Combination Therapy With Vigabatrin and Prednisolone Versus Vigabatrin Alone for Infantile Spasms

ABSTRACT Objective The study evaluated the effectiveness of combination therapy with vigabatrin and prednisolone versus vigabatrin alone for treating infantile epileptic spasms syndrome (IESS). Methods This single‐center, single‐blind, randomized trial enrolled infants aged 2–14 months with new‐onse...

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Main Authors: Rachata Boonkrongsak, Kantapon Trongkamolchai, Sirorat Suwannachote, Somjit Sri‐Udomkajorn, Raviwan Wittawassamrankul, Ravindra Arya, Kullasate Sakpichaisakul
Format: Article
Language:English
Published: Wiley 2025-05-01
Series:Annals of Clinical and Translational Neurology
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Online Access:https://doi.org/10.1002/acn3.70034
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author Rachata Boonkrongsak
Kantapon Trongkamolchai
Sirorat Suwannachote
Somjit Sri‐Udomkajorn
Raviwan Wittawassamrankul
Ravindra Arya
Kullasate Sakpichaisakul
author_facet Rachata Boonkrongsak
Kantapon Trongkamolchai
Sirorat Suwannachote
Somjit Sri‐Udomkajorn
Raviwan Wittawassamrankul
Ravindra Arya
Kullasate Sakpichaisakul
author_sort Rachata Boonkrongsak
collection DOAJ
description ABSTRACT Objective The study evaluated the effectiveness of combination therapy with vigabatrin and prednisolone versus vigabatrin alone for treating infantile epileptic spasms syndrome (IESS). Methods This single‐center, single‐blind, randomized trial enrolled infants aged 2–14 months with new‐onset IESS, randomly assigned them (1:1) to receive either combination therapy with vigabatrin (100–150 mg/kg/day) and prednisolone (40–60 mg/day) or vigabatrin alone. The primary outcome was sustained spasm remission between days 14 and 42. The trial (ClinicalTrials.gov identifier) was terminated early due to an interim analysis revealing a significant difference between treatment groups. Results In all, 17 infants were assigned to combination therapy and 24 to vigabatrin alone. The median lead time to treatment for combination therapy was 30 days, compared to 60 days for the vigabatrin group (p = 0.442). Sustained spasm remission between days 14 and 42 occurred in 13 (77%) of 17 infants on combination therapy and in 8 (33%) of 24 infants on vigabatrin alone (OR 6.5, 95% CI 1.7, 29.6, p = 0.009). Combination therapy was more effective in achieving an electroclinical response by day 14 (OR 9.3, 95% CI 2.0, 54.3, p = 0.006) but not by day 42 (OR 1.9, 95% CI 0.5, 7.1, p = 0.351). Hospitalization occurred in six (35%) infants in the combination therapy group and two (8%) in the vigabatrin alone group (p = 0.05). One death was reported in the vigabatrin group. Interpretation Despite early termination, this study showed that combination therapy is significantly more effective in achieving clinical remission of spasms and an electroclinical response by day 14. Trial Registration NCT04302116
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spelling doaj-art-4ff025c93f98422a8229d99db5eec4cf2025-08-20T03:08:21ZengWileyAnnals of Clinical and Translational Neurology2328-95032025-05-011251012102110.1002/acn3.70034Combination Therapy With Vigabatrin and Prednisolone Versus Vigabatrin Alone for Infantile SpasmsRachata Boonkrongsak0Kantapon Trongkamolchai1Sirorat Suwannachote2Somjit Sri‐Udomkajorn3Raviwan Wittawassamrankul4Ravindra Arya5Kullasate Sakpichaisakul6Division of Neurology, Department of Pediatrics Queen Sirikit National Institute of Child Health, Ministry of Public Health Bangkok ThailandDivision of Neurology, Department of Pediatrics Queen Sirikit National Institute of Child Health, Ministry of Public Health Bangkok ThailandDivision