Quality of Life After Deep Brain Stimulation of Pediatric Patients with Dyskinetic Cerebral Palsy: A Prospective, Single-Arm, Multicenter Study with a Subsequent Randomized Double-Blind Crossover (STIM-CP).

BACKGROUND: Patients with dyskinetic cerebral palsy are often severely impaired with limited treatment options. The effects of deep brain stimulation (DBS) are less pronounced than those in inherited dystonia but can be associated with favorable quality of life outcomes even in patients without changes in dystonia severity. OBJECTIVE: The aim is to assess DBS effects in pediatric patients with pharmacorefractory dyskinetic cerebral palsy with focus on quality of life. METHODS: The method used is a prospective, single-arm, multicenter study. The primary endpoint is improvement in quality of life (CPCHILD [Caregiver Priorities & Child Health Index of Life with Disabilities]) from baseline to 12 months under therapeutic stimulation. The main key secondary outcomes are changes in Burke-Fahn-Marsden Dystonia Rating Scale, Dyskinesia Impairment Scale, Gross Motor Function Measure-66, Canadian Occupational Performance Measure (COPM), and Short-Form (SF)-36. After 12 months, patients were randomly assigned to a blinded crossover to receive active or sham stimulation for 24 hours each. Severity of dystonia and chorea were blindly rated. Safety was assessed throughout. The trial was registered at ClinicalTrials.gov, number NCT02097693. RESULTS: Sixteen patients (age: 13.4 ± 2.9 years) were recruited by seven clinical sites. Primary outcome at 12-month follow-up is as follows: mean CPCHILD increased by 4.2 ± 10.4 points (95% CI [confidence interval] -1.3 to 9.7; P = 0.125); among secondary outcomes: improvement in COPM performance measure of 1.1 ± 1.5 points (95% CI 0.2 to 1.9; P = 0.02) and in the SF-36 physical health component by 5.1 ± 6.2 points (95% CI 0.7 to 9.6; P = 0.028). Otherwise, there are no significant changes. CONCLUSION: Evidence to recommend DBS as routine treatment to improve quality of life in pediatric patients with dyskinetic cerebral palsy is not yet sufficient. Extended follow-up in larger cohorts will determine the impact of DBS further to guide treatment decisions in these often severely disabled patients. © 2021 The Authors. Movement Disorders published by Wiley Periodicals LLC on behalf of International Parkinson and Movement Disorder Society.

Pallidal stimulation in children: comparison between cerebral palsy and DYT1 dystonia.

The authors compared the outcomes of 17 children aged 7 to 15 years with DYT1 dystonia or cerebral palsy following deep brain stimulation. While patients with cerebral palsy presented with significantly greater motor disability than the DYT1 cohort at baseline, both groups demonstrated improvement at 1 year (cerebral palsy = 24%; DYT1 = 6%). The group as a whole demonstrated significant improvement on the Barry-Albright Dystonia Scale across time. Gains in motor function were apparent in both axial and appendicular distributions involving both upper and lower extremities. Gains achieved by 6 months were sustained in the cerebral palsy group, whereas the DYT1 group demonstrated continued improvement with ongoing pallidal stimulation beyond 18 months. Young patients with dystonia due to cerebral palsy responded comparably to patients with DYT1 dystonia. The severity of motor impairment in patients with cerebral palsy at baseline and follow-up raises the issue of even earlier intervention with neuromodulation in this population to limit long-term motor impairments due to dystonia.

Dystonia due to cerebral palsy responds to deep brain stimulation of the globus pallidus internus.

BACKGROUND: Cerebral palsy is the most common cause of pediatric-onset dystonia. Deep brain stimulation is gaining acceptance for treating dystonias in children. There is minimal reported experience regarding the efficacy of deep brain stimulation in cerebral palsy. METHODS: Fourteen patients, including 8 younger than 16 years, received bilateral implants (13 patients) or a unilateral implant (1 patient) of the internal globus pallidus and were observed in a noncontrolled, nonblinded study for at least 6 months. Motor function was assessed using the Burke-Fahn-Marsden Dystonia Movement and Disability scales and the Barry Albright Dystonia Scale. RESULTS: By 6 months, significant improvement was observed in the Burke-Fahn-Marsden Dystonia Movement scale (P=.004), the Burke-Fahn-Marsden Dystonia Disability scale (P=.027), and the Barry Albright Dystonia Scale (P=.029) for the whole cohort (n=14) and in the patients treated before skeletal maturity (group 1; n=8): Burke-Fahn-Marsden Dystonia Movement scale, P=.012; Burke-Fahn-Marsden Dystonia Disability scale, P=.020; and Barry Albright Dystonia Scale, P=.027. CONCLUSIONS: Deep brain stimulation may offer an effective treatment option for cerebral palsy-related dystonia, especially in those treated before skeletal maturity.