Development and refinement of the Clinical Global Impression of Improvement for Non-seizure Symptoms measure in Dravet syndrome and Lennox-Gastaut syndrome.

ABSTRACT:

Background

Dravet syndrome (DS) and Lennox-Gastaut syndrome (LGS) are rare, severe, childhood-onset developmental and epileptic encephalopathies characterized by treatment-resistant epilepsy and varying intellectual disability levels. Clinical outcome assessments (COAs) describe how patients feel, function, or survive, thus providing valuable information on a therapy's efficacy and impact. Individuals with DS or LGS are heterogeneous, and many have limited verbal abilities and intellectual disability. Existing epilepsy-specific COA measures are unsuitable for DS and LGS clinical trials as many items demonstrate floor effects in these populations. As patients often cannot self-report symptoms, caregiver feedback on the measures' relevance and understandability is critical when devel

SUBMITTER: Andrews JS 

PROVIDER: S-EPMC11845656 | biostudies-literature | 2025 Feb

REPOSITORIES: biostudies-literature