Evaluation of miglustat as maintenance therapy after enzyme therapy in adults with stable type 1 Gaucher disease: a prospective, open-label non-inferiority study

Table 1 Patient demographics and characteristics at baseline (all enrolled population)

	All patients (N = 42)
Demographics:
Age, years, mean ± SD	45.1 ± 12.7
Gender, male: female, n	22: 20
Body weight, kg, mean ± SD	76.7 ± 16.1
Height in cm, mean ± SD	169.9 ± 9.0
Race, n (%):
Caucasian	38 (90.5)
Asian	1 (2.4)
Hispanic	2 (4.8)
Other	1 (2.4)
Treatment history, mean ± SD:
Age at diagnosis, years	24.8 ± 15.0
Age at enzyme therapy initiation, years	35.6 ± 12.5
Duration of enzyme therapy, years	9.5 ± 4.0
Duration of current dose of enzyme therapy, years*	4.9 ± 2.8
Enzyme therapy dose at switch, IU/kg/month	61.3 ± 35.7
Disease parameters, mean ± SD:
Liver volume in mL (n = 40)	1726 ± 459.6
Liver volume in MN (n = 40)	0.91 ± 0.22
Spleen volume^† in mL (n = 30)	484.7 ± 333.2
Spleen volume^† in MN (n = 30)	3.13 ± 1.76
Hemoglobin, g/dL (n = 41)	14.89 ± 1.49
Platelet count, × 10⁹/L (n = 41)	193.8 ± 92.06

*Median (range) dose of enzyme therapy 54.7 (5.2–130.7) IU/kg/month; ^†excludes splenectomized patients; MN, multiples of normal.

ISSN: 1750-1172