Q&A: Novel measure may better assess outcomes in Lennox-Gastaut, Dravet syndromes
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Clinical outcome assessments currently used for people with Lennox-Gastaut syndrome and Dravet syndrome were “not fit” for evaluating non-seizure outcomes, researchers said, prompting the development of a global clinical impression measure.
Lennox-Gastaut syndrome (LGS) and Dravet syndrome (DS) are rare developmental and epileptic encephalopathies that involve treatment-resistant seizures, as well as cognitive and neurological delays or declines, Mahnaz Asgharnejad, PharmD, vice president and global program leader at Takeda Pharmaceuticals, and colleagues wrote in a poster presented at the American Epilepsy Society’s annual meeting.
Healio spoke with Asgharnejad to learn more about the researchers’ development of a new global impression approach for measuring non-seizure symptoms in patients with LGS or DS.
Healio: What was the aim of your study?
Asgharnejad: To evaluate current clinical outcome assessments in use to measure non-seizure symptoms in patients with LGS or DS.
Healio: What did you and your colleagues find?
Asgharnejad: Currently used clinical outcome assessments (COAs) in LGS or DS were not fit for measuring non-seizure outcomes in patients with LGS or DS. Some COA items lacked relevancy or had high levels of missing data, leading to a reduced sensitivity to change. Other COA measures were burdensome for caregivers to complete.
Expert interviews supported the development of a clinical global impression (CGI)-type approach for assessment of within-patient change in non-seizure domains. Following Delphi panel review, three non-seizure items — communication, alertness and disruptive behaviors — were selected due to their clinical relevance and importance in these populations under study.
Healio: What are the clinical implications of these findings?
Asgharnejad: The CGI approach was used to develop a novel measure that addresses the limitations of existing COA measures by assessing within-patient change to create the three-item CGI-I non-seizure symptom measure. This is designed to be sensitive to change in the highly heterogeneous populations of patients with LGS and DS, flexible enough to assess improvement in patients with profound intellectual disabilities, and intuitive enough to use and interpret, reducing caregiver burden.
Healio: What else needs to be done in the future?
Asgharnejad: The next step for the CGI-I non-seizure symptom scale is clinical validation. This study developed the conceptual framework with input from caregiver and [health care professional] interviews as well as a Delphi panel, so we have to see how it performs in clinical use.