Abstract
OBJECTIVE: Due to the conflicting evidence and expected regional therapeutic variations for cost-effectiveness in children with infantile epileptic spasms syndrome (IESS), this study assessed treatment effectiveness, safety, Health-Related Quality of Life (HRQoL), and performed a cost-effectiveness analysis (CEA) for hormonal therapies in Indian children with IESS. METHODS: This prospective observational study was conducted at a referral pediatrics center in North India. Children with IESS (aged 3-18 months), who were prescribed either a synthetic form of adrenocorticotrophic hormone (ACTH) or oral prednisolone, were included and compared for cessation of epileptic spasms (ES), treatment-emergent adverse events (TEAEs), HRQoL (assessed through Hi-QUALIN), and cost-effectiveness using a decision-tree model. Incremental cost effectiveness ratio (ICER) and number needed to treat were calculated. One-way sensitivity analyses predicted the impact of each variable on the cost-effectiveness (CE) model using a Tornado diagram. RESULTS: Of 93 children with IESS [73 (79%) boys; mean age (SD) = 10.1 (3.7) months; median (IQR) daily burden of ES: 27.8 (14.0, 61.9)], 55 (59%) were initiated on ACTH, while the rest on oral prednisolone (38 [41%]). After 2 weeks of treatment, ES cessation was achieved in 29/55 (53%) on ACTH and 13/38 (34%) on oral prednisolone. Additionally, an insignificantly different incidence of TEAEs was reported in the ACTH group (23/55 [42%]) compared with the oral prednisolone group (19/38 [50%]). Children aged 3-12 months showed statistically significant improvement in overall HRQoL scores at 2 weeks of treatment (p < 0.01). Using a decision tree model, CEA showed higher ICER (ICER: 122 185 INR; ~1468.90 USD and QALY: 331 643 INR; ~3987.0 USD) for ACTH than oral prednisolone, suggesting caregiver or parents need to spend at least 122 185 INR for one in five children on ACTH to be ES free compared with prednisolone. SIGNIFICANCE: This analysis suggests ACTH was more cost-effective than oral prednisolone for managing IESS from the Indian patient's perspective. ACTH was also a cost-effective option for short-term quality of life outcomes. PLAIN LANGUAGE SUMMARY: This study looked at two first-line treatment options, that is, ACTH and oral prednisolone, for infantile epileptic spasms syndrome (IESS) at a referral pediatric center in North India. We found that ACTH was more effective than oral prednisolone for epileptic spasm cessation and improved health-related quality of life in the short term. Although ACTH costs more, it was found to be a cost-effective option. These findings can help families and clinicians make better treatment choices based on both health benefits and costs.