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Table 9 Summary table of anchor-based NPCCSS results for the CGI-I

From: Validation of the 5-domain Niemann-Pick type C Clinical Severity Scale

Visit Collapsed / Uncollapsed Anchor Category N Mean Change (SD) 95% CI Effect Size Median Change
Visit 1 to 6 Collapsed No Change 18 0.83 (2.176) (-0.249, 1.915) 0.38 0.00
Worsening 13 2.69 (3.225) (0.744, 4.641) 0.83 2.00
  1. Abbreviations: CGI-I = Clinician Global Impression of Improvement; CI = confidence interval; NPCCSS = Niemann-Pick type C Clinical Severity Scale; SD = standard deviation
  2. Visit 1 = Screening; Visit 3 = 3 months; Visit 4 = 6 months; Visit 5 = 9 months; Visit 6 = 12 months
  3. All patients in the study (age range 2–19 years) were assessed using the CGI-I