Age at treatment initiation, months
|
Median (range)
|
3.0 (1.1–5.8)
|
Gender, n (%)
|
Male
|
5 (56)
|
Female
|
4 (44)
|
Race
|
White
|
4 (44)
|
Black
|
1 (11)
|
Asian
|
1 (11)
|
Unknown*
|
3 (33)
|
Age at symptom onset, months
|
Range
|
0–5.0
|
Age at diagnosis, months
|
Range
|
0–5.8
|
LAL deficiency manifestations, n (%)
|
Hepatosplenomegaly
|
9 (100)
|
Abdominal distension
|
9 (100)
|
Vomiting
|
9 (100)
|
Diarrhea
|
9 (100)
|
Adrenal calcifications
|
9 (100)
|
Failure to thrive
|
9 (100)
|
Anemia
|
6 (67)
|
Ascites
|
4 (44)
|
Thrombocytopenia (<150 × 109/L)
|
3 (33)
|
Hematological parameters, median (range)
|
Hemoglobin, g/L
|
93 (1.4–103.0)
|
Platelets, 109/L
|
173 (2.6–563)
|
Serum ferritin, μg/L, median (range)
|
586 (253–48,740)
|
Multiple organ dysfunction syndrome, n (%)
|
3 (33)
|
Growth failure/entry criteria met,†
n (%)
|
Weight decreasing across ≥2 of the 11 major centiles
|
7 (78)
|
Body weight <10th centile and no weight increase during 2 weeks before screening
|
1 (11)
|
Loss of >5% of birth weight after 2 weeks of age
|
0
|
Rapidly progressive course of LAL deficiency without meeting growth failure criteria
|
1 (11)
|