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.

Neuromotor actions in PPT remained largely stable over time. Specific educational actions and communication increased, indicating larger family involvement during treatment sessions.

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 greatly The effect of DBS on functional priorities did not correlate with motor outcome Half of the motor ‘non-responder’ patients reported important changes in their priorities The effect of DBS in dystonia should not be measured by motor outcome alone