Table 1 Demographics and clinical characteristics of study patients
Patient characteristic | Drug Registry cohort (N = 106) | |
|---|---|---|
Sex, n (%) | ||
Male | 49 (46.2) | |
Female | 57 (53.8) | |
Age category (years) | ||
< 12 | 8 (7.5) | |
12–< 18 | 8 (7.5) | |
18–44 | 36 (34.0) | |
> 44–64 | 32 (30.2) | |
≥ 65 | 22 (20.8) | |
Mean age (years) | ||
< 18 | 11.2 | |
≥ 18 | 49.7 | |
Race, n (%) | ||
White | 94 (88.7) | |
Black | 1 (0.9) | |
Other | 11 (10.4) | |
Ethnicity, n (%) | ||
Ashkenazi Jewish | 61 (57.5) | |
Geographic location, n (%) | ||
Israel | 53 (50.0) | |
United States | 28 (26.4) | |
Albania | 25 (23.6) | |
Employment status, n (%) | ||
Currently working (adult patients only, n = 90) | 47 (52.2) | |
Gaucher disease diagnosis, n (%) | ||
Type 1 | 102 (96.2) | |
Type 2 | 0 (0.0) | |
Type 3 | 4 (3.8) | |
Symptoms at Gaucher disease diagnosis, n (%) | ||
Splenomegaly | 86 (81.1) | |
Thrombocytopenia | 63 (59.4) | |
Hepatomegaly | 49 (46.2) | |
Anemia | 45 (42.5) | |
Fatigue | 39 (36.8) | |
Bone pain | 36 (34.0) | |
Other symptoms | 29 (27.4) | |
Comorbidities/conditions at enrollment, n (%) | ||
Surgical and medical procedures | 31 (29.2) | |
Vascular disorders including hemorrhage | 31 (29.2) | |
Musculoskeletal/connective tissue disorders, including osteoporosis | 29 (27.4) | |
Cardiac disorders | 25 (23.6) | |
Hepatobiliary disorders | 12 (11.3) | |
Psychiatric disorders | 10 (9.4) | |
Splenectomy at enrollment, n (%) | ||
Yes | 14 (13.2) | |
Historical ERT, n (%) | ||
Taliglucerase alfa | 94 (95.9) | |
Imiglucerase | 57 (58.2) | |
Velaglucerase alfa | 14 (14.3) | |
Alglucerase | 3 (3.1) | |