Response to nitrazepam in infantile epileptic spasms syndrome after failed standard treatment

Area of Science:

• Pediatric Neurology
• Epileptology
• Clinical Pharmacology

Background:

• Infantile epileptic spasms syndrome (IESS) presents a treatment challenge, with over half of patients unresponsive to standard therapies.
• Nitrazepam demonstrates comparable efficacy to adrenocorticotrophic hormone in managing IESS.
• Evaluating nitrazepam's effectiveness and safety in refractory IESS cases is crucial.

Purpose of the Study:

• To assess the response rates of nitrazepam in children with IESS who have failed prior standard treatments.
• To identify adverse effects associated with nitrazepam therapy in this pediatric population.
• To determine factors influencing treatment response in children with IESS receiving nitrazepam.

Main Methods:

• A retrospective observational study design was employed, analyzing data from January 2013 to December 2021.
• Participants included children diagnosed with IESS who received nitrazepam after failing standard treatments.
• Response was defined as sustained clinical resolution of epileptic spasms at 3-month follow-up; etiologies were classified using the 2017 ILAE criteria.

Main Results:

• Out of 85 children, 26 (30.5%) responded to nitrazepam. Sustained resolution at 12 months was observed in 23.5% of patients.
• Adverse effects were reported in 61.2% of children, most commonly hypersalivation (47.1%).
• Known etiology (OR 5.78) increased the likelihood of response, while longer delay to nitrazepam treatment (OR 0.85) decreased response odds.

Conclusions:

• Nitrazepam achieved a response in approximately 31% of children with IESS refractory to standard treatments.
• Early initiation of nitrazepam treatment appears beneficial, particularly for patients with a known etiology.
• Nitrazepam represents a viable treatment option for refractory IESS, though careful monitoring for adverse effects is warranted.