Authors :
Presenting Author: Chellamani Harini, MD – Boston Children's Hospital
Akshat Katyayan, MD – Baylor College of Medicine/Texas Children's Hospital
Christina Briscoe, MD, EdM – Boston Children's Hospital
Maria Planchart Ferretto, MD – Boston Children's Hospital
Debopam Samanta, MD – University of Arkansas
Amanda Sandoval Karamian, MD – University of Utah
Senyene Hunter, MD, PhD – University of North Carolina - Chapel Hill
John Mytinger, MD – Nationwide Children's Hospital
Deepa Sirsi, MD – University of Texas Southwestern Medical Center, Dallas, Texas
Shital Patel, MD – Duke University. Department of Pediatrics Durham, North Carolina.
Avantika Singh, MD – Medical College of Wisconsin
Danielle Takacs, MD – Baylor College of Medicine/Texas Children's Hospital
Sonam Bhalla, MD – Emory University/ Children's Healthcare of Atlanta
Archana Pasupuleti, MD – Children’s National Health System
Sonal Bhatia, MD – Medical University of South Carolina
Stephanie Donatelli, MD – Boston Children's Hospital
Gozde Erdemir, MD – Penn State Health, Penn State College of Medicine. Department of Pediatrics. Hershey, Pennsylvania.
Emily Spelbrink, MD, PhD – Stanford
Adam Numis, MD – University of California San Francisco. Department of Neurology and Weill Institute for Neuroscience. San Francisco, California.
Shaun A. Hussain, MD, MS – Division of Pediatric Neurology, Department of Pediatrics, UCLA Mattel Children's Hospital, David Geffen School of Medicine
Rationale:
Infantile Epileptic Spasms Syndrome (IESS) is a severe early-life epilepsy requiring timely diagnosis and treatment. While U.S. and international guidelines clearly use standard therapy as first-line treatment, there are gaps in recommendations regarding sequence, timing, and diagnostic modalities. Understanding current practices can inform future guideline development and improve care.
Methods:
A 21-item survey was developed by the Pediatric Epilepsy Research Consortium (PERC) IESS Special Interest Group, addressing diagnostic, medical, and surgical management, following a review of guidelines from PERC centers in our prior work. One pediatric epileptologist at each of the 45 U.S. tertiary centers in the PERC IESS Special Interest Group was invited to respond. Surveys were collected between March to April 2025.
Results:
Thirty-three centers (73% response) completed the survey. Most (91%) managed new-onset IESS in the inpatient setting. Overnight video EEG was the most common diagnostic modality (76%); 91% obtained follow-up EEG in all cases, and most assessed treatment response at 14 days (78%). Treatment response was assessed using the BASED score in most cases (58%), while traditional evaluation for hypsarrhythmia resolution was less commonly used (39%). All centers obtained MRIs, and 97% pursued genetic testing on patients without known etiology. Pyridoxine challenge was not routinely performed at most centers (n=26, 78%). Regarding treatment, high-dose prednisolone was the preferred first-line treatment across clinical scenarios: 67% (n=22) of centers for any non-TSC-associated IESS, 73% (n=24) for patients with unknown etiology and normal development, and 76% (n=25) for patients with presumed high risk of neurodevelopmental disabilities. ACTH or combination therapy was used less frequently. Vigabatrin (42%) was the most common second-line therapy, followed by alternate hormonal agents (33%) and combination therapy (21%). Among vigabatrin responders, treatment duration was typically 5–15 months. The ketogenic diet was the most common third-line option (39%). Sixteen (52 %) centers endorsed the use of maintenance antiseizure medication after remission. Surgery was usually pursued only after failure of both hormonal and vigabatrin treatment (63%). However, 78% (n=26) would consider surgery even after clinical cessation of epileptic spasms if EEG continued to show a probable epileptic encephalopathy (hypsarrhythmia or BASED score of 4 or 5), with 5 centers (15%) having a threshold of BASED score 3.
Conclusions:
Clinical practice across PERC centers has evolved according to evolving evidence, generating broad consensus regarding the use of high-dose prednisolone and sequential therapy, increased use of the BASED score, and ubiquity of genetic testing in most cases. However, there were more discrepancies regarding second and third-lines therapies, duration of vigabatrin therapy in responders, and criteria for referral for epilepsy surgery. These findings underscore the need for updated evidence-based guidelines to standardize care for IESS nationwide.
Funding: There was no funding for this abstract.