of Neurology, Department of Pediatrics Queen Sirikit National Institute of Child Health, Ministry of Public Health Bangkok ThailandDivision of Neurology, Department of Pediatrics Queen Sirikit National Institute of Child Health, Ministry of Public Health Bangkok ThailandDepartment of Pharmacy Queen Sirikit National Institute of Child Health, Ministry of Public Health Bangkok ThailandComprehensive Epilepsy Center, Division of Neurology, Cincinnati Children's Hospital Medical Center Cincinnati Ohio USADivision of Neurology, Department of Pediatrics Queen Sirikit National Institute of Child Health, Ministry of Public Health Bangkok ThailandABSTRACT Objective The study evaluated the effectiveness of combination therapy with vigabatrin and prednisolone versus vigabatrin alone for treating infantile epileptic spasms syndrome (IESS). Methods This single‐center, single‐blind, randomized trial enrolled infants aged 2–14 months with new‐onset IESS, randomly assigned them (1:1) to receive either combination therapy with vigabatrin (100–150 mg/kg/day) and prednisolone (40–60 mg/day) or vigabatrin alone. The primary outcome was sustained spasm remission between days 14 and 42. The trial (ClinicalTrials.gov identifier) was terminated early due to an interim analysis revealing a significant difference between treatment groups. Results In all, 17 infants were assigned to combination therapy and 24 to vigabatrin alone. The median lead time to treatment for combination therapy was 30 days, compared to 60 days for the vigabatrin group (p = 0.442). Sustained spasm remission between days 14 and 42 occurred in 13 (77%) of 17 infants on combination therapy and in 8 (33%) of 24 infants on vigabatrin alone (OR 6.5, 95% CI 1.7, 29.6, p = 0.009). Combination therapy was more effective in achieving an electroclinical response by day 14 (OR 9.3, 95% CI 2.0, 54.3, p = 0.006) but not by day 42 (OR 1.9, 95% CI 0.5, 7.1, p = 0.351). Hospitalization occurred in six (35%) infants in the combination therapy group and two (8%) in the vigabatrin alone group (p = 0.05). One death was reported in the vigabatrin group. Interpretation Despite early termination, this study showed that combination therapy is significantly more effective in achieving clinical remission of spasms and an electroclinical response by day 14. Trial Registration NCT04302116https://doi.org/10.1002/acn3.70034combination therapyinfantile epileptic spasms syndromeinfantile spasmsvigabatrinwest syndrome
spellingShingle Rachata Boonkrongsak
Kantapon Trongkamolchai
Sirorat Suwannachote
Somjit Sri‐Udomkajorn
Raviwan Wittawassamrankul
Ravindra Arya
Kullasate Sakpichaisakul
Combination Therapy With Vigabatrin and Prednisolone Versus Vigabatrin Alone for Infantile Spasms
Annals of Clinical and Translational Neurology
combination therapy
infantile epileptic spasms syndrome
infantile spasms
vigabatrin
west syndrome
title Combination Therapy With Vigabatrin and Prednisolone Versus Vigabatrin Alone for Infantile Spasms
title_full Combination Therapy With Vigabatrin and Prednisolone Versus Vigabatrin Alone for Infantile Spasms
title_fullStr Combination Therapy With Vigabatrin and Prednisolone Versus Vigabatrin Alone for Infantile Spasms
title_full_unstemmed Combination Therapy With Vigabatrin and Prednisolone Versus Vigabatrin Alone for Infantile Spasms
title_short Combination Therapy With Vigabatrin and Prednisolone Versus Vigabatrin Alone for Infantile Spasms
title_sort combination therapy with vigabatrin and prednisolone versus vigabatrin alone for infantile spasms
topic combination therapy
infantile epileptic spasms syndrome
infantile spasms
vigabatrin
west syndrome
url https://doi.org/10.1002/acn3.70034
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