Abstracts

Rationale: Infantile spasms (IS) is a form of epileptic encephalopathy that is associated with developmental delay, especially in patients with delay in diagnosis and treatment initiation. For patients diagnosed with IS from an etiology other than tuberous sclerosis; high dose oral corticosteroids (OCS), adrenocorticotropic hormone (ACTH), and vigabatrin are preferred treatment options. With the present literature, and lack of clear benefit for one agent over another; providers must weigh the risks and benefits for each by addressing severity of adverse effects, ease of administration, health literacy of parents or guardians, previous therapies, past medical history, and medication cost. If OCS is considered for treatment, providers must determine proper dosing for each agent, as various dosing strategies are available in literature. With the current gaps in literature, the goal of this study was to compare outcomes with different treatment regimens of high dose OCS, ACTH, and vigabatrin, and address cost and time to treatment initiation for each of the treatments.

Methods: This single center retrospective review was completed at Cleveland Clinic Children’s Pediatric Epilepsy Center from May 2014 to June 2021. Patients were included if they were admitted with a diagnosis for infantile spasms during the hospital admission, and followed with the outpatient Pediatric Epilepsy Center for at least 3 months after hospital admission date. Descriptive statistics was used to compare treatment groups.

Results: Forty-six patients were included, of which 17 patients were treated initially with ACTH, 4 patients with OCS, 22 patients with vigabatrin, and 3 patients with other treatment. The median dose of nonstandard OCS was 4 mg/kg/day. Twenty-five percent of patients initially treated with OCS had a late treatment response compared to 41.2% for ACTH. Hypertension, hyperglycemia, and infection combined were greatest in patients treated with ACTH compared to OCS, 70.6% vs. 50.0% respectively. Time to treatment initiation was a median of 0 days for OCS and 2 days for ACTH, and cost based on treatment was $48.10 for OCS and $126,815.80 for ACTH.

Conclusions: Patients with newly diagnosed IS require prompt treatment initiation with guideline directed therapy for improved outcomes. It is key to recognize barriers to treatment approval and time to initiation with agents like vigabatrin and ACTH; as findings in this study highlight that time to treatment initiation is shortest with OCS and longest with vigabatrin and ACTH. Overall, when comparing patients initially treated with ACTH versus OCS; patients with ACTH had higher rates of adverse effects, greater treatment costs, but a larger number of patients had late treatment response.

Funding: There are no sources of funding to disclose.