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) | |
ERT enzyme replacement therapy