Prescription patterns of antiseizure drugs in tuberous sclerosis complex (TSC)-associated epilepsy: a multicenter cohort study from Germany and review of the literature

Expert Rev Clin Pharmacol. 2021 Jun;14(6):749-760. doi: 10.1080/17512433.2021.1911643. Epub 2021 May 31.

Abstract

Objective: Seizures are a primary and early disease manifestation of Tuberous Sclerosis Complex (TSC). We aimed to describe the age-stratified patterns of antiseizure drug (ASD) treatments among children, adolescents, and adults with TSC in Germany. Additionally, we reviewed real-world and clinical study evidence regarding ASD utilization in patients with TSC.

Methods: We evaluated the pattern of routine ASD use and everolimus prescriptions based on a 2019 multicenter survey of 268 individuals with TSC-associated epilepsy. We contextualized the results with a structured review of real-world and clinical study evidence.

Results: TSC-associated epilepsy treatment comprises a wide variety of ASDs. In this German sample, the majority of patients were treated with polytherapy, and lamotrigine (34.7%), valproate (32.8%), oxcarbazepine (28.7%), vigabatrin (19.0%), and levetiracetam (17.9%) were identified as the most-commonly used ASDs. In addition, everolimus was used by 32.5% of patients. In adherence to current TSC guidelines, the disease-modifying ASD vigabatrin was widely used in children (58% below the age of 5 years), whereas treatment in adults did not necessarily reflect guideline preference for (partial) GABAergic ASDs.

Conclusions: The selection of ASDs for patients with TSC-associated epilepsy follows well-evaluated recommendations, including the guidelines regarding vigabatrin use in children. Several characteristics, such as the comparatively high frequency of valproate use and polytherapy, reflect the severity of TSC-associated epilepsy.

Keywords: Epilepsy; antiepileptic drug; mTOR inhibitor; medication; prescription pattern; rare disease; seizure; vigabatrin.

Publication types

  • Multicenter Study
  • Review

MeSH terms

  • Adolescent
  • Age Factors
  • Anticonvulsants / administration & dosage*
  • Child
  • Child, Preschool
  • Cohort Studies
  • Cross-Sectional Studies
  • Epilepsy / drug therapy*
  • Epilepsy / etiology
  • Everolimus / administration & dosage
  • Germany
  • Humans
  • Infant
  • Infant, Newborn
  • Practice Patterns, Physicians'
  • Tuberous Sclerosis / complications
  • Tuberous Sclerosis / drug therapy*
  • Young Adult

Substances

  • Anticonvulsants
  • Everolimus