Authors :
Presenting Author: Seth Smith, Pharm D., MBA – Pyros Pharmaceuticals
Ray Pourfarzib, Ph D – Consultant, Medical Affairs, StrongTower; Gerard Buonpane, Pharm D – Medical Affairs and Field Medical Team Leader, Medical Affairs, Pyros Pharmaceuticals
Rationale:
Infantile epileptic spasms syndrome (IESS) is a rare developmental and epileptic encephalopathy characterized by the onset of infantile spasms (IS) between 1 and 24 (peak three and twelve) months of age with devastating epilepsy syndrome. Steroids, adrenocorticotropic hormone (ACTH), and vigabatrin (VGB) are considered the most effective treatments.
1
Vigabatrin is indicated for treating IESS in children from one month to two years of age and refractory focal epilepsy in children two years of age and older. Administering oral medications to infants presents various challenges, including their inability to swallow the medicine, difficulty in ensuring proper dosage, and treatment adherence. The current analysis evaluated adherence trends with VGB powder solution in IESS-treated patients 24 months of age and younger versus those 25 months of age and older.
Methods:
FDA Adverse Event Reporting System (FAERS)
2 supports the FDA’s post-marketing safety surveillance program for all marketed drugs. The adverse event (AE) data was obtained from the FAERS database using the following criteria: patients diagnosed with IESS, treated with VGB powder solution, incorrect dose administered, wrong technique in product usage process, and preparation issues. Between 2014 and 2022, sixty-seven IESS patients treated with VGB powder solution who voluntarily reported their AEs to the FAERS database were analyzed. The AEs, defined as product preparation error, incorrect dose administration, and patient or caregiver non-adherence, were measured between the IESS groups divided by age (patients 24 months and younger versus 25 months and older). The AE trends were calculated for the two groups.
Results:
Sixty-seven IESS patients were evaluated for the following AEs: preparation errors, dosing errors, and patient or caregiver non-adherence. Ninety-two events (59%) were reported in the 24-month or younger group versus 64 events (41%) in the 25-month or older group (Table 1). IESS patients (24 months or less) demonstrated higher preparation error (35%) than patients who were 25 months and older (27%).
Conclusions:
The increased AEs, such as preparation error incidents in the VGB powder formulation observed in the FAERS database, suggest that patient non-adherence in infants is a clinical challenge. Unlike older patients who can easily swallow medication, infants lack the necessary development to perform these tasks. As a result, parents and healthcare providers must resort to alternative medication administration methods. This approach may lead to imprecise dosing and potential side effects. Pediatricians and pharmaceutical companies must continue to work together to develop innovative solutions that make medication administration easier and more effective in infants, ultimately improving their overall health and well-being.
Funding: Pyros Pharmaceuticals provided funding to the FDA to receive the blinded adverse event data reports.