Model parameters
| Parameter . | . | Estimate . | . |
|---|---|---|---|
| Baseline ABR | |||
| Baseline annual number of bleeds | |||
| Low9 | 11.0 | ||
| Medium (base case)8 | 26.5 | ||
| High7 | 39.6 | ||
| LTP treatment-specific inputs | Estimate | ||
| LTP dosing, VWF IU/kg | |||
| Dose strength, VWF IU/kg | 32.5 | ||
| Dose frequency, doses per week | 2 | ||
| Derived relative risk of bleed for LTP8 | 0.04 | ||
| Bleed-related inputs | Minor bleed | Major bleed | |
| Dosing for bleeds, VWF IU/kg | |||
| Dose strength, VWF IU/kg | 30.0 | 45.0 | |
| Daily doses | 2 | 2 | |
| Duration of treatment, d | 1 | 4 | |
| Probability of medical resource use | |||
| Outpatient visit | 25.0% | 50.0% | |
| Inpatient stay for hospitalization | 0.0% | 50.0% | |
| Percentage of bleeds that are joint bleeds17 | 24.0% | 24.0% | |
| Severity of bleeds while receiving ODT8 | 61.84% | 38.16% | |
| Severity of bleeds while receiving prophylaxis treatment8 | 70.00% | 30.00% | |
| Cost inputs | Unit cost | ||
| Voncento list price per IU of VWF18 | ₤0.32 | ||
| Other costs19 | |||
| Outpatient visit | £166.51 | ||
| Inpatient stay for hospitalization | £2992.13 | ||
| Joint surgery costs | £1383.50 | ||
| Weight inputs | Males | Females | |
| Average patient weight, kg20 | |||
| 11-12 y | 46.90 | 47.06 | |
| 13-15 y | 60.99 | 58.29 | |
| 16-24 y | 77.54 | 65.80 | |
| 25-34 y | 85.65 | 72.56 | |
| 35-44 y | 86.82 | 74.47 | |
| 45-54 y | 88.84 | 75.69 | |
| 55-64 y | 87.98 | 73.86 | |
| 65-74 y | 87.45 | 71.73 | |
| 75+ y | 79.98 | 67.34 | |
| Utility inputs | PS 0 | PS 1-27 | Surgery |
| Health state utility weights21-24 | |||
| Age 0-30 y | 0.94 | 0.82 | 0.72 |
| Age 31-40 y | 0.84 | 0.74 | 0.65 |
| Age 41-50 y | 0.86 | 0.69 | 0.61 |
| Age 51-60 y | 0.83 | 0.63 | 0.56 |
| Age 61-100 y | 0.73 | 0.54 | 0.48 |
| Parameter . | . | Estimate . | . |
|---|---|---|---|
| Baseline ABR | |||
| Baseline annual number of bleeds | |||
| Low9 | 11.0 | ||
| Medium (base case)8 | 26.5 | ||
| High7 | 39.6 | ||
| LTP treatment-specific inputs | Estimate | ||
| LTP dosing, VWF IU/kg | |||
| Dose strength, VWF IU/kg | 32.5 | ||
| Dose frequency, doses per week | 2 | ||
| Derived relative risk of bleed for LTP8 | 0.04 | ||
| Bleed-related inputs | Minor bleed | Major bleed | |
| Dosing for bleeds, VWF IU/kg | |||
| Dose strength, VWF IU/kg | 30.0 | 45.0 | |
| Daily doses | 2 | 2 | |
| Duration of treatment, d | 1 | 4 | |
| Probability of medical resource use | |||
| Outpatient visit | 25.0% | 50.0% | |
| Inpatient stay for hospitalization | 0.0% | 50.0% | |
| Percentage of bleeds that are joint bleeds17 | 24.0% | 24.0% | |
| Severity of bleeds while receiving ODT8 | 61.84% | 38.16% | |
| Severity of bleeds while receiving prophylaxis treatment8 | 70.00% | 30.00% | |
| Cost inputs | Unit cost | ||
| Voncento list price per IU of VWF18 | ₤0.32 | ||
| Other costs19 | |||
| Outpatient visit | £166.51 | ||
| Inpatient stay for hospitalization | £2992.13 | ||
| Joint surgery costs | £1383.50 | ||
| Weight inputs | Males | Females | |
| Average patient weight, kg20 | |||
| 11-12 y | 46.90 | 47.06 | |
| 13-15 y | 60.99 | 58.29 | |
| 16-24 y | 77.54 | 65.80 | |
| 25-34 y | 85.65 | 72.56 | |
| 35-44 y | 86.82 | 74.47 | |
| 45-54 y | 88.84 | 75.69 | |
| 55-64 y | 87.98 | 73.86 | |
| 65-74 y | 87.45 | 71.73 | |
| 75+ y | 79.98 | 67.34 | |
| Utility inputs | PS 0 | PS 1-27 | Surgery |
| Health state utility weights21-24 | |||
| Age 0-30 y | 0.94 | 0.82 | 0.72 |
| Age 31-40 y | 0.84 | 0.74 | 0.65 |
| Age 41-50 y | 0.86 | 0.69 | 0.61 |
| Age 51-60 y | 0.83 | 0.63 | 0.56 |
| Age 61-100 y | 0.73 | 0.54 | 0.48 |
IU, international unit; PS, Pettersson score.