Vigabatrin versus ACTH as first-line treatment for infantile spasms: a randomized, prospective study
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1Department of Child Neurology, Bambino Gesù Children's Hospital, Rome, Italy.
Insights
Vigabatrin (VGB) and adrenocorticotrophic hormone (ACTH) were compared for infantile spasms. VGB showed comparable efficacy to ACTH with fewer side effects, suggesting it as a potential first-line treatment.
Area of Science:
- Pediatric Neurology
- Pharmacology
- Clinical Trials
Background:
- Infantile spasms (IS) are a severe form of epilepsy in infants.
- First-line treatment options for IS include vigabatrin (VGB) and adrenocorticotrophic hormone (ACTH).
- Comparative efficacy and tolerability data are crucial for optimal treatment selection.
Purpose of the Study:
- To compare the efficacy and tolerability of vigabatrin (VGB) and adrenocorticotrophic hormone (ACTH) as first-line therapy for infantile spasms (IS).
Main Methods:
- A randomized trial involving 42 infants (aged 2-9 months) with newly diagnosed infantile spasms.
- Patients were randomized to receive either VGB (100-150 mg/kg/day) or Depot ACTH (10 IU/day).
- Alternative drug administration was permitted for non-responders or intolerant patients.
Main Results:
- Cessation of spasms was achieved in 48% of VGB-treated patients and 74% of ACTH-treated patients.
- VGB exhibited fewer side effects (13%) compared to ACTH (37%).
- VGB was more effective for spasms associated with cerebral malformations or tuberous sclerosis, while ACTH was more effective for perinatal hypoxic/ischemic injury.
Conclusions:
- Vigabatrin (VGB) demonstrates significant efficacy and better tolerability compared to ACTH in treating infantile spasms.
- VGB may be considered a viable first-choice drug for infantile spasms, particularly in specific etiologies.
- Further research may explore optimal VGB dosing and long-term outcomes.
Purpose:
To compare the efficacy and tolerability of vigabatrin (VGB) and adrenocorticotrophic hormone (ACTH) as first-line therapy in infantile spasms.
Methods:
Forty-two infants (22 males, 20 females) aged 2-9 months with newly diagnosed infantile spasms, were included in the trial. Patients were randomized to receive VGB 100-150 mg/kg/day or Depot ACTH 10 IU/day. The alternative drug was given if spasms were not controlled within 20 days or in cases of intolerance to initial therapy. Twenty-three patients (7 cryptogenic, 16 symptomatic) received VGB as first-line therapy; 19 patients (8 cryptogenic, 11 symptomatic) received ACTH as the first drug.
Results:
Cessation of spasms was observed in 11 (48%) of the patients randomized to VGB and in 14 (74%) of those randomized to ACTH. Response to VGB was observed within 1-14 days, but two-thirds of patients (7/11) responded within 3 days. In the group treated with VGB, side effects such as drowsiness, hypotonia and irritability were observed in 13% of patients, compared with 37% in the group treated with ACTH. VGB was more effective than ACTH as treatment for cerebral malformations or tuberous sclerosis, whereas ACTH proved more effective in perinatal hypoxic/ischemic injury. The efficacy of the two drugs was similar in cryptogenic cases. Disappearance of interictal EEG abnormalities occurred sooner in patients randomized to ACTH than in those who received VGB as initial therapy. During the second phase, the alternative drug was given to the resistant patients. Spasms ceased in 2 of 5 patients treated with VGB and in 11 of 12 patients treated with ACTH. After 3 months, relapses of spasms were observed in 6 patients treated with ACTH and in 1 treated with VGB. VGB produced a therapeutic response in nearly half the patients receiving this drug.
Conclusions:
Our data lend further support to the view that VGB may be considered a first-choice drug in the treatment of IS.
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