Early Treatment with Vigabatrin Does Not Decrease Focal Seizures or Improve Cognition in Tuberous Sclerosis Complex: The PREVeNT Trial.

OBJECTIVE: This study was undertaken to test the hypothesis that early vigabatrin treatment in tuberous sclerosis complex (TSC) infants improves neurocognitive outcome at 24 months of age. METHODS: A phase IIb multicenter randomized double-blind placebo-controlled trial was conducted of vigabatrin at first epileptiform electroencephalogram (EEG) versus vigabatrin at seizure onset in infants with TSC. Primary outcome was Bayley Scales of Infant and Toddler Development, Third Edition (Bayley-III) cognitive assessment score at 24 months. Secondary outcomes were prevalence of drug-resistant epilepsy, additional developmental outcomes, and safety of vigabatrin. RESULTS: Of 84 infants enrolled, 12 were screen failures, 4 went straight to open label vigabatrin, and 12 were not randomized (normal EEG throughout). Fifty-six were randomized to early vigabatrin (n = 29) or placebo (n = 27). Nineteen of 27 in the placebo arm transitioned to open label vigabatrin, with a median delay of 44 days after randomization. Bayley-III cognitive composite scores at 24 months were similar for participants randomized to vigabatrin or placebo. Additionally, no significant differences were found between groups in overall epilepsy incidence and drug-resistant epilepsy at 24 months, time to first seizure after randomization, and secondary developmental outcomes. Incidence of infantile spasms was lower and time to spasms after randomization was later in the vigabatrin group. Adverse events were similar across groups. INTERPRETATION: Preventative treatment with vigabatrin based on EEG epileptiform activity prior to seizure onset does not improve neurocognitive outcome at 24 months in TSC children, nor does it delay onset or lower the incidence of focal seizures and drug-resistant epilepsy at 24 months. Preventative vigabatrin was associated with later time to onset and lower incidence of infantile spasms. ANN NEUROL 2023.

Attitudes and Beliefs Toward Thymectomy in the Myasthenia Gravis Patient Registry.

OBJECTIVES: To evaluate patient attitudes and beliefs toward thymectomy for myasthenia gravis (MG). METHODS: The Myasthenia Gravis Foundation of America administered a questionnaire to the MG Patient Registry, an ongoing longitudinal survey of adult MG patients. Questions assessed reasons for or against thymectomy and how hypothetical scenarios would have affected their decision. RESULTS: Of 621 respondents, 190 (31%) reported a history of thymectomy. Of those who underwent thymectomy for nonthymomatous MG, 97 (51.6%) ranked symptom improvement as most important and 100 (53.2%) ranked reducing medication as least important. Among 431 nonthymectomy patients, the most frequent reason for not undergoing thymectomy was that their doctor did not discuss it (152 of 431 = 35.2%) and 235 (56.8%) said that they would have considered it more strongly if their doctor spent more time discussing it. CONCLUSIONS: Thymectomies are motivated more by symptoms than by medication, and a lack of neurologist discussion is the most common barrier to thymectomy.

Neurocognitive Predictors of Declining Financial Capacity in Persons with Mild Cognitive Impairment.

OBJECTIVE: To identify cognitive predictors of declining financial capacity (FC) in persons with mild cognitive impairment (MCI). METHODS: Participants were 66 cognitively normal older adults and 49 persons with MCI who completed neuropsychological testing and a performance measure of financial capacity (Financial Capacity Instrument; FCI) at baseline and two-year follow-up. We calculated two-year change scores for neuropsychological tests and FCI total score. We examined bivariate correlations between demographic/clinical variables and FCI change score, and between neuropsychological and FCI change scores. The five strongest bivariate correlates were entered into a linear regression analysis to identify longitudinal predictors of financial decline within group. RESULTS: Persons with MCI showed significant decline on the FCI and most cognitive variables, while controls demonstrated relatively stable performance. For persons with MCI, education correlated with FCI change score. The top four cognitive variable-FCI change score correlations were written arithmetic, confrontation naming, immediate visual memory, and visual attention. In the regression model, written arithmetic was the primary predictor and visual memory and visual attention were secondary predictors of two-year FCI change scores. CONCLUSION: Semantic arithmetic knowledge, and to a lesser extent visual memory and attention, are key longitudinal cognitive predictors of financial skill decline in individuals with MCI. CLINICAL IMPLICATIONS: Clinicians should consider neurocognitive abilities of written arithmetic, visual memory, and processing speed in their assessments of financial capacity in person with MCI.