Responder analysis
neuropathic progression
Responder analysis was conducted to evaluate the efficacy of inotersen for slowing neuropathic progression at the patient level. For each NIS and NIS-LL measure, the proportion of patients classified as responders (i.e., patients whose score increased by less than the recommended RD threshold after 65 weeks of treatment) were compared between treatment groups using odds ratios (OR) with 95% confidence intervals (CI), and Fisher’s exact tests (two-tailed α) for statistical significance. Because these analyses were exploratory, no adjustments were made to the familywise Type 1 error rate for multiple comparisons.To be consistent with the efficacy analysis of NIS/NIS-LL in the NEURO-TTR study, no imputation of missing values was performed in the primary responder analysis. As such, the responder analysis used a complete-case analysis, in which only patients with non-missing scores at the week 65 visit were included.