Project Summary

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 Element Description
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)

  • Antiseizure medicine
  • Palliative surgery

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 Timeframe Length of follow-up for collecting data on primary outcomes. View Glossary 24-month follow-up for primary outcome

*Patient-Centered Economic Outcomes Funding Supplement 
This study received supplemental funding to build on existing PCORI-funded comparative clinical effectiveness research (CER) studies to improve the understanding of appropriate costs and other economic burdens that are meaningful and inclusive to patients, caregivers and other stakeholders.

Project Information

Sandi Lam, MD, MBA
Ann & Robert H. Lurie Children's Hospital of Chicago
Comparative Effectiveness of Palliative Surgery versus Additional Anti-Seizure Medications for Lennox-Gastaut Syndrome

Key Dates

March 2021
August 2026

Study Registration Information


Health Conditions Health Conditions These are the broad terms we use to categorize our funded research studies; specific diseases or conditions are included within the appropriate larger category. Note: not all of our funded projects focus on a single disease or condition; some touch on multiple diseases or conditions, research methods, or broader health system interventions. Such projects won’t be listed by a primary disease/condition and so won’t appear if you use this filter tool to find them. View Glossary
Populations Populations PCORI is interested in research that seeks to better understand how different clinical and health system options work for different people. These populations are frequently studied in our portfolio or identified as being of interest by our stakeholders. View Glossary
Intervention Strategy Intervention Strategies PCORI funds comparative clinical effectiveness research (CER) studies that compare two or more options or approaches to health care, or that compare different ways of delivering or receiving care. View Glossary
State State The state where the project originates, or where the primary institution or organization is located. View Glossary
Last updated: April 12, 2024