Patient characteristics of 106 adult Gaucher type 1 patients
. | AMC . | HHU . | P . |
|---|---|---|---|
| No. of patients | 49 | 57 | NA |
| Age in 2003, y, median (range) | 47.0 (21-74) | 49.0 (21-82) | NS |
| Men, no. (%) | 27 (55%) | 25 (43%) | NS |
| Splenectomies, no. (%) | 19 (39%) | 19 (33%) | NS |
| SSI, median (range) | 8 (3-18) | 7.0 (2-16) | NS |
| Hb, g/dL, median (range) | 12.0 (8.0-15.8) | 12.6 (7.7-16.5) | .028 |
| Platelet count × 109/L, median (range) | 93 (17-726) | 73 (9-463) | NS |
| Liver volume, mL, median (range) | 3095 (1504-6542) | 1741 (1048-2821) | < .001 |
| Spleen volume, mL, median (range) | 1400 (470-4821) | 1597 (365-4195) | NS |
| BMB score, median (range) | 6.5 (0-8) | 7 (3-8) | NS |
| Chitotriosidase, nmol/mL/h, median (range) | 16 703 (5316-102 324) | 11 869 (592-39 791) | .056 |
| Patients with a serious bone complication in the 10 y prior to start of ERT, no. (%) | 21 (43) | 25 (44) | NS |
| Start dose, U/kg/4 wk, median (range) | 15 (15-50) | 80 (60-120) | < .001 |
| ERT cost per y for a 70-kg (154-lb) patient at start dose, median, US $ (€) | 69.85 (55.165) | 376.44 (294.221) | NA |
. | AMC . | HHU . | P . |
|---|---|---|---|
| No. of patients | 49 | 57 | NA |
| Age in 2003, y, median (range) | 47.0 (21-74) | 49.0 (21-82) | NS |
| Men, no. (%) | 27 (55%) | 25 (43%) | NS |
| Splenectomies, no. (%) | 19 (39%) | 19 (33%) | NS |
| SSI, median (range) | 8 (3-18) | 7.0 (2-16) | NS |
| Hb, g/dL, median (range) | 12.0 (8.0-15.8) | 12.6 (7.7-16.5) | .028 |
| Platelet count × 109/L, median (range) | 93 (17-726) | 73 (9-463) | NS |
| Liver volume, mL, median (range) | 3095 (1504-6542) | 1741 (1048-2821) | < .001 |
| Spleen volume, mL, median (range) | 1400 (470-4821) | 1597 (365-4195) | NS |
| BMB score, median (range) | 6.5 (0-8) | 7 (3-8) | NS |
| Chitotriosidase, nmol/mL/h, median (range) | 16 703 (5316-102 324) | 11 869 (592-39 791) | .056 |
| Patients with a serious bone complication in the 10 y prior to start of ERT, no. (%) | 21 (43) | 25 (44) | NS |
| Start dose, U/kg/4 wk, median (range) | 15 (15-50) | 80 (60-120) | < .001 |
| ERT cost per y for a 70-kg (154-lb) patient at start dose, median, US $ (€) | 69.85 (55.165) | 376.44 (294.221) | NA |
NA indicates not applicable; NS, not significant.