Childhood Hypertonia of Central Origin: A Trial of Anticholinergic Treatment Effects
This study is an open-label trial of trihexyphenidyl in children with upper extremity dystonia due to cerebral palsy. It is hypothesized that trihexyphenidyl in doses up to 0.75mg/kg/day would be well-tolerated and show significant changes on the Melbourne scale of upper extremity function.
|Study Design:||Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||Childhood Hypertonia of Central Origin: An Open Label Trial of Anticholinergic Treatment Effects|
- Melbourne assessment of upper extremity function
- Barry-Albright Dystonia Scale
- Burke-Fahn-Marsden Dystonia Scale
- Pediatric Outcomes Data Collection Instrument
- Pediatric Quality of Life
- Gross Motor Function Measure
|Study Start Date:||January 2003|
|Estimated Study Completion Date:||December 2004|
BACKGROUND: Although trihexyphenidyl has been used to treat both primary and secondary dystonia in children, previous studies have not investigated efficacy in secondary dystonia. We describe the results of a prospective, open-label, multi-center trial of high-dose trihexyphenidyl in children with secondary dystonia of the arms due to cerebral palsy.
METHODS: Twenty-six children age 4-15 years with cerebral palsy and dystonia that impairs function of the dominant upper extremity were enrolled. All children were given trihexyphenidyl at increasing doses over 9 weeks up to 0.75mg/kg/day. Trihexyphenidyl was subsequently tapered over 5 weeks. Visits occurred at baseline, 9 weeks, and 15 weeks. The primary outcome measure was the Melbourne assessment of upper extremity function, tested in the dominant arm.
RESULTS: Three children withdrew due to non-serious adverse events (chorea, drug rash, hyperactivity). 3 children reduced dosage due to non-serious adverse events. The 23 children who completed the study showed a significant improvement in arm function at 15 weeks (p=0.045) but not at 9 weeks. Post-hoc analysis showed that a subgroup (N=10) with hyperkinetic dystonia worsened at 9 weeks (p=0.04) but subsequently returned to baseline following taper of the medicine.
CONCLUSIONS: Trihexyphenidyl appears to be safe and effective for treatment of arm dystonia in children with cerebral palsy. Children with hyperkinetic dystonia may worsen. A larger randomized prospective trial is needed to confirm these results.
|United States, Alabama|
|University of Alabama School of Medicine|
|Birmingham, Alabama, United States, 35233|
|United States, California|
|Stanford, California, United States, 94305-5235|
|United States, Illinois|
|Rehabilitation Institute of Chicago|
|Chicago, Illinois, United States, 60611|
|United States, Maryland|
|Kennedy Krieger Institute|
|Baltimore, Maryland, United States, 21205|
|United States, Missouri|
|Washington University School of Medicine|
|St. Louis, Missouri, United States, 63110|
|United States, New York|
|University of Rochester Medical Center|
|Rochester, New York, United States, 14642|
|United States, Texas|
|Texas Scottish Rite Hospital for Children|
|Dallas, Texas, United States, 75219|
|Principal Investigator:||Terence D Sanger, MD, PhD||Stanford University|