Nilotinib treatment outcomes in autosomal dominant spinocerebellar ataxia over one year.

ABSTRACT: We evaluated the efficacy and safety of 1-year treatment with nilotinib (Tasigna^®) in patients with autosomal dominant spinocerebellar ataxia (ADSCA) and the factors associated with responsiveness. From an institutional cohort, patients with ADSCA who completed a 1-year treatment with nilotinib (150-300 mg/day) were included. Ataxia severity was assessed using the Scale for the Rating and Assessment of Ataxia (SARA), scores at baseline and 1, 3, 6, and 12 months. A subject was categorized 'responsive' when the SARA score reduction at 12 M was > 0. Pretreatment serum proteomic analysis included subjects with the highest (n = 5) and lowest (n = 5) SARA score change at 12 months and five non-ataxia controls. Thirty-two subjects (18 [56.2%] females, median age 42 [30-49.5] years) we

SUBMITTER: Lee WJ 

PROVIDER: S-EPMC11251258 | biostudies-literature | 2024 Jul

REPOSITORIES: biostudies-literature