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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