Treatment Practices for Infantile Epileptic Spasms Syndrome: Consensus and Variation in Major U.S. Pediatric Epilepsy Centers
AUTHORS
Christina Briscoe Abath, MD, EdM; Akshat Katyayan, MD; Chellamani Harini, MD; Shaun Hussain, MD, MS; Amanda Sandoval Karamian, MD; Debopam Samanta, MD; Deepa Sirsi, MD; Eva Catenaccio, MD; Stephanie Donatelli, MD; Liu Lin Thio, MD, PhD; Senyene E. Hunter, MD, PhD; Spriha Pavuluri, MD; Sonal Bhatia, MBBS, MD; Duyu Nie, MD, PhD; Gozde Erdemir, MD; Sonam Bhalla, MD; John Mytinger, MD, FAES; Daniel Shrey, MD; Adam Numis, MD
ABSTRACT
Rationale:
Infantile epileptic spasms syndrome (IESS) is the most common infantile onset pediatric epilepsy, affecting approximately 1 in 3000 children (1,2). There is broad consensus on the use of standard therapies as the first treatment, including adrenocorticotropic hormone, corticosteroids, and vigabatrin (3-5). We sought to better understand the practices of IESS at major U.S. pediatric epilepsy centers regarding second and third line treatments, EEG practices, and side effect monitoring. This descriptive study is the first step to developing a consensus-based Pediatric Epilepsy Research Consortium (PERC) guideline and quality metric.
Methods:
Methods were modeled after the neonatal seizures treatment pathways (6). The PERC Infantile Spasms Special Interest Group included 75 pediatric epilepsy centers at the time of the survey. Following ethics approval, we distributed a RedCap survey to all members. For sites with multiple members, one member was designated to complete the survey. Treatment pathways were uploaded by sites and anonymized (Table 2). AK, AN, and CBA developed a variable list after the first 3 were analyzed as a group for consensus and variable refinement. When all treatment pathways are analyzed, they will inform the creation of a modified Delphi instrument.
Results:
Thirty-six sites participated in the survey (48% completion rate) (Table 1). They were all National Association of Epilepsy Centers accredited and 35 were level 4 centers. Most (89%, n=32) had IESS treatment pathways. Influence of pathways by insurance issues was common (n=23, 64%). Guidelines represent current practice, as (1) Most treating neurologists reportedly adhered to the pathways (n=35, 97%) (2) Most pathways had been updated in the last 1-3 years (n=26, 81%).
Six treatment pathways have been analyzed to date. All recommended standard therapy as 1st line and 2nd line treatment. One site (17%) recommended dual therapy with vigabatrin and steroid at onset; 2 sites recommended considering dual therapy (33%). Most sites (83%) recommended repeat EEG and adding a different standard therapy at 14 days for patients without electroclinical remission. Three sites’ guidelines (50%) included a 3rd line therapy. None provided guidance for repeat EEG beyond 14-day follow-up. Four (67%) guidelines mentioned ketogenic diet, whereas 1 (17%) mentioned epilepsy surgery evaluation. Side effect monitoring for steroids was heterogeneous, ranging from blood pressure (67%), urine glucose (50%), stool guiac (17%), pneumocystis prophylaxis (17%), baseline labs (33%), or none.
Conclusions:
The majority of PERC site participants had institution specific IESS treatment pathways. While there was broad consensus regarding first and second line treatment for IESS with standard therapies, preliminary analysis of the first 6 pathways showed variability in use of dual therapy at onset, inclusion of a third line treatment, side effect monitoring, and inclusion of ketogenic diet or epilepsy surgery. These results will allow us to develop a second modified Delphi PERC survey. Our long-term goal is to use these studies to create a publicly available IESS PERC consensus treatment pathway and quality metrics.
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