The Effectiveness of Deep Brain Stimulation in Dystonia: A Patient-Centered Approach.

Background: To systematically evaluate the effectiveness of deep brain stimulation of the globus pallidus internus (GPi-DBS) in dystonia on pre-operatively set functional priorities in daily living. Methods: Fifteen pediatric and adult dystonia patients (8 male; median age 32y, range 8-65) receiving GPi-DBS were recruited. All patients underwent a multidisciplinary evaluation before and 1-year post DBS implantation. The Canadian Occupational Performance Measure (COPM) first identified and then measured changes in functional priorities. The Burke-Fahn-Marsden Dystonia Rating Scale (BFMDRS) was used to evaluate dystonia severity. Results: Priorities in daily functioning substantially varied between patients but showed significant improvements on performance and satisfaction after DBS. Clinically significant COPM-score improvements were present in 7/8 motor responders, but also in 4/7 motor non-responders. Discussion: The use of a patient-oriented approach to measure GPi-DBS effectiveness in dystonia provides an unique insight in patients' priorities and demonstrates that tangible improvements can be achieved irrespective of motor response. Highlights: Functional priorities in life of dystonia patients and their caregivers vary greatlyThe effect of DBS on functional priorities did not correlate with motor outcomeHalf of the motor 'non-responder' patients reported important changes in their prioritiesThe effect of DBS in dystonia should not be measured by motor outcome alone.

Motor and non-motor determinants of health-related quality of life in young dystonia patients.

OBJECTIVES: To systematically investigate the relationship between motor and non-motor symptoms, and health-related quality of life (HR-QoL) in children and young adults with dystonia. METHODS: In this prospective observational cross-sectional study, 60 patients (6-25 years) with childhood-onset dystonia underwent a multidisciplinary assessment of dystonia severity (Burke-Fahn-Marsden Dystonia Rating Scale, Global Clinical Impression), motor function (Gross Motor Function Measure, Melbourne Assessment of Unilateral Upper Limb Function), pain (visual analogue scale), intelligence (Wechsler Intelligence Scale), executive functioning (Behavior Rating Inventory of Executive Function) and anxiety/depression (Child/Adult Behavior Checklist). Measures were analyzed using a principal component analysis and subsequent multiple regression to evaluate which components were associated with HR-QoL (Pediatric Quality of life Inventory) for total group, and non-lesional (primary) and lesional (secondary) subgroups. RESULTS: Patients (29 non-lesional, 31 lesional dystonia) had a mean age of 13.6 ±â€¯5.9 years. The principal component analysis revealed three components: 1) motor symptoms; 2) psychiatric and behavioral symptoms; and 3) pain. HR-QoL was associated with motor symptoms and psychiatric and behavioral symptoms (R2 = 0.66) for the total sample and lesional dystonia, but in the non-lesional dystonia subgroup only with psychiatric and behavioral symptoms (R2 = 0.51). CONCLUSIONS: Non-motor symptoms are important for HR-QoL in childhood-onset dystonia. We suggest a multidisciplinary assessment of motor and non-motor symptoms to optimize individual patient management.

Changes in the Content of Pediatric Physical Therapy for Infants: A Quantitative, Observational Study.

AIMS: The aim of our observational longitudinal study is to evaluate changes over time in standard pediatric physical therapy (PPT) for infants at risk of neurodevelopmental disorders. METHODS: Treatment sessions in two time periods (2003-2005 [n = 22] and 2008-2014 [n = 16]) were video recorded and analyzed quantitatively in five categories: neuromotor actions, educational actions, communication, position, and situation of treatment session. Differences in percentages of time spent on therapeutic actions between periods were tested with Mann-Whitney U and Hodges Lehmann's tests. RESULTS: No significant changes appeared in the main categories of neuromotor actions. Time spent on not-specified educational actions toward caregivers (median from 99% to 81%, p = .042) and not-specified communication (median from 72% to 52%, p = .002) decreased. Consequently, time spent on specific educational actions (caregiver training and coaching; median from 1% to 19%, p = .042) and specific communication (information exchange, instruct, provide feedback; median from 21% to 38%, p = .007) increased. Infant position changed only minimally: time spent on transitions-that is, change of position-decreased slightly over time (median from 7% to 6%, p = .042). Situation of treatment session did not change significantly over time. CONCLUSIONS: Neuromotor actions in PPT remained largely stable over time. Specific educational actions and communication increased, indicating larger family involvement during treatment sessions.

Effect of early intervention on functional outcome at school age: Follow-up and process evaluation of a randomised controlled trial in infants at risk.

BACKGROUND: The long-term effect of early intervention in infants at risk for developmental disorders is unclear. The VIP project (n=46, originally) evaluated by means of a randomised controlled trial the effect of the family centred early intervention programme COPCA (Coping with and Caring for infants with special needs) in comparison to that of traditional infant physiotherapy (TIP). AIMS: To evaluate the effect of early intervention on functional outcome at school age. METHODS AND PROCEDURES: Parents of 40 children (median age 8.3years) participated in this follow-up study. Outcome was assessed with a standardised parental interview (Vineland Adaptive Behaviour Scale) and questionnaires (Developmental Coordination Disorder Questionnaire, Child Behaviour Checklist, Utrechtse Coping List, and questions on educational approach). Quantified video information on physiotherapeutic actions during infancy was available. OUTCOMES AND RESULTS: Child functional outcome in the two randomised groups was similar. Process evaluation revealed that some physiotherapeutic actions were associated with child mobility and parental educational approach at follow-up: e.g., training and instructing were associated with worse mobility. CONCLUSIONS AND IMPLICATIONS: Functional outcome at school age after early intervention with COPCA is similar to that after TIP. However, some specific physiotherapeutic actions, in particular the physiotherapist's approach, are associated with outcome. WHAT THIS PAPER ADDS: Early intervention is generally applied in infants at risk for developing disorders, with the aim of improving overall functional outcome. However, little is known on the long-term effect. The VIP project evaluated by means of a randomised controlled trial the effect of the family centred early intervention programme COPCA (Coping with and Caring for infants with special needs) in comparison to that of traditional infant physical therapy (TIP). Outcome at 18months corrected age was virtually similar. Process evaluation of the physiotherapy actions revealed that some characteristics of COPCA were associated with improved developmental outcome at 18months. This paper presents data on functional outcome at school age (median 8.3years) in 87% of the original participants. Outcome of infants who received three months of COPCA and that of infants who received three months of TIP was similar. Yet, parents of families who had received the COPCA intervention still more often used a trial and error approach when the child learned a new skill than parents of children who had received TIP. Process evaluation showed that more time spent on caregiver training and strict instructions during early intervention was associated with worse mobility. Four other physiotherapeutic actions were associated with parental educational approach. None of the neuromotor actions were associated with child outcome at school age. We conclude that long-term outcome after three months of COPCA or TIP is similar. However, our study does suggest that the professional approach of the physiotherapist can make a difference.