Patients enrolled | N = 443 |
---|---|
Gender, n (%) | n = 443 |
Male | 201 (45.4) |
Female | 242 (54.6) |
Genotype b , n (%) | n = 224 |
N370S/Rare allelec | 69 (31) |
N370S/L444P | 56 (25) |
N370S/?d | 30 (13) |
L444P/?d | 14 (6) |
Rare allelec/rare allelec | 13 (6) |
N370S/IVS2 + 1 | 8 (4) |
N370S/N370S | 7 (3) |
L444P/rare allelec | 7 (3) |
N370S/84GG | 6 (3) |
L444P/L444P | 4 (2) |
Rare allelec/?d | 3 (1) |
N370S/D409H | 2 (1) |
D409H/rare allelec | 2 (1) |
? d/?d | 2 (1) |
IVS2 + 1/rare allelec | 1 (<1) |
Age at diagnosis, n (%) | n = 443 |
0a to <6 years | 190 (43.8) |
6 to <12 years | 173 (40.1) |
12 to <18 years | 70 (16.1) |
Treatment dose, mean ± SD, U/kg/2wks | |
Baseline | 43.4 ± 16.61 |
After 8 years of imiglucerase | 36.8 ± 12.37 |
Age at first infusion, n (%) | n = 443 |
0 to <6 years | 122 (27.5) |
6 to <12 years | 180 (40.6) |
12 to <18 years | 141 (31.8) |
Splenectomized patients , n (%) | 33 (7.4%) |
Distribution by country, n (%) | n = 443 |
Brazil | 272 (61.4) |
Argentina | 78 (17.6) |
Colombia | 34 (7.7) |
Venezuela | 32 (7.2) |
Chile | 9 (2.0) |
Mexico | 7 (1.6) |
Otherse | 11 (2.5) |
Ethnicity, n (%) | n = 443 |
Black/Caribbean | 130 (29.3) |
Caucasian, non-Jewish | 121 (27.3) |
Hispanic | 116 (26.2) |
Multi-ethnic, non-Jewish or Arab | 15 (3.4) |
American Indian | 4 (0.9) |
Jewish, Ashkenazi | 4 (0.9) |
Other | 0 (0.0) |
Unknown | 53 (12.0) |