Dystonic Cerebral Palsy Clinical Trial
Background:
Cerebral palsy (CP) is the main cause of childhood immobility and is defined as a non
progressive injury to the developing central nervous system in children younger than 3
years, resulting in neurological and musculoskeletal abnormalities. The main
pathophysiological causes are encephalopathy of prematurity (periventricular leukomalacia)
hypoxic ischemic encephalopathy. Infections, infracts and migration defects are other less
common causes of CP. The brain injury leads to functional motor impairment impacting on
daily activities commonly manifests as a movement disorder: pyramidal, leading to spasticity
and extra-pyramidal leading to dystonia and chorea. In most cases extensive brain injury
causes a mixed movement disorder. Dystonia is defined as involuntary muscle contractions
causing twisting and abnormal postures. While the neurological underpinnings of CP remain
unknown, a link between low dopamine and increased acetylcholine release has recently been
reported in dystonia. Dopamine is considered the first line of treatment in children with
dystonia and CP followed by anticholiergic treatment with trihexphenidyl. The recommendation
of dopaminergic treatment is based on need to rule out dopamine-responsive-dystonia, a rare
genetic disorder, and on single case study reporting improvement in CP. A double blind study
support or refute the use of dopamine treatment for dystonic CP was never reported. Working
hypothesis and
Aims:
In children with CP due to a clear underlying pathology, dopamine treatment will not improve
daily function. Methods: the investigators will perform a double blinded randomized
controlled crossover study. 50 children ages 4-18 years with a clear pathophysiological
cause for CP will be enrolled. Each child will receive dopamine and placebo treatment for 2
weeks with a 2 week washout interval. Participants will be randomized into 2 groups; one
will receive placebo followed by dopamine and the other vice versa. The primary outcome
measure, goal-attainment-scale, and secondary outcome functional measures (such as box and
blocks, 9 hole pegs, pronation/ supination, finger sequencing) will be assessed at the
beginning and end of each treatment as well as parent questionnaires regarding satisfaction
and side effects.
Expected results:
No functional improvement with dopamine treatment compared to placebo.
Importance:
supplying sufficient data to support or refute the use of dopamine treatment for dystonic
CP.
Probable implications to Medicine:
this may lead to a change in medical treatment guidelines for children with CP.
n/a
Allocation: Randomized, Intervention Model: Crossover Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment
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