Antiseizure Medication Treatment Patterns, Reasons for Disruption, and Side Effects for Patients with Dravet Syndrome or Lennox-Gastaut Syndrome: A Retrospective Analysis of US Physician Notes
Mei Lu1, Dave Iwanyckyj2, Sally W. Wade3, Pablo Racana2, Fernando Otalora2, Satish Rao1
1Takeda Pharmaceuticals USA, Inc., 2Amplity Health, 3Wade Outcomes Research and Consulting
Objective:
To characterize the unmet need and treatment paradigms patients with Dravet syndrome (DS) and Lennox-Gastaut syndrome (LGS) face.  
Background:
Patients with DS and LGS are refractory to treatment and experience inadequate seizure control and safety issues from polypharmacy with antiseizure medications (ASMs). 
Design/Methods:

Natural language processing (NLP) analyzed Amplity Insights’ database of transcribed medical records from US clinician-patient interactions. NLP identified patients with a DS or LGS diagnosis and ≥1 ASM. Patient characteristics, ASM use, treatment disruptions, side effects, safety monitoring, and non-ASM therapies were described.

Results:
NLP identified 166 patients with DS and 1063 patients with LGS between 2010 and 2022. Patients with data were predominantly White (DS: 95%; LGS: 90%) with a mean age at diagnosis of 1.1 (DS) and 2.1 (LGS) years. Combination ASM therapy was used by 67% and 58% of patients with DS and LGS, respectively. ASMs clobazam (DS: 54%; LGS: 49%) and levetiracetam (DS: 53%; LGS: 47%) were used by nearly half of the patients. Up to 58% (DS) and 51% (LGS) of patients received rescue ASMs. ASM treatment disruptions (DS: 39%; LGS: 28%) were frequently caused by switching (DS: 57%; LGS: 45%). Reasons for treatment disruption included side effects/adverse events/tolerability (DS: 57%; LGS: 53%) and insufficient efficacy (DS: 26%; LGS: 30%). For both groups, 23% of patients reported ASM side effects; most commonly drowsiness/somnolence/sedation (DS: 21%; LGS: 32%). Approximately 40% of patients with DS and LGS had blood-based laboratory testing. Many patients used non-ASM therapies, such as antipsychotics, antidepressants, or anxiolytics (DS: 10%; LGS: 17%). Other interventions included diet (DS: 23%; LGS: 16%), developmental delay therapy (DS: 16%; LGS: 14%), and surgery (DS: 1%; LGS: 8%).
Conclusions:
Available ASMs provide inadequate seizure control and have tolerability and safety issues. This real-world study suggests that patients may benefit from more effective and tolerable ASMs.
10.1212/WNL.0000000000205173