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Table 2 Lipid profile, markers of lipoprotein quality and functionality, and markers of inflammation of patients with Niemann–Pick disease type B before and after ERT

From: Changes in PCSK 9 and apolipoprotein B100 in Niemann–Pick disease after enzyme replacement therapy with olipudase alfa

 

Niemann–Pick disease type B (n = 5)

Baseline

Week 26

Week 26 change from baseline

Week 26 % change from baseline

Total cholesterol (mmol/l)

4.5 ± 0.8

3.6 ± 0.7

− 0.9 ± 1.0*

− 18.3 ± 20.4*

Triglyceride (mmol/l)

2.2 ± 1.5

1.0 ± 0.4

− 1.2 ± 1.1*

− 46.9 ± 17.3*

HDL-C (mmol/l)

0.79 ± 0.28

0.74 ± 0.24

− 0.04 ± 0.18*

− 3.1 ± 23.3*

LDL-C (mmol/l)

2.9 ± 0.5

2.4 ± 0.5

− 0.5 ± 0.7*

− 16.2 ± 22.1*

ApoA1 (g/l)

0.84 ± 0.25

0.89 ± 0.20

0.05 ± 0.06

8.0 ± 13.3

ApoB100 (g/l)

0.94 ± 0.21

0.75 ± 0.15

− 0.18 ± 0.26

− 18.4 ± 20.4

ApoB48 (µg/ml)

89.8 ± 11.9

92.51 ± 27.3

7.29 ± 12.6

1.2 ± 19.7

sdLDL-C (mg/dl)

44.7 ± 18.2

32.5 ± 13.6

− 12.2 ± 11.2*

− 24.0 ± 29.1*

oxLDL (ng/ml)

131.1 ± 22.8

123.4 ± 37.9

− 14.4 ± 20.8

− 6.7 ± 19.2

PCSK9 mass (ng/ml)

599.0 ± 237.6

492.4 ± 97.6

− 220.8 ± 253.1*

− 10.0 ± 29.1*

TNF-α (pg/ml)

76.0 ± 10.7

65.6 ± 6.5

− 32.7 ± 21.7

− 22.3 ± 7.0

  1. Data are presented as mean ± SD
  2. apoA1, apolipoprotein A1; apoB-100, apolipoprotein B-100; apoB48, apolipoprotein B48; HDL-C, high-density lipoprotein cholesterol; LDL-C, low-density lipoprotein cholesterol; oxLDL, oxidised low-density lipoprotein; PCSK9, proprotein convertase subtilisin/kexin type 9; sdLDL-C, small dense low-density lipoprotein cholesterol; TNF-α, tumour necrosis factor α
  3. *Values represent pre-dose measured in serum
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Orphanet Journal of Rare Diseases

ISSN: 1750-1172