Response to First Treatment for Infantile Epileptic Spasms Syndrome: Initial Analysis of the NIMBIS Study
Abstract number :
3.427
Submission category :
4. Clinical Epilepsy / 4C. Clinical Treatments
Year :
2023
Submission ID :
1411
Source :
www.aesnet.org
Presentation date :
12/4/2023 12:00:00 AM
Published date :
Authors :
Presenting Author: Sonal Bhatia, MD – Medical University of South Carolina
Shaun Hussain, MD – University of California, Los Angeles; Rani Singh, MD – Atrium Health; Sonam Bhalla, MD – Children's Healthcare of Atlanta; Peter Chang, MD – UC Irvine; Wei-Liang Chen, MD – Children's National Hospital; Michael Ciliberto, MD – University of Iowa; Jason Coryell, MD, MS – Oregon Health & Sciences University; Chellamani Harini, MD – Harvard University; Ann Hyslop, MD – Stanford University; Cynthia Keator, MD – Cook Children's; Hollie Lai, MD – Children's Hospital of Orange County; Beth Lopour, PhD – UC Irvine; Jennifer Madan Cohen, MD – Connecticut Children's; Venus Mostaghimi, MS – UC Irvine; John Mytinger, MD – Nationwide Children's Hospital, The Ohio State University; Sunil Naik, MD – Penn State Children's Hospital; Kerri Neville, MD – University of Michigan; Adam Numis, MD – UC San Francisco; Archana Pasupuleti, MD – Children's National Hospital; Debopam Samanta, MD – Arkansas Children's/University of Arkansas for Medical Sciences; Amanda Sandoval Karamian, MD – University of Utah; Nilika Singhal, MD – UC San Francisco; Deepa Sirsi, MD – University of Texas Southwestern Medical Center; Emily Spelbrink, MD, PhD – Stanford University; Carl Stafstrom, MD, PhD – Johns Hopkins University; Danielle Takacs, MD – Baylor College of Medicine; Tyler Terrill, MD – University of Texas Southwestern Medical Center; Linh Tran, DO, MS – Cook Children's; Elissa Yozawit, MD – Montefiore Medical Center; Christopher Yuskaitis, MD, PhD – Harvard University; Kelly Knupp, MD – University of Colorado, Anschutz Medical Campus; Renée Shellhaas, MD, MS – Washington University in St Louis; Zachary Grinspan, MD, MS – Weill Cornell Medicine; Daniel Shrey, MD – Children's Hospital of Orange County
Rationale:
Treatment protocols for Infantile Epileptic Spasms Syndrome (IESS) vary across centers and have evolved in recent years. To reexamine treatment patterns and identify novel multimodal biomarkers, twenty centers have collected comprehensive data on children with IESS, including demographics, treatment, outcome, EEG, and MRI data, as part of the National Investigation of Multi-modal Biomarkers for Infantile Spasms (NIMBIS) Study. We explored short-term clinical response based on the first treatment regimen.
Methods:
Clinical and demographic data were abstracted from the electronic medical record of children with new-onset IESS before twelve months of age, diagnosed between 2016 and 2020. Evaluation and treatment were at the discretion of the local clinical team. Clinical response was defined as the resolution of clinical infantile spasms without relapse for at least 28 days. “Standard” therapy was defined as a regimen including prednisolone (PRED), adrenocorticotropic hormone (ACTH), vigabatrin (VGB), or a combination thereof. Developmental delay was defined as “definite” delay identified by the treating child neurologist in at least one developmental domain. Multivariable logistic regression was used to compare treatment regimens. Multivariable models were developed with a forward stepwise approach, using two-fold cross-validation.
Results:
A total of 456 children had sufficient data to characterize short-term clinical response to first treatment regimen; 256 (56%) responded (Table 1). In sequential bivariate analyses, response was associated with lack of epilepsy before IESS onset (P < 0.001), standard therapy (P = 0.001), normal development at onset (P = 0.001), lack of microcephaly (P = 0.001), older age of onset of IESS (P = 0.003), and private health insurance (P = 0.008). In multivariable analysis, response was independently associated with standard treatment (OR 3.9; 95%CI 1.7 – 9.3), lack of epilepsy prior to IESS (OR 2.3; 95%CI 1.4 – 3.6), and normal development at IESS onset (OR 2.0; 95%CI 1.3 – 3.1). Unadjusted response rates were as follows: ACTH (101/159; 64%), prednisolone (84/156; 54%), vigabatrin (38/75; 51%), combination therapy (any hormonal therapy plus vigabatrin; 23/34; 68%), and other (10/32; 31%). With adjustment for prior epilepsy and normal development, there was no significant difference in response between ACTH versus prednisolone (OR 1.39; 95%CI 0.87 – 2.2, direction of effect in favor of ACTH). After exclusion of children with Tuberous Sclerosis Complex and adjustment for prior epilepsy, response to hormonal therapy was higher in comparison to VGB (OR 1.8; 95% 1.02 – 4.3).
Clinical Epilepsy