Comparative effectiveness of epilepsy surgery versus additional anti-seizure medications for Lennox–Gastaut syndrome: study protocol for a multicenter, mixed-methods study
IntroductionLennox–Gastaut Syndrome (LGS) is a severe developmental epileptic encephalopathy without a known cure. Management of symptoms requires substantial care. Treatment options include anti-seizure medications, dietary therapy, and epilepsy surgery. Two main treatment pathways for patients wit...
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Frontiers Media S.A.
2025-06-01
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| author | Sandi Lam Sandi Lam Marc Rosenman Marc Rosenman Tracy Dixon-Salazar Kelly G. Knupp Liu Lin Thio Taylor J. Abel Taylor J. Abel William P. Welch William P. Welch Laurel Reed Laurel Reed Stephanie C. Randle Stephanie C. Randle Rebecca Garcia-Sosa Rebecca Garcia-Sosa Jason S. Hauptman Carolyn C. Foster Carolyn C. Foster Elizabeth R. Alpern Elizabeth R. Alpern Lu Zhang Lu Zhang Nicole Villalba Nicole Villalba Maura Carroll Anup D. Patel |
| author_facet | Sandi Lam Sandi Lam Marc Rosenman Marc Rosenman Tracy Dixon-Salazar Kelly G. Knupp Liu Lin Thio Taylor J. Abel Taylor J. Abel William P. Welch William P. Welch Laurel Reed Laurel Reed Stephanie C. Randle Stephanie C. Randle Rebecca Garcia-Sosa Rebecca Garcia-Sosa Jason S. Hauptman Carolyn C. Foster Carolyn C. Foster Elizabeth R. Alpern Elizabeth R. Alpern Lu Zhang Lu Zhang Nicole Villalba Nicole Villalba Maura Carroll Anup D. Patel |
| author_sort | Sandi Lam |
| collection | DOAJ |
| description | IntroductionLennox–Gastaut Syndrome (LGS) is a severe developmental epileptic encephalopathy without a known cure. Management of symptoms requires substantial care. Treatment options include anti-seizure medications, dietary therapy, and epilepsy surgery. Two main treatment pathways for patients with LGS with drug resistant epilepsy are additional anti-seizure medications or epilepsy surgery, which have been reported to be effective in reduction of seizure burden and improving quality of life. No studies have directly compared the outcomes of using epilepsy surgery versus using additional anti-seizure medications for the treatment of LGS.MethodsThis study is a multicenter, mixed-methods comparative effectiveness study of LGS patients who have undergone epilepsy surgery or have received an LGS-approved medication for treatment resistant seizures. Aim 1 will analyze the effect of surgical therapies and additional medication on two clinical outcomes: (1a) seizure-related healthcare utilization, and (1b) expressive communication, behavior, and parent-reported quality of life. Based on electronic health record review and coding validation as part of Aim 1a, we will develop computable phenotypes for LGS. The phenotypes will inform the analyses in Aim 1a and Aim 2. Aim 2 will describe the real-world utilization of these treatments across multiple healthcare institutions in the United States. Data will be collected from electronic health records, data marts in the National Patient-Centered Clinical Research Network (PCORnet®) format, caregiver surveys, and focus groups.DiscussionThis study of LGS will provide currently unavailable evidence concerning the real-world comparative effectiveness of epilepsy surgeries and additional anti-seizure medications. The outcomes are those that families identify as important: emergency medical care for seizures and patients’ functional outcomes. The results of this study may help guide decisions regarding the treatment of LGS and development of computable phenotypes for this rare disease. This study using PCORnet® data will also lay the groundwork for future large-scale studies on LGS and other rare epilepsies.Clinical trial registrationClinicalTrials.gov, identifier NCT05374824. |
| format | Article |
| id | doaj-art-26282e741a2942e182cddd64e8cda848 |
| institution | Kabale University |
| issn | 1664-2295 |
| language | English |
| publishDate | 2025-06-01 |
| publisher | Frontiers Media S.A. |
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| series | Frontiers in Neurology |
