Skip to main content

Table 2 Median percent decrease (range) of biochemical markers in treatment naïve patients

From: Biochemical response to substrate reduction therapy versus enzyme replacement therapy in Gaucher disease type 1 patients

  

Eliglustat

ERT

Miglustat

n = 4

n = 4

n = 2

Year

Median (range)

Median (range)

Median (range)

Chitotriosidase

1

68.3 (40–80.9)

79.6 (61.9–91.1)

41.8 (41.4–42.2)

2

88.8 (77–97.7)

88.1 (77.9–91.9)

37.3 (28.9–45.6)

CCL18

1

45.4 (35.3–52.9)

43.1 (21.5–65)

-9.6 (-22.1–2.9)

2

72.9 (62.7–78.3)

53.7 (43.4–85.7)

10.3 (2.8–17.7)

GlcSph

1

75.9 (62.4–78.3)

71.9 (20.6–81.3)

25.8 (23.8–27.7)

2

86.2 (78.4–92.9)

78.4 (64.7–90.6)

47.8 (45.4–50.2)

GlcCer

1

79.8 (76.4–89.4)

35.1 (-6.9–56.8)

37.6 (37.5–37.6)

2

84.1 (74.3–90.6)

36.4 (19–67.1)

39.5 (25.3–53.6)