Demography and baseline characteristics at commencement of the 6-month randomized trial (safety population)
| Characteristic . | Miglustat . | Imiglucerase . | Combination . | Total . |
|---|---|---|---|---|
| n | 12 | 12 | 12 | 36 |
| Sex, no. (%) | ||||
| Male | 5 (42) | 6 (50) | 5 (42) | 16 (44) |
| Female | 7 (58) | 6 (50) | 7 (58) | 20 (56) |
| Race, no. (%) | ||||
| Ashkenazi Jew | 11 (92) | 12 (100) | 10 (83) | 33 (92) |
| Other | 1 (8) | 0 | 2 (17) | 3 (8) |
| Age, y | ||||
| Mean (SD) | 34.6 (11.1) | 40.4 (15.7) | 36.7 (13.4) | 37.2 (13.4) |
| Range | 18-49 | 19-69 | 17-55 | 17-69 |
| Weight, kg | ||||
| Mean (SD) | 60.80 (9.02) | 70.17 (15.09) | 70.59 (11.51) | 67.19 (12.64) |
| Range | 48.0-82.0 | 48.0-101.0 | 57.0-89.0 | 48.0-101.0 |
| Height, cm | ||||
| Mean (SD) | 163.6 (12.4) | 168.8 (8.7) | 169.8 (10.9) | 167.4 (10.8) |
| Range | 140-189 | 153-184 | 155-189 | 140-189 |
| Severity scoring index | ||||
| Mean (SD) | 9.17 (4.78) | 10.42 (4.01) | 9.00 (5.61) | 9.53 (4.75) |
| Range | 2.0-17.0 | 6.0-17.0 | 3.0-21.0 | 2.0-21.0 |
| Liver organ volume, L | n = 11 | n = 35 | ||
| Mean (SD) | 1.53 (0.32) | 1.81 (0.57) | 1.91 (0.42) | 1.74 (0.47) |
| Range | 1.0-2.1 | 1.0-2.6 | 1.3-2.6 | 1.0-2.6 |
| Spleen organ volume, L* | ||||
| Mean (SD) | 0.63 (0.43) | 0.74 (0.56) | 0.76 (0.37) | 0.71 (0.45) |
| Range | 0.2-1.4 | 0.2-1.8 | 0.3-1.2 | 0.2-1.8 |
| Characteristic . | Miglustat . | Imiglucerase . | Combination . | Total . |
|---|---|---|---|---|
| n | 12 | 12 | 12 | 36 |
| Sex, no. (%) | ||||
| Male | 5 (42) | 6 (50) | 5 (42) | 16 (44) |
| Female | 7 (58) | 6 (50) | 7 (58) | 20 (56) |
| Race, no. (%) | ||||
| Ashkenazi Jew | 11 (92) | 12 (100) | 10 (83) | 33 (92) |
| Other | 1 (8) | 0 | 2 (17) | 3 (8) |
| Age, y | ||||
| Mean (SD) | 34.6 (11.1) | 40.4 (15.7) | 36.7 (13.4) | 37.2 (13.4) |
| Range | 18-49 | 19-69 | 17-55 | 17-69 |
| Weight, kg | ||||
| Mean (SD) | 60.80 (9.02) | 70.17 (15.09) | 70.59 (11.51) | 67.19 (12.64) |
| Range | 48.0-82.0 | 48.0-101.0 | 57.0-89.0 | 48.0-101.0 |
| Height, cm | ||||
| Mean (SD) | 163.6 (12.4) | 168.8 (8.7) | 169.8 (10.9) | 167.4 (10.8) |
| Range | 140-189 | 153-184 | 155-189 | 140-189 |
| Severity scoring index | ||||
| Mean (SD) | 9.17 (4.78) | 10.42 (4.01) | 9.00 (5.61) | 9.53 (4.75) |
| Range | 2.0-17.0 | 6.0-17.0 | 3.0-21.0 | 2.0-21.0 |
| Liver organ volume, L | n = 11 | n = 35 | ||
| Mean (SD) | 1.53 (0.32) | 1.81 (0.57) | 1.91 (0.42) | 1.74 (0.47) |
| Range | 1.0-2.1 | 1.0-2.6 | 1.3-2.6 | 1.0-2.6 |
| Spleen organ volume, L* | ||||
| Mean (SD) | 0.63 (0.43) | 0.74 (0.56) | 0.76 (0.37) | 0.71 (0.45) |
| Range | 0.2-1.4 | 0.2-1.8 | 0.3-1.2 | 0.2-1.8 |
For miglustat, n = 8; for imiglucerase, n = 9; for combination, n = 7; and for total, n = 24.