ECONOMIC EVALUATION OF HORMONAL TREATMENT STRATEGIES FOR INFANTILE EPILEPTIC SPASMS SYNDROME IN INDIAN CHILDREN
Author(s)
Dipika Bansal, MD1, Nagita Devi II, MS, PhD2, Jitendra Kumar Sahu, MBBS, MD, DM3;
1National Institute of Pharmaceutical Education and Research, Professor, Mohali, India, 2MM College of Pharmacy, Maharishi Markandeshwar (Deemed To Be) University, Mohali, India, 3PGIMER Chandigarh, Chandigarh, India
1National Institute of Pharmaceutical Education and Research, Professor, Mohali, India, 2MM College of Pharmacy, Maharishi Markandeshwar (Deemed To Be) University, Mohali, India, 3PGIMER Chandigarh, Chandigarh, India
OBJECTIVES: Evidence comparing hormonal therapies for infantile epileptic spasms syndrome (IESS) remains inconsistent, and treatment choices are strongly influenced by regional costs and healthcare delivery. This study evaluated the comparative effectiveness, safety, health-related quality of life (HRQoL), and cost-effectiveness of hormonal therapies among Indian children diagnosed with IESS
METHODS: A prospective observational study was carried out at a tertiary pediatric referral center in North India. Children aged 3-18 months with IESS who were initiated on either synthetic adrenocorticotrophic hormone (ACTH) or oral prednisolone were enrolled. Outcomes included cessation of epileptic spasms (ES) at 2 weeks, treatment-emergent adverse events (TEAEs), and HRQoL measured using the Hi-QUALIN instrument. A decision-tree model was developed to assess cost-effectiveness from the patient perspective. Incremental cost-effectiveness ratios (ICERs) and numbers needed to treat were calculated, and one-way sensitivity analyses were performed, with results summarized using a Tornado diagram.
RESULTS: A total of 93 children with IESS were included (79% boys; mean age 10.1 ± 3.7 months; median daily ES frequency 27.8 [IQR 14.0-61.9]). ACTH was prescribed to 55 children (59%), while 38 (41%) received oral prednisolone. After 2 weeks, ES cessation occurred in 53% of children treated with ACTH compared with 34% receiving prednisolone. TEAEs were reported in 42% of the ACTH group and 50% of the prednisolone group, with no statistically significant difference. Children aged 3-12 months demonstrated significant improvement in overall HRQoL scores after treatment initiation (p < 0.01). Cost-effectiveness analysis indicated a higher ICER for ACTH (₹122,185 per additional responder; ₹331,643 per QALY gained), reflecting greater upfront costs but improved clinical outcomes.
CONCLUSIONS: From an Indian healthcare perspective, ACTH demonstrated superior cost-effectiveness compared with oral prednisolone for short-term management of IESS, with better seizure control and favorable HRQoL outcomes despite higher treatment costs.
METHODS: A prospective observational study was carried out at a tertiary pediatric referral center in North India. Children aged 3-18 months with IESS who were initiated on either synthetic adrenocorticotrophic hormone (ACTH) or oral prednisolone were enrolled. Outcomes included cessation of epileptic spasms (ES) at 2 weeks, treatment-emergent adverse events (TEAEs), and HRQoL measured using the Hi-QUALIN instrument. A decision-tree model was developed to assess cost-effectiveness from the patient perspective. Incremental cost-effectiveness ratios (ICERs) and numbers needed to treat were calculated, and one-way sensitivity analyses were performed, with results summarized using a Tornado diagram.
RESULTS: A total of 93 children with IESS were included (79% boys; mean age 10.1 ± 3.7 months; median daily ES frequency 27.8 [IQR 14.0-61.9]). ACTH was prescribed to 55 children (59%), while 38 (41%) received oral prednisolone. After 2 weeks, ES cessation occurred in 53% of children treated with ACTH compared with 34% receiving prednisolone. TEAEs were reported in 42% of the ACTH group and 50% of the prednisolone group, with no statistically significant difference. Children aged 3-12 months demonstrated significant improvement in overall HRQoL scores after treatment initiation (p < 0.01). Cost-effectiveness analysis indicated a higher ICER for ACTH (₹122,185 per additional responder; ₹331,643 per QALY gained), reflecting greater upfront costs but improved clinical outcomes.
CONCLUSIONS: From an Indian healthcare perspective, ACTH demonstrated superior cost-effectiveness compared with oral prednisolone for short-term management of IESS, with better seizure control and favorable HRQoL outcomes despite higher treatment costs.
Conference/Value in Health Info
2026-05, ISPOR 2026, Philadelphia, PA, USA
Value in Health, Volume 29, Issue S6
Code
EE484
Topic
Economic Evaluation
Disease
SDC: Neurological Disorders, SDC: Pediatrics