| spelling | doaj-art-26282e741a2942e182cddd64e8cda8482025-08-20T03:31:01ZengFrontiers Media S.A.Frontiers in Neurology1664-22952025-06-011610.3389/fneur.2025.15695511569551Comparative effectiveness of epilepsy surgery versus additional anti-seizure medications for Lennox–Gastaut syndrome: study protocol for a multicenter, mixed-methods studySandi Lam0Sandi Lam1Marc Rosenman2Marc Rosenman3Tracy Dixon-Salazar4Kelly G. Knupp5Liu Lin Thio6Taylor J. Abel7Taylor J. Abel8William P. Welch9William P. Welch10Laurel Reed11Laurel Reed12Stephanie C. Randle13Stephanie C. Randle14Rebecca Garcia-Sosa15Rebecca Garcia-Sosa16Jason S. Hauptman17Carolyn C. Foster18Carolyn C. Foster19Elizabeth R. Alpern20Elizabeth R. Alpern21Lu Zhang22Lu Zhang23Nicole Villalba24Nicole Villalba25Maura Carroll26Anup D. Patel27Division of Pediatric Neurosurgery, Department of Surgery, Ann & Robert H. Lurie Children’s Hospital of Chicago, Chicago, IL, United StatesDepartment of Neurological Surgery, Northwestern University Feinberg School of Medicine, Chicago, IL, United StatesMary Ann & J. Milburn Smith Child Health Outcomes, Research, and Evaluation Center (SCHORE), Ann & Robert H. Lurie Children’s Hospital of Chicago, Chicago, IL, United StatesDepartment of Pediatrics, Northwestern University Feinberg School of Medicine, Chicago, IL, United StatesLennox-Gastaut Syndrome (LGS) Foundation, San Diego, CA, United StatesDepartment of Pediatrics, Division of Neurology, University of Colorado School of Medicine Anschutz Medical Campus, Aurora, CO, United StatesDepartment of Neurology, Washington University School of Medicine, St. Louis, MO, United StatesDepartment of Neurological Surgery, University of Pittsburgh School of Medicine, Pittsburgh, PA, United StatesDivision of Pediatric Neurosurgery, University of Pittsburgh Medical Center Children’s Hospital of Pittsburgh, Pittsburgh, PA, United States0Division of Neurology and Child Development Unit, Department of Pediatrics, University of Pittsburgh School of Medicine, Pittsburgh, PA, United States1Division of Child Neurology, University of Pittsburgh Medical Center Children’s Hospital of Pittsburgh, Pittsburgh, PA, United States2Division of Pediatric Neurology, Department of Pediatrics, Michigan Medicine, University of Michigan, Ann Arbor, MI, United States3Pediatric Neurology Department, C.S. Mott Children’s Hospital, Ann Arbor, MI, United States4Division of Pediatric Neurology, Department of Neurology, University of Washington, Seattle, WA, United States5Division of Pediatric Neurology and Epilepsy, Department of Pediatrics, Seattle Children’s Hospital, Seattle, WA, United States6Division of Neurology, Department of Pediatrics, Northwestern University Feinberg School of Medicine, Chicago, IL, United States7Division of Pediatric Neurology, Ann & Robert H. Lurie Children’s Hospital of Chicago, Chicago, IL, United States8Division of Neurological Surgery, Phoenix Children’s Hospital, Phoenix, AZ, United StatesMary Ann & J. Milburn Smith Child Health Outcomes, Research, and Evaluation Center (SCHORE), Ann & Robert H. Lurie Children’s Hospital of Chicago, Chicago, IL, United StatesDepartment of Pediatrics, Northwestern University Feinberg School of Medicine, Chicago, IL, United StatesDepartment of Pediatrics, Northwestern University Feinberg School of Medicine, Chicago, IL, United States9Division of Pediatric Emergency Medicine, Ann & Robert H. Lurie Children’s Hospital of Chicago, Chicago, IL, United StatesDivision of Pediatric Neurosurgery, Department of Surgery, Ann & Robert H. Lurie Children’s Hospital of Chicago, Chicago, IL, United StatesDepartment of Neurological Surgery, Northwestern University Feinberg School of Medicine, Chicago, IL, United StatesDivision of Pediatric Neurosurgery, Department of Surgery, Ann & Robert H. Lurie Children’s Hospital of Chicago, Chicago, IL, United StatesDepartment of Neurological Surgery, Northwestern University Feinberg School of Medicine, Chicago, IL, United StatesDivision of Pediatric Neurosurgery, Department of Surgery, Ann & Robert H. Lurie Children’s Hospital of Chicago, Chicago, IL, United States0Division of Neurology, Nationwide Children’s Hospital, Department of Pediatrics, The Ohio State University College of Medicine, Columbus, OH, United StatesIntroductionLennox–Gastaut Syndrome (LGS) is a severe developmental epileptic encephalopathy without a known cure. Management of symptoms requires substantial care. Treatment options include anti-seizure medications, dietary therapy, and