AI Article Synopsis
- Lennox-Gastaut syndrome (LGS) is a severe form of epilepsy in children that often doesn’t respond to treatments, and the study aims to improve therapy development by identifying challenges and setting trial priorities.
- The review outlines various treatment modalities for LGS, including medications, dietary approaches, and surgical options, while emphasizing the need for standardized care based on expert consensus and research data.
- Future clinical trials should focus on a broader range of patient-centered outcomes, informed by recent findings on LGS mechanisms, to develop effective, disease-modifying therapies tailored to the unique characteristics of LGS.
Article Abstract
Objective: Lennox-Gastaut syndrome (LGS) is a severe, childhood-onset epilepsy that is typically refractory to treatment. We surveyed the current landscape of LGS treatment, aiming to identify challenges to the development of efficacious therapies, and to articulate corresponding priorities toward clinical trials that improve outcomes.
Methods: The LGS Special Interest Group of the Pediatric Epilepsy Research Consortium integrated evidence from the literature and expert opinion, into a narrative review.
Results: We provide an overview of approved and emerging medical, dietary, surgical and neuromodulation approaches for LGS. We note that quality of care could be improved by standardizing LGS treatment based on expert consensus and empirical data. Whereas LGS natural history is incompletely understood, prospective studies and use of large retrospective datasets to understand LGS across the lifespan would enable clinical trials that address these dynamics. Recent discoveries related to LGS pathophysiology should enable development of disease-modifying therapies, which are currently lacking. Finally, clinical trials have focused chiefly on seizures involving "drops," but should incorporate additional patient-centered outcomes, using emerging measures adapted to people with LGS.
Interpretation: Clinicians and researchers should enact these priorities, with the goal of patient-centered clinical trials that are tailored to LGS pathophysiology and natural history.
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|---|---|
| http://www.ncbi.nlm.nih.gov/pmc/articles/PMC11572735 | PMC |
| http://dx.doi.org/10.1002/acn3.52211 | DOI Listing |
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