Rationale:
Although Tuberous Sclerosis Complex-related epilepsy is often medically refractory, little is known about the most effective anti-seizure medications in this population. Here, we queried standardized electronic health record data from 17 centers in the Pediatric Epilepsy Learning Healthcare System data coordinating center to understand anti-seizure medication prescription patterns for children with TSC-related epilepsy and identify sources of practice variation and potential comparative effectiveness questions for further research.
Methods:
Study Design: We conducted a retrospective study of children with TSC at 17 centers to characterize patterns of anti-seizure medication prescription and to develop and field test quality measures. The study was approved by the IRB at all centers, facilitated by a central IRB protocol (Biomedical Research Alliance of New York, Lake Success NY).
Data Source: The PEHLS data coordinating center includes electronic health record data on children with epilepsy from 17 US academic medical centers. Data are stored using limited PHI and include information about demographics, healthcare encounters, diagnosis codes, procedure codes, prescribed medications, administered medications.
Cohort Generation: Inclusion criteria included CPT codes 1) epilepsy (ICD9 345 or ICD10 G40) and 2) TSC (ICD9 759.5 or ICD10 Q85. For a proxy for the captured TSC population, we included patients with at least six years of data with at least one visit in the last 24 months. Data included for each patient was anti-seizure name and age when prescribed.
Results:
One thousand five hundred forty nine (1549) patients with a diagnosis of TSC and epilepsy. The median age of first medication in this population was 4.53 years. For each ASM, we looked at the first outpatient prescription by age for all centers (Figure 1). These data reflect expected age-related differences in their use. For example, ACTH and phenobarbital less often and at a younger LTG Vigabatrin shows a high rate of use in infants and young children, reflecting its use in TSC-related infantile spasms but can be started in older children or even adulthood. Figure 2 shows the percentage of patients at each center which have had a prescription for each medication. We highlight two of the 17 centers for emphasis. Unsurprisingly, the most frequently prescribed ASM across all centers is levetiracetam followed by oxcarbazepine.
Conclusions:
By using our large, multicenter database of pediatric epilepsy centers we highlight significant variability in ASM prescription patterns in TSC-related epilepsy. Our data highlights the need for further comparative effectiveness studies, for example, in levetiracetam and oxcarbazepine, to understand which treatments within TSC which could lead to better outcomes for our patients. We also demonstrated the potential of large-scale data extraction in the study of disease-specific and rare epilepsies using the existing PELHS infrastructure.
Funding:
Pediatric Epilepsy Research Foundation, Morris & Alma Schapiro Fund, BAND Foundation, Weill Cornell Medicine, University of Virginia