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Table 4 Change in disease biomarkers over the 6–14-month observation period compared with those of healthy individuals

From: Clinical disease progression and biomarkers in Niemann–Pick disease type C: a prospective cohort study

  Skin unesterified cholesterol (μg/mg skin) PBMC unesterified cholesterol (μg/mg protein) Serum cholestane-triol (ng/mL) PBMC cholesterol esterification (ng/mg protein) PBMC HSP70 (pg/mL)
Visit 1, n 26 30 28 35 28
Mean (± SD) 2.79 (± 0.96) 77.56 (± 21.53) 88.31 (± 28.66) 462.14 (± 283.60) 1804.09 (± 713.67)
Visit 2, n 14 28 32 28 24
Mean (± SD) 1.89 (± 0.64) 98.45 (± 99.56) 88.52 (± 31.56) 616.89 (± 443.10) 1469.94 (± 581.21)
p value for the comparison between visits* 0.1748 0.8015 0.3069 0.1212 0.0121
Change in parameter per 6 months, n 11 23 26 27 20
Mean (± SD) –0.23 (± 0.47) 25.94 (± 97.56) 2.64 (± 10.69) 57.41 (± 303.81) –225.36 (± 411.76)
Healthy individuals, n 6 6 3 37 19
Mean (± SD) 0.36 (± 0.14) 76.39 (± 33.82) 5.97 (± 1.13) 1197.15 (± 763.65) 12,310.21 (± 4247.23)
p values compared with healthy individuals* 0.0006 0.8979 0.008  < 0.0001  < 0.0001
  1. *Comparisons between visits utilized the Wilcoxon–Mann–Whitney test. Comparisons between individuals with NPC and healthy individuals utilized the Wilcoxon signed-rank test
  2. HSP70: heat shock protein 70; PBMC: peripheral blood mononuclear cell; SD: standard deviation