Table 3 Mean yearly volume and costs of enzyme replacement therapy by disease state
From: Cost-effectiveness of enzyme replacement therapy for type 1 Gaucher disease
Disease state | N* | Number of vials | 95% LCL | 95% UCL | Costs | 95% UCL | 95% UCL |
|---|---|---|---|---|---|---|---|
Signs/symptoms | 28 | 76 | 62 | 91 | 151,147 | 123,501 | 180,088 |
Recovery | 19 | 63 | 49 | 75 | 124,183 | 97,529 | 149,041 |
Splenectomy | 6 | 78 | 50 | 111 | 155,082 | 98,443 | 220,004 |
Bone complication | 9 | 102 | 75 | 132 | 202,348 | 149,319 | 261,573 |
Multiple complications | 18 | 86 | 70 | 102 | 170,317 | 138,201 | 202,653 |
Malignancy | 5 | 130 | 68 | 209 | 257,469 | 135,582 | 414,084 |