Model input parameters
| Input parameter . | Value . | Probability distribution . | References . |
|---|---|---|---|
| Utilities | |||
| Baseline HA utility, prophylaxis | 0.9378-0.0026 × age | β-PERT (varied by ±0.05 from base case) | 14,15,16 |
| Baseline HA utility, no prophylaxis | 0.6705-0.0019 × age | β-PERT (varied by ±0.05 from base case) | 14,15,16 |
| Disutility of infusion, assuming 12 FVIII infusions per month | 0.005 | β-PERT (0.004, 0.006) | 17 |
| Disutility of CVAD infection | 0.045 | β-PERT (0.036, 0.054) | 18 |
| Utilities by mRS | |||
| mRS 0 | 0.936 | 19 | |
| mRS 1 | 0.817 | ||
| mRS 2 | 0.681 | ||
| mRS 3 | 0.558 | ||
| mRS 4 | 0.265 | ||
| mRS 5 | –0.054 | ||
| Utility for no disability/mild disability after ICH, mRS 0-2 | 0.78 | β-PERT (0.63, 0.94) | Calculation (supplemental, see “Disability-specific utilities”) |
| Utility for disability after ICH, mRS 3-5 | 0.35 | β-PERT (0.28, 0.42) | Calculation (supplemental, see “Disability-specific utilities”) |
| Transition probabilities | |||
| Probability of ICH on emicizumab, yearly, age 0-1 year | 0 | β-PERT (0, 0.00000001) | 6,9 |
| Probability of ICH on standard of care, yearly, age 0-1 year | 0.024 | β-PERT (varied by ±20%) | 6,9 |
| Probability of death after ICH, age 0-1 year | 0.0714 | β-PERT (0.05712, 0.08568) | 7 |
| Probability of disability after ICH, age 0-1 year | 0.38 | β-PERT (0.304, 0.456) | 7 |
| Background mortality for males | Age dependent | US life tables | |
| Disability mortality hazard, relative to background mortality | 151% | Fixed | 10 |
| Distribution of outcomes across the mRS after ICH, 3 months after ICH | |||
| mRS 0 | 0.038 (5/132) | 20 | |
| mRS 1 | 0.22 (29/132) | ||
| mRS 2 | 0.129 (17/132) | ||
| mRS 3 | 0.234 (31/132) | ||
| mRS 4 | 0.318 (42/132) | ||
| mRS 5 | 0.061 (8/132) | ||
| Probability of CVAD infection | 0.0196 | β-PERT (0.0131, 0.0287) | 4 |
| Costs | |||
| Cost of emicizumab prophylaxis, monthly per kg | $610.10 | Fixed | CMS |
| Cost of Advate prophylaxis, monthly per kg | $454.2 | Fixed | CMS |
| Male body weight, kg | Age dependent | 21 (age 0-2 years) 22 (age 2-20 years) 23 (age 20-100 years) | |
| Cost of treating ICH | |||
| Cost of Advate to treat ICH, per kg | $3 179.40 | Fixed | CMS |
| Inpatient costs of ICH | $14 230.77 | γ (225, 63.2) | CMS |
| General population male yearly health care expenditures | Age dependent | γ (varied by ±20% from base case) | MEPS |
| Health care expenditures by mRS, 2010-2014 | |||
| mRS 0 | $10 500 | 24 | |
| mRS 1 | $10 500 | ||
| mRS 2 | $10 500 | ||
| mRS 3 | $11 484 | ||
| mRS 4 | $11 484 | ||
| mRS 5 | $11 484 | ||
| Average baseline health care expenditures for males, 2010-2014 | $6 870 | Fixed | MEPS |
| Post-ICH no-disability/mild-disability baseline cost multiplier | 1.53 | Fixed | Calculation (supplemental, see “cost multipliers”) |
| Post-ICH disability baseline cost multiplier | 1.67 | Fixed | Calculation (supplemental, see “cost multipliers”) |
| Hourly wage for a working adult | $31.48 | Fixed | 25 |
| Cost of death: child, adjusted to 2024 USD | $124 530 | γ (225.1, 553.2) | 26 |
| Cost of death: adult, adjusted to 2024 USD | $64 117 | γ (225.5, 284.4) | 27 |
| Cost of CVAD insertion | $1 144.1 | Fixed | CMS |
| Cost of CVAD removal | $214.46 | Fixed | CMS |
| Input parameter . | Value . | Probability distribution . | References . |
