Table 1 Requested variables
Variable | Description |
|---|---|
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 |