|
Gender
|
Male, female
|
|
Age at baseline
|
Year
|
|
Age at GD diagnosis
|
Year
|
|
Site of enrollment
|
Country
|
|
Acid beta-glucosidase genotype
|
–
|
|
Chitotriosidase genotype
|
Deficient[homozygous]; heterozygous; wild type
|
|
Previous history of enzyme replacement therapy at enrollment
|
Never treated with ERT, no ERT within the previous year, ERT within the previous year
|
|
Previous history of substrate reduction therapy at enrollment
|
Never treated with SRT, no SRT within the previous year, SRT within the previous year
|
|
Splenectomy
|
Yes, no
|
|
Time to FU visit
|
Month (0 for baseline)
|
|
Treatment received at FU
|
Placebo, untreated, imiglucerase, velaglucerase alpha, taliglucerase, miglustat, eliglustat, other
|
|
Plasma chitotriosidase activity at FU
|
nmol/mL/h
|
|
Serum CCL18 level at FU
|
ng/mL
|
|
Hemoglobin concentration at FU
|
g/dL
|
|
Platelet count at FU
|
109/L
|
|
White blood cell count at FU
|
109/L
|
|
Liver volume at FU
|
MN
|
|
Spleen volume at FU
|
MN
|
|
Previous history of bone event
|
Yes, no
|
|
Osteonecrosis (i.e., clinical history of bone crises with radiologic or magnetic resonance imaging confirmation) within the previous 12 months
|
Yes, no
|
|
Fracture with imaging confirmation within the previous 12 months
|
Yes, no
|
|
Skeletal site of fracture
|
Spine, hip, femur, knee, distal to the knee, shoulder, clavicle, humerus, elbow, distal to the elbow, rib, other
|
