sessions took place 5 days/week. Children received 3 or 6 hr therapy sessions for 16-19 days followed by 2-5 days in which bimanual tasks were performed. Outcomes were assessed at baseline and following CIMT. Statistically significant gains were made on the Melbourne Assessment of Unilateral Upper Limb Function, Quality of Upper Extremity Skills Test (except the Protective Extension subtest), Assisting Hand Assessment, and the Canadian Occupational Performance Measure. The effect sizes varied from 0.46 to 0.70 indicating a moderate effect size. The results support the effectiveness of CIMT provided through a center-based program.