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 ACTH, 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.

The Pediatric Epilepsy Research Consortium (PERC) 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.

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%).

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.