|---|---|---|---|
| Utilities | |||
| Baseline HA utility, prophylaxis | 0.9378-0.0026 × age | β-PERT (varied by ±0.05 from base case) | 14,15,16 |
| Baseline HA utility, no prophylaxis | 0.6705-0.0019 × age | β-PERT (varied by ±0.05 from base case) | 14,15,16 |
| Disutility of infusion, assuming 12 FVIII infusions per month | 0.005 | β-PERT (0.004, 0.006) | 17 |
| Disutility of CVAD infection | 0.045 | β-PERT (0.036, 0.054) | 18 |
| Utilities by mRS | |||
| mRS 0 | 0.936 | 19 | |
| mRS 1 | 0.817 | ||
| mRS 2 | 0.681 | ||
| mRS 3 | 0.558 | ||
| mRS 4 | 0.265 | ||
| mRS 5 | –0.054 | ||
| Utility for no disability/mild disability after ICH, mRS 0-2 | 0.78 | β-PERT (0.63, 0.94) | Calculation (supplemental, see “Disability-specific utilities”) |
| Utility for disability after ICH, mRS 3-5 | 0.35 | β-PERT (0.28, 0.42) | Calculation (supplemental, see “Disability-specific utilities”) |
| Transition probabilities | |||
| Probability of ICH on emicizumab, yearly, age 0-1 year | 0 | β-PERT (0, 0.00000001) | 6,9 |
| Probability of ICH on standard of care, yearly, age 0-1 year | 0.024 | β-PERT (varied by ±20%) | 6,9 |
| Probability of death after ICH, age 0-1 year | 0.0714 | β-PERT (0.05712, 0.08568) | 7 |
| Probability of disability after ICH, age 0-1 year | 0.38 | β-PERT (0.304, 0.456) | 7 |
| Background mortality for males | Age dependent | US life tables | |
| Disability mortality hazard, relative to background mortality | 151% | Fixed | 10 |
| Distribution of outcomes across the mRS after ICH, 3 months after ICH | |||
| mRS 0 | 0.038 (5/132) | 20 | |
| mRS 1 | 0.22 (29/132) | ||
| mRS 2 | 0.129 (17/132) | ||
| mRS 3 | 0.234 (31/132) | ||
| mRS 4 | 0.318 (42/132) | ||
| mRS 5 | 0.061 (8/132) | ||
| Probability of CVAD infection | 0.0196 | β-PERT (0.0131, 0.0287) | 4 |
| Costs | |||
| Cost of emicizumab prophylaxis, monthly per kg | $610.10 | Fixed | CMS |
| Cost of Advate prophylaxis, monthly per kg | $454.2 | Fixed | CMS |
| Male body weight, kg | Age dependent | 21 (age 0-2 years) 22 (age 2-20 years) 23 (age 20-100 years) | |
| Cost of treating ICH | |||
| Cost of Advate to treat ICH, per kg | $3 179.40 | Fixed | CMS |
| Inpatient costs of ICH | $14 230.77 | γ (225, 63.2) | CMS |
| General population male yearly health care expenditures | Age dependent | γ (varied by ±20% from base case) | MEPS |
| Health care expenditures by mRS, 2010-2014 | |||
| mRS 0 | $10 500 | 24 | |
| mRS 1 | $10 500 | ||
| mRS 2 | $10 500 | ||
| mRS 3 | $11 484 | ||
| mRS 4 | $11 484 | ||
| mRS 5 | $11 484 | ||
| Average baseline health care expenditures for males, 2010-2014 | $6 870 | Fixed | MEPS |
| Post-ICH no-disability/mild-disability baseline cost multiplier | 1.53 | Fixed | Calculation (supplemental, see “cost multipliers”) |
| Post-ICH disability baseline cost multiplier | 1.67 | Fixed | Calculation (supplemental, see “cost multipliers”) |
| Hourly wage for a working adult | $31.48 | Fixed | 25 |
| Cost of death: child, adjusted to 2024 USD | $124 530 | γ (225.1, 553.2) | 26 |
| Cost of death: adult, adjusted to 2024 USD | $64 117 | γ (225.5, 284.4) | 27 |
| Cost of CVAD insertion | $1 144.1 | Fixed | CMS |
| Cost of CVAD removal | $214.46 | Fixed | CMS |
CDC, Centers for Disease Control and Prevention; CMS, Centers for Medicare and Medicaid Services; MEPS, Medical Expenditure Panel Survey; USD, US dollar.