Comparison of Baseline Factors for the 3 Treatment Groups
| Factor | Group I (n = 6) | Group II (n = 17) | Group III (n = 29) | P |
| Age (y) | 30.7 ± 13.8 | 25.7 ± 11.1 | 34.3 ± 17.6 | 0.19 |
| Sex, female (n) | 2 (33.3%) | 13 (76.5%) | 15 (51.7%) | 0.11 |
| Splenectomy (n) | 2 (33.3%) | 2 (11.8%) | 10 (34.5%) | 0.22 |
| Age at diagnosis (y) | 18.8 ± 16.7 | 11.4 ± 8.7 | 26.1 ± 18.3 | 0.01 |
| Hepatomegaly score | 1.5 ± 0.8 | 1.2 ± 0.9 | 1.8 ± 0.8 | 0.06 |
| Splenomegaly score* | 2.0 ± 1.4 | 1.5 ± 0.2 | 2.5 ± 0.8 | 0.01 |
| Bone pain score | 0.7 ± 1.2 | 1.2 ± 1.3 | 1.8 ± 1.1 | 0.06 |
| x-ray score | 1.7 ± 0.5 | 1.8 ± 0.8 | 2.4 ± 0.9 | 0.06 |
| Hemoglobin (g/dL) | 13.6 ± 1.2 | 13.3 ± 1.8 | 12.1 ± 1.7 | 0.03 |
| Platelet count/μL | 140,500 ± 37,308 | 192,705 ± 165,234 | 135,482 ± 95,249 | 0.28 |
| Zimran SSI | 7.2 ± 4.5 | 9.1 ± 6.9 | 11.3 ± 5.8 | 0.23 |
| 99mTc ± sestamibi score | 4.8 ± 2.4 | 4.4 ± 1.5 | 6.1 ± 2.0 | 0.02 |
| Serum chitotriosidase† (nmol/mL/h) | 4,311 ± 4,378 | 15,823 ± 14,120 | 0.05 | |
| Duration of prior ERT (mo) | 47.6 ± 25.3 | |||
| Prior cumulative ERT (U/kg of body weight) | 1,730 ± 1,323 | |||
| Prior ERT dose (U/kg/mo) | 35.2 ± 17.4 |
↵* Only for nonsplenectomized patients (n = 4, 19, and 15 for groups I, II, and III, respectively).
↵† Only measured for groups II and III (n = 27 and 17, respectively).
Qualitative data are expressed as numbers, followed by percentages in parentheses; continuous data are expressed as mean ± SD. Zimran SSI and scores for hepatomegaly, splenomegaly, and bone pain are as defined by Zimran et al. (28); x-ray score is as defined by Hermann et al. (30); and 99mTc-sestamibi score is as defined by Mariani et al. (25).