Skip to main content

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)