Parameter | VITAL (N = 9) | CL08 (N = 10) |
---|---|---|
Age at first dose, months, median (range) | 3.0 (1–6) | 2.8 (0.5–4) |
Sex, n (%) | ||
Male | 5 (56) | 5 (50) |
Female | 4 (44) | 5 (50) |
Race, n (%) | ||
American Indian or Alaska Native | 0 | 1 (10) |
Asian | 1 (11) | 6 (60) |
Black | 1 (11) | 0 |
White | 4 (44) | 1 (10) |
Other | 0 | 2 (20) |
Unknown | 3 (33) | 0 |
Anthropometric parameters, median (IQR) | ||
Weight-for-age, WHO percentilea | 3.1 (1–8)b | 1.1 (0.0–8) |
Length (height)-for-age, WHO percentilea | 1.8 (0.1–39)b | 2.9 (0.6–50)c |
Mid-upper arm circumference-for-age, WHO percentilea | 0.01 (0.0–0.3)d | 0.001 (0.0–0.3)e |
Liver parameters, median (range) | ||
ALT | ||
U/L | 145.0 (16–297) | 37.0 (28–248)c |
µkat/L | 2.42 (0.3–5.0) | 0.62 (0.5–4.1)c |
AST | ||
U/L | 125.0 (71–716) | 99.5 (56–441)b |
µkat/L | 2.09 (1.2–12.0) | 1.66 (0.9–7.4)b |
Ferritin | ||
µg/L (ng/mL) | 586.3 (253–48,740)f | 1750.5 (481–3020)g |
Albumin, g/L | 29.0 (13–40) | 20.0 (18–29)c |
Total bilirubin | ||
mg/dL | 1.7 (0.2–27) | 0.7 (0.2–3) |
µmol/L | 29.0 (3–464)b | 12.0 (4–52)c |
Hematologic parameters, median (range) | ||
Hemoglobin, g/L | 93.0 (1–103) | 90.0 (81–131)f |
Platelets, 109/L | 173.0 (3–563) | 146.0 (56–235)f |
LAL-D manifestations, n (%) | ||
Hepatomegaly and/or splenomegaly | 9 (100) | 9 (90) |
Diarrhea and/or vomiting | 9 (100) | 9 (90) |
Adrenal calcification | 9 (100) | 5 (50) |
Failure to thrive/malnutrition | 9 (100) | 5 (50) |
Liver volume, MN, median (range) | 3.4 (3–4)h | 3.2 (0.1–4)f |
Spleen volume, MN, median (range) | 7.0 (3–10)g | 5.8 (0.7–15)b |