BEST- A Multicenter Retrospective Chart Review of the Pediatric Population Using Oral Baclofen to Manage the Spasticity of Cerebral Palsy (BEST-CHART)

Baclofen (generic name) or Lioresal (brand name used for intrathecal use; newer brand name oral formulations- 25 mg oral tablet and 10mg or 20mg disintegrating tablets) is a skeletal muscle relaxant that has been approved by the Food and Drug Administration (FDA) for use in adults with multiple sclerosis and spinal cord injuries. Baclofen has been shown to reduce spasticity and painful muscle spasms in adults although data are limited and somewhat inconsistent. Some reports suggest that baclofen may be more effective in patients with spasticity of spinal origin rather than cerebral origin. Far less data are available on the use of this medication in children.

This is a multicenter retrospective chart review of pediatric patients started on oral baclofen as a treatment for spasticity of cerebral palsy. The charts of approximately 200 (203-210) pediatric patients will be reviewed across seven investigative sites. This chart review will be conducted in patients with cerebral palsy with the current age of 3-17 years inclusive (at the start of the chart review) who have attended the outpatient cerebral palsy clinics at one of the seven investigative sites for at least one year. This will yield data on patients started on baclofen between the ages of 2-16 years. The sites will review a total of 200 patient charts (29-30 patients per site) who are currently on oral baclofen or 5 years prior to the chart review. There is no evidence that spasticity of cerebral palsy occurs more commonly or frequently in one gender, or that there is a greater prevalence in specific ethnic or racial groups. Based on the estimates obtained from the seven clinical sites, scattered throughout the United States, a diverse ethnic and racial mix should be obtained in the study sample.

Charts will be selected by obtaining a retrospective series of sequential lists of all patients who arrived for their cerebral palsy outpatient clinic visit, beginning with the clinic date immediately preceding the start date of the chart review and working backwards in time until sufficient charts have been obtained to complete the study. The start date of the chart review will be determined by the primary site once all sites have IRB approval for study initiation. Patient lists from each successive clinic visit prior to the first eligible clinic visit will be obtained and every chart from these lists will be screened in order to identify those that meet study criteria for inclusion. For eligible subjects, charts from each of their clinic visits up to 5 years before the study initiation date, will be reviewed and data recorded. The screening process at each site will terminate when 29- 30 charts that qualify for the full review have been identified.

The primary study objectives for this multisite project are to: 1) describe the characteristics of the pediatric population using oral baclofen to manage the spasticity of cerebral palsy, including age at onset of taking the drug, severity of spasticity, and level of functional mobility, 2) describe the safety of oral baclofen in 200 pediatric patients using oral baclofen (under the assumption that at least 50% will use oral baclofen for a full year or more), 3) describe treatment course and reason for treatment changes (from oral baclofen to other therapies, additional therapies, etc), withdrawal, and adverse events associated with treatment and withdrawal, 4) estimate the patient population size needed to result in a sample of 100 children using oral baclofen continuously for one year or more (in preparation for a safety study), and 5)provide any information detailed in the charts that may support the effectiveness of baclofen for reducing spasticity or improving function or care that can potentially be used as preliminary data for an efficacy trial.