TY - JOUR
T1 - Treatment Practices for Infantile Epileptic Spasms Syndrome
T2 - Consensus and Variation in Major Pediatric Epilepsy Centers
AU - Briscoe, Christina
AU - Katyayan, Akshat
AU - Harini, Chellamani
AU - Hussain, Shaun A.
AU - Bhalla, Sonam
AU - Singh, Avantika
AU - Donatelli, Stephanie
AU - Sandoval Karamian, Amanda G.
AU - Samanta, Debopam
AU - Sirsi, Deepa
AU - Catenaccio, Eva
AU - Planchart Ferretto, Maria A.
AU - Thio, Liu Lin
AU - Hunter, Senyene E.
AU - Spriha, Pavuluri
AU - Rao, Chethan K.
AU - Bhatia, Sonal
AU - Erdemir, Gozde
AU - Shrey, Daniel W.
AU - Numis, Adam L.
N1 - Publisher Copyright:
© 2025 Elsevier Inc.
PY - 2026/1
Y1 - 2026/1
N2 - Background: Infantile epileptic spasms syndrome (IESS) is a developmental and epileptic encephalopathy that requires prompt, effective treatment to optimize outcomes. While the first therapies for IESS with adrenocorticotrophic hormone, prednisolone, or vigabatrin are widely established as a standard, we hypothesized that the treatment protocols of how these therapies should be implemented varied across medical centers. Methods: The Pediatric Epilepsy Research Consortium Infantile Spasms Special Interest Group distributed a REDCap survey to 75 US epilepsy centers. Predefined treatment pathway characteristics were extracted and compared. Standard therapy regimens were defined before data collection. Results: Thirty-six centers participated (48% completion rate). Most (89%, n = 32) had IESS treatment pathways, with 72% (n = 23) influenced by insurance barriers such as prior authorizations. Of these, 75% (n = 24) contributed pathways for analysis. Most protocols (88%, n = 21) recommended a standard treatment course for new-onset IESS. Of these, 63% (n = 15) endorsed a sequential approach to using hormonal therapy and vigabatrin, while 17% (n = 4) recommended combination therapy with both for all children. Thirteen centers (54%) provided recommendations for treating persistent epileptic spasms. Approaches to side-effect mitigation varied widely, with gastrointestinal prophylaxis and blood pressure control being the most common (79%, n = 19). Half of the pathways mentioned ketogenic diet (58%) or epilepsy surgery (46%). Conclusions: While there was broad consensus regarding first and second therapy treatment for IESS, variability existed in using sequential versus combination therapy, third therapies, and adverse event monitoring. These findings will guide next research steps in defining key questions on sequential versus combination therapy, third line therapy, and adverse event monitoring in order to develop a standardized consensus-driven treatment protocol for IESS in the future.
AB - Background: Infantile epileptic spasms syndrome (IESS) is a developmental and epileptic encephalopathy that requires prompt, effective treatment to optimize outcomes. While the first therapies for IESS with adrenocorticotrophic hormone, prednisolone, or vigabatrin are widely established as a standard, we hypothesized that the treatment protocols of how these therapies should be implemented varied across medical centers. Methods: The Pediatric Epilepsy Research Consortium Infantile Spasms Special Interest Group distributed a REDCap survey to 75 US epilepsy centers. Predefined treatment pathway characteristics were extracted and compared. Standard therapy regimens were defined before data collection. Results: Thirty-six centers participated (48% completion rate). Most (89%, n = 32) had IESS treatment pathways, with 72% (n = 23) influenced by insurance barriers such as prior authorizations. Of these, 75% (n = 24) contributed pathways for analysis. Most protocols (88%, n = 21) recommended a standard treatment course for new-onset IESS. Of these, 63% (n = 15) endorsed a sequential approach to using hormonal therapy and vigabatrin, while 17% (n = 4) recommended combination therapy with both for all children. Thirteen centers (54%) provided recommendations for treating persistent epileptic spasms. Approaches to side-effect mitigation varied widely, with gastrointestinal prophylaxis and blood pressure control being the most common (79%, n = 19). Half of the pathways mentioned ketogenic diet (58%) or epilepsy surgery (46%). Conclusions: While there was broad consensus regarding first and second therapy treatment for IESS, variability existed in using sequential versus combination therapy, third therapies, and adverse event monitoring. These findings will guide next research steps in defining key questions on sequential versus combination therapy, third line therapy, and adverse event monitoring in order to develop a standardized consensus-driven treatment protocol for IESS in the future.
UR - https://www.scopus.com/pages/publications/105020454959
UR - https://www.scopus.com/pages/publications/105020454959#tab=citedBy
U2 - 10.1016/j.pediatrneurol.2025.10.005
DO - 10.1016/j.pediatrneurol.2025.10.005
M3 - Article
C2 - 41172580
AN - SCOPUS:105020454959
SN - 0887-8994
VL - 174
SP - 46
EP - 53
JO - Pediatric Neurology
JF - Pediatric Neurology
ER -