Cost, clinical variables, probabilities, and utilities (United States health care system model)
| Model variable . | Upfront HSCT . | Delayed HSCT . | Reference . | ||
|---|---|---|---|---|---|
| Base-case scenario . | Distribution used in PSAs . | Base-case scenario . | Distribution used in PSAs . | ||
| Primary treatment | |||||
| Cost of HSCT∗ | $1 012 100 | Gamma (400, 2739.36) | N/A | N/A | |
| Cost of CCT∗ | N/A | N/A | $82 300 | Gamma (400, 205.72) | 13 |
| Cost of 1 cycle of HiDAC before HSCT∗ | $27 300 | Gamma (400, 67.16) | N/A | N/A | 36 |
| Probability of HiDAC before HSCT∗ | 0.16 | Beta-PERT (0.13, 0.19) | N/A | N/A | 6 |
| Salvage treatment | |||||
| Overall probability of HSCT after relapse† | 0.139 | N/A | 1 | Beta-PERT (0.8, 1) | 6,18,19 |
| Probability of only HSCT after relapse† | 0 | Beta-PERT (0.12, 0.18) | 0.49 0.0513‡ | Beta-PERT (0.39, 0.59) Beta-PERT (0.04, 0.06)‡ | 6,18 |
| Cost of HIC plus HSCT after relapse∗ | $1 191 900 | NA | $1 191 900 | NA | 14,37 |
| Probability of HIC plus HSCT after relapse† | 0.532 | Beta-PERT (0.425, 0.638) | 0.43 0.532‡ | Beta-PERT (0.34, 0.51) Beta-PERT (0.425, 0.638)‡ | 6,19 |
| Cost of LIC plus HSCT after relapse∗ | $1 075 400 | NA | $1 075 400 | NA | 14,37 |
| Probability of LIC plus HSCT after relapse† | 0.468 | Beta-PERT (0.374,0.562) | 0.08 0.468‡ | Beta-PERT (0.07, 0.1) Beta-PERT (0.374, 0.562) | 6,19 |
| Cost of HIC only after relapse∗ | $179 800 | Gamma (400, 449.51) | $179 800 | Gamma (400, 449.51) | 37 |
| Probability of HIC only after relapse† | 0.179 | Beta-PERT (0.143, 0.215) | 0 0.179‡ | N/A Beta-PERT (0.143, 0.215)‡ | 6,18,19,37 |
| Cost of LIC only after relapse∗ | $63 300 | Gamma (400, 158.16) | $63 300 | Gamma (400, 158.16) | 37 |
| Probability of LIC only after relapse† | 0.212 | Beta-PERT (0.170, 0.255) | 0 0.212‡ | N/A Beta-PERT (0.170, 0.255)‡ | 6,18,19,37 |
| Cost of AZA + VEN only after relapse∗ | $81 100 | Gamma (400, 202.73) | $81 100 | Gamma