PCORnet®, the National Patient-Centered Clinical Research Network, aims to improve the nation’s capacity to conduct health research by creating a large, highly representative network for conducting clinical outcomes research. In 2020, PCORI launched an initiative on Conducting Rare Disease Research using PCORnet to answer important questions about the treatment and management of rare diseases or conditions. The initiative funded this research project and others.
This research project is in progress. PCORI will post the research findings on this page within 90 days after the results are final.
What is the research about?
Lennox-Gastaut syndrome, or LGS, is a rare form of epilepsy that has no cure. Epilepsy is a condition in which electrical activity in the brain causes seizures. LGS often starts before the age of four. Children with LGS often have long-term health problems, such as physical disabilities.
Treatments for LGS include medicine and surgery. Both treatments can help reduce the number of seizures patients have, relieve pain, and improve quality of life. But both treatments can also have serious side effects.
In this study, the research team is comparing outcomes among children with LGS who receive medicine or surgery. The team is comparing healthcare use, behavior, and physical function between children in these two groups.
Who can this research help?
Results may help parents, caregivers, and doctors when considering treatments for children with LGS.
What is the research team doing?
First, the research team is using electronic health records, or EHRs, from seven children’s hospitals to identify about 928 patients ages 26 and younger. These patients received an LGS diagnosis or treatment for LGS between 2016 and 2021. The team is looking to see how often children visit the emergency room, or ER, or are admitted to the hospital for seizures. For about half of the patients, parents and caregivers are answering questions about patients’ physical function, communication, and quality of life.
Next, the research team is looking at data from PCORnet® about the patients’ use of medicine and surgery to treat LGS. PCORnet is a national network of health systems created to conduct research funded by PCORI. These networks gather data from EHRs and transform the data into a common format. The team is looking to see if receipt of LGS treatment differs based on patients’ traits, such as age and race.
Parents and caregivers of children with LGS are helping the research team choose outcomes for this study.
Research methods at a glance
|Design||Retrospective cohort study (aims 1a and 2), cross-sectional study (aim 1b)|
Aim 1: 928 patients up to age 26 with a validated diagnosis of LGS between January 1, 2016, and December 31, 2021
Aim 2: ≥4,000 patients with LGS from 18 pediatric hospitals with diagnosis codes from the International Classification of Diseases, Tenth Revision (ICD-10)
Primary: seizure-related ER visits and inpatient admissions, use of surgical and medical treatment
Secondary: parent-reported clinical outcome assessments of patient behavior, communication
|Timeframe||24-month follow-up for primary outcome|
*Patient-Centered Economic Outcomes Funding Supplement