Bilateral pallidal deep brain stimulation for the treatment of patients with dystonia-choreoathetosis cerebral palsy: a prospective pilot study.
BACKGROUND
Cerebral palsy (CP) with dystonia-choreoathetosis is a common cause of disability in children and in adults, and responds poorly to medical treatment. Bilateral pallidal deep brain stimulation (BP-DBS) of the globus pallidus internus (GPi) is an effective treatment for primary dystonia, but the effect of this reversible surgical procedure on dystonia-choreoathetosis CP, which is a subtype of secondary dystonia, is unknown. Our aim was to test the effectiveness of BP-DBS in adults with dystonia-choreoathetosis CP.


METHODS
We did a multicentre prospective pilot study of BP-DBS in 13 adults with dystonia-choreoathetosis CP who had no cognitive impairment, little spasticity, and only slight abnormalities of the basal ganglia on MRI. The primary endpoint was change in the severity of dystonia-choreoathetosis after 1 year of neurostimulation, as assessed with the Burke-Fahn-Marsden dystonia rating scale. The accuracy of surgical targeting to the GPi was assessed masked to the results of neurostimulation. Analysis was by intention to treat.


RESULTS
The mean Burke-Fahn-Marsden dystonia rating scale movement score improved from 44.2 (SD 21.1) before surgery to 34.7 (21.9) at 1 year post-operatively (p=0.009; mean improvement 24.4 [21.1]%, 95% CI 11.6-37.1). Functional disability, pain, and mental health-related quality of life were significantly improved. There was no worsening of cognition or mood. Adverse events were related to stimulation (arrest of the stimulator in one patient, and an adjustment to the current intensity in four patients). The optimum therapeutic target was the posterolateroventral region of the GPi. Little improvement was seen when the neurostimulation diffused to adjacent structures (mainly to the globus pallidus externus [GPe]).


CONCLUSIONS
Bilateral pallidal neurostimulation could be an effective treatment option for patients with dystonia-choreoathetosis CP. However, given the heterogeneity of motor outcomes and the small sample size, results should be interpreted with caution. The optimum placement of the leads seemed to be a crucial, but not exclusive, factor that could affect a good outcome.


BACKGROUND
National PHRC; Cerebral Palsy Foundation: Fondation Motrice/APETREIMC; French INSERM Dystonia National Network; Medtronic.
Vidailhet
Marie
M
AP-HP, Groupe hospitalier Pitié-Salpêtrière, Fédération de Neurologie, Paris, F-75013, France. marie.vidailhet@psl.aphp.fr
Yelnik
Jerome
J
Lagrange
Christelle
C
Fraix
Valerie
V
Grabli
David
D
Thobois
Stephane
S
Burbaud
Pierre
P
Welter
Marie-Laure
ML
Xie-Brustolin
Jin
J
Braga
Maria-Clara Coelho
MC
Ardouin
Claire
C
Czernecki
Virginie
V
Klinger
Hélène
H
Chabardes
Stephan
S
Seigneuret
Eric
E
Mertens
Patrick
P
Cuny
Emmanuel
E
Navarro
Soledad
S
Cornu
Philippe
P
Benabid
Alim-Louis
AL
Le Bas
Jean-Francois
JF
Dormont
Didier
D
Hermier
Marc
M
Dujardin
Kathy
K
Blond
Serge
S
Krystkowiak
Pierre
P
Destée
Alain
A
Bardinet
Eric
E
Agid
Yves
Y
Krack
Paul
P
Broussolle
Emmanuel
E
Pollak
Pierre
P
D000328:Adult; D001264:Athetosis / Q000150:complications / Q000628:therapy*; D001479:Basal Ganglia / Q000473:pathology; D002547:Cerebral Palsy / Q000150:complications / Q000628:therapy*; D002819:Chorea / Q000150:complications / Q000628:therapy*; D046690:Deep Brain Stimulation / Q000379:methods*; D004185:Disability Evaluation; D004421:Dystonia / Q000150:complications / Q000628:therapy*; D005260:Female; D007839:Functional Laterality; D005917:Globus Pallidus / Q000502:physiology*; D006801:Humans; D008279:Magnetic Resonance Imaging / Q000379:methods; D008297:Male; D010865:Pilot Projects; D011446:Prospective Studies; D012720:Severity of Illness Index; D018709:Statistics, Nonparametric; D055815:Young Adult