Table 1.
Patient disposition and demographics
| Parameter | Measure |
|---|---|
| Number of patients treated | 33 |
| Number of patients not completing study, n (%) | 10 (30) |
| Principal reason for withdrawal, n (%) | |
| Adverse events | 1 (3) |
| Uncooperative (could not make visits) | 1 (3) |
| Wishes to withdraw | 3 (9) |
| Sponsor ended study | 3 (9) |
| Others (lung cancer and circumstances prevented further participation) | 2 (6) |
| Study treatment duration, months, median (range) | 49.4 (4.2–58.6) |
| Time of last efficacy visit, n (%) | |
| ≤12 months | 6 (18) |
| >12 to ≤24 months | 0 |
| >24 to ≤36 months | 4 (12) |
| >36 to 48 months | 23 (70) |
| Gender, n (%) | |
| Male | 19 (58) |
| Female | 14 (42) |
| Ethnicity, n (%) | |
| Ashkenazi Jewish | 23 (70) |
| Non-Jewish Caucasian | 6 (18) |
| African-American/Caribbean | 1 (3) |
| Hispanic | 2 (6) |
| American Indian | 1 (3) |
| Others | 2 (6) |
| Genotype, n (%) | |
| N370S N370S | 22 (67) |
| N370S-L444P | 6 (18) |
| N370S-D409H | 1 (3) |
| N370S-IVS2+1 | 1 (3) |
| N370S-unknown | 2 (6) |
| Unknown | 1 (3) |
| Patients with total splenectomy, n (%) | 5 (15) |
| Median age at Gaucher symptom onset, years (range) | 32.0 (11.0–52.0) |
| Median age at consent, years (range) | 43.0 (12.0–70.0) |
| Median weight at baseline, kg (range) | 64.2 (34.0–105.0) |
| Median height at baseline, cm (range) | 169.9 (146.5–190.5) |