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Table 2 Liver, hematologic, and lipid effects

From: Long-term survival with sebelipase alfa enzyme replacement therapy in infants with rapidly progressive lysosomal acid lipase deficiency: final results from 2 open-label studies

Parameter

VITAL (N = 9)

CL08 (N = 10)

Baseline (n = 9),

median (range)

End of studya (n = 4),

median (range)

Baseline (n = 9),

median (range)

End of studyb (n = 5),

median (range)

ALT

    

 U/L

145.0 (16–297)

26.5 (18–38)

37.0 (28–248)

29.0 (22–106)

 µkat/L

2.42 (0.3–5.0)

0.44 (0.3–0.6)

0.62 (0.5–4.1)

0.48 (0.4–1.8)

AST

    

 U/L

125.0 (71–716)

44.5 (41–54)

99.5 (56–441)c

44.0 (38–110)

 µkat/L

2.09 (1.2–12.0)

0.74 (0.7–0.9)

1.66 (0.9–7.4)c

0.73 (0.6–1.8)

Ferritin

    

 µg/L (ng/mL)

586.3 (253–48,740)d

93.5 (42–123)

1750.5 (481–3020)e

62.1 (49–75)

 Albumin, g/L

29.0 (13–40)

32.0 (27–37)

20.0 (18–29)

33.0 (20–37)f

 Hemoglobin, g/L

93.0 (1–103)

115.5 (99–123)

90.0 (81–131)d

113.0 (103–129)f

Total cholesterol

    

 mg/dL

139.2 (67–225)g

112.1 (93–131)h

125.7 (97–1063)i

106.3 (85–205)f

 mmol/L

3.6 (2–6)g

2.9 (2–3)h

3.3 (3–28)i

2.8 (2–5)f

LDL-C

    

 mg/dL

109.4 (19–194)g

64.2 (63–75)h

119.0 (62–143)h

76.6 (53–137)h

 mmol/L

2.8 (0.5–5)g

1.7 (2–2)h

3.1 (2–4)h

2.0 (1–4)h

HDL-C

    

 mg/dL

8.9 (0–10)g

18.9 (13–19)h

9.4 (8–12)f

13.1 (13–29)h

 mmol/L

0.2 (0.0–0.3)g

0.5 (0.3–0.5)h

0.2 (0.2–0.3)f

0.3 (0.3–0.8)h

Triglycerides

    

 mg/dL

163.9 (31–218)g

99.2 (90–237)h

265.7 (71–424)g

151.1 (133–195)h

 mmol/L

1.9 (0.4–3)g

1.1 (1–3)h

3.0 (0.8–5)g

1.7 (2–2)h

 Liver volume, MN

3.4 (3–4)h

1.6 (0.3–3)h,j

3.2 (0.1–4)d

1.9 (1–2)e

 Spleen volume, MN

7.0 (3–11)e

2.6 (2–7)h,j

5.8 (0.7–15)c

4.0 (2–6)e

  1. ALT alanine aminotransferase, AST aspartate aminotransferase, HDL-C high-density lipoprotein cholesterol; LDL-C, low-density lipoprotein cholesterol; MN, multiples of normal
  2. aWeek 240 (month 60; last visit with n > 2)
  3. bWeek 156 (month 39)
  4. cn = 8
  5. dn = 7
  6. en = 2
  7. fn = 4
  8. gn = 5
  9. hn = 3
  10. in = 6
  11. jWeek 120 (month 30; last visit with n > 1)