Vigabatrin therapy for infantile spasms: review of major trials in Europe, Canada, and the United States; and recommendations for dosing
Bibliographic record
Abstract
Infantile spasms (IS) are a unique and severe form of epilepsy associated with poor neurologic and developmental outcomes. The refractory spasms and abnormal electroencephalogram (EEG) patterns associated with the condition are believed to have a progressively detrimental impact. Therefore, rapid and complete control of spasms is the primary goal of treatment. Well-controlled clinical trials in Europe, Canada, and the United States have demonstrated that vigabatrin is efficacious and generally well-tolerated as monotherapy for IS. Several key studies, including pivotal trials that led to United States approval of vigabatrin in 2009, as well as comparative trials of vigabatrin and hormonal treatment, are the focus of this review. All studies assessed spasm cessation - usually as the primary endpoint - and adverse events. Vigabatrin dosages generally ranging from 100 to 150 mg/kg/day demonstrated efficacy to decrease or eradicate spasms and eliminate hypsarrhythmic EEG in patients with newly diagnosed IS. Several studies demonstrated long-term sustainability of spasm freedom with no negative impact on developmental outcomes. Vigabatrin was generally well-tolerated with few severe adverse events. Visual field defects cannot be adequately assessed in infants and young children, so this potential adverse effect was not evaluated in children with spasms. Notably, the time to response with vigabatrin was very rapid, generally occurring within 2 weeks of initial treatment. This allows for early treatment modification as needed. For infants who respond well to vigabatrin, treatment duration up to 6 months appears to be appropriate for realizing spasm freedom while limiting potential risks of adverse events and recurrences.
Fetched live from OpenAlex and de-inverted. Abstracts are not stored in this database: the inverted indexes are 8.6 GB of the frame’s 9.3 GB of text, and the host has 13 GB free.
How this classification was reachedexpand
Full frame machine prediction
Teacher imitationNot calibrated prevalence, not ground truth. Human validation pending. The Gemma side is a direct model label for every work in the frame, read from the title-only record. The Codex side is a classifier learned from the 10,348 direct Codex labels and calibrated to design-weighted sample rates; fields without enough sample support carry no Codex call. Candidate is the union of the two sides; consensus is their intersection. These outputs are machine_predicted_unvalidated and are not human labels.
Distilled classifier scores by category (both heads)
| Category | Codex | Gemma |
|---|---|---|
| Metaresearch | 0.003 | 0.007 |
| Meta-epidemiology (narrow) | 0.001 | 0.000 |
| Meta-epidemiology (broad) | 0.003 | 0.003 |
| Bibliometrics | 0.004 | 0.005 |
| Science and technology studies | 0.000 | 0.001 |
| Scholarly communication | 0.002 | 0.001 |
| Open science | 0.002 | 0.001 |
| Research integrity | 0.001 | 0.002 |
| Insufficient payload (model declined to judge) | 0.003 | 0.001 |
Machine scores (provisional)
The two teacher heads of the student model, read on this work. A score orders the frame for review; it never asserts a category, and the validation status ships verbatim with every row.
Baseline scores from an immature model (maturity gate not passed, 7 training rounds). Scores rank; they never assert a category.
score_only:v0-immature-baseline · verbatim from the scoring run: score_only means the number may rank works, and no category label ships from itClassification
machine, unvalidatedMachine predicted; a candidate call from one source (direct Gemma or distilled Codex), not a consensus.
How this classification was reached, model by model and score by score, is at the end of the page under "How this classification was reached".