epilepsy surgery. Two main treatment pathways for patients with LGS with drug resistant epilepsy are additional anti-seizure medications or epilepsy surgery, which have been reported to be effective in reduction of seizure burden and improving quality of life. No studies have directly compared the outcomes of using epilepsy surgery versus using additional anti-seizure medications for the treatment of LGS.MethodsThis study is a multicenter, mixed-methods comparative effectiveness study of LGS patients who have undergone epilepsy surgery or have received an LGS-approved medication for treatment resistant seizures. Aim 1 will analyze the effect of surgical therapies and additional medication on two clinical outcomes: (1a) seizure-related healthcare utilization, and (1b) expressive communication, behavior, and parent-reported quality of life. Based on electronic health record review and coding validation as part of Aim 1a, we will develop computable phenotypes for LGS. The phenotypes will inform the analyses in Aim 1a and Aim 2. Aim 2 will describe the real-world utilization of these treatments across multiple healthcare institutions in the United States. Data will be collected from electronic health records, data marts in the National Patient-Centered Clinical Research Network (PCORnet®) format, caregiver surveys, and focus groups.DiscussionThis study of LGS will provide currently unavailable evidence concerning the real-world comparative effectiveness of epilepsy surgeries and additional anti-seizure medications. The outcomes are those that families identify as important: emergency medical care for seizures and patients’ functional outcomes. The results of this study may help guide decisions regarding the treatment of LGS and development of computable phenotypes for this rare disease. This study using PCORnet® data will also lay the groundwork for future large-scale studies on LGS and other rare epilepsies.Clinical trial registrationClinicalTrials.gov, identifier NCT05374824.https://www.frontiersin.org/articles/10.3389/fneur.2025.1569551/fullLennox–Gastaut syndromeepilepsyepilepsy surgeryanti-seizure medicationscomparative effectiveness study |
| spellingShingle | Sandi Lam Sandi Lam Marc Rosenman Marc Rosenman Tracy Dixon-Salazar Kelly G. Knupp Liu Lin Thio Taylor J. Abel Taylor J. Abel William P. Welch William P. Welch Laurel Reed Laurel Reed Stephanie C. Randle Stephanie C. Randle Rebecca Garcia-Sosa Rebecca Garcia-Sosa Jason S. Hauptman Carolyn C. Foster Carolyn C. Foster Elizabeth R. Alpern Elizabeth R. Alpern Lu Zhang Lu Zhang Nicole Villalba Nicole Villalba Maura Carroll Anup D. Patel Comparative effectiveness of epilepsy surgery versus additional anti-seizure medications for Lennox–Gastaut syndrome: study protocol for a multicenter, mixed-methods study Frontiers in Neurology Lennox–Gastaut syndrome epilepsy epilepsy surgery anti-seizure medications comparative effectiveness study |
| title | Comparative effectiveness of epilepsy surgery versus additional anti-seizure medications for Lennox–Gastaut syndrome: study protocol for a multicenter, mixed-methods study |
| title_full | Comparative effectiveness of epilepsy surgery versus additional anti-seizure medications for Lennox–Gastaut syndrome: study protocol for a multicenter, mixed-methods study |
| title_fullStr | Comparative effectiveness of epilepsy surgery versus additional anti-seizure medications for Lennox–Gastaut syndrome: study protocol for a multicenter, mixed-methods study |
| title_full_unstemmed | Comparative effectiveness of epilepsy surgery versus additional anti-seizure medications for Lennox–Gastaut syndrome: study protocol for a multicenter, mixed-methods study |
| title_short | Comparative effectiveness of epilepsy surgery versus additional anti-seizure medications for Lennox–Gastaut syndrome: study protocol for a multicenter, mixed-methods study |
| title_sort | comparative effectiveness of epilepsy surgery versus additional anti seizure medications for lennox gastaut syndrome study protocol for a multicenter mixed methods study |
| topic | Lennox–Gastaut syndrome epilepsy epilepsy surgery anti-seizure medications comparative effectiveness study |
| url | https://www.frontiersin.org/articles/10.3389/fneur.2025.1569551/full |
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