Table 1.
Baseline characteristics of patient cohort.
|
Demographic characteristics | ||
|---|---|---|
| Parameter | Before Switch/ERT | After Switch/SRT |
| No. of patients | 38 | 38 |
| Sex, n (%) | ||
| Male | 23 (61) | |
| Female | 15 (39) | |
| Age at time of visit in years, mean (min-max) |
47 (15-80) | 51 (16-84) |
| N = 38 | N = 38 | |
| Weight at time of visit in kilograms, mean (min-max) |
78.7 (48.2-131.8) | 80.0 (49.9-120.0) |
| N = 36 | N = 36 | |
| No. of splenectomized patients, n (%) | 4 (11) | 4 (11) |
| Length of treatment in months, mean (min-max) |
171.5 (24-312) | 36.7 (5-85) |
| N = 38 | N = 38 | |
| Length of treatment in years, mean (min-max) |
14.3 (2-26) | 3.1 (0.4-7) |
| N = 38 | N = 38 | |
| Patient years of treatment | 543 | 116 |
| N = 38 | N = 38 | |
| CYP2D6-metabolizer phenotype, n (%) | ||
| Ultra-rapid | 1 (3) | |
| Extensive | 26 (68) | |
| Intermediate | 9 (23) | |
| Poor | 2 (6) | |
| Genotype, n (%) | ||
| N370S/N370S | 19 (50) | |
| N370S/Other | 17 (45) | |
| Other/Other | 2 (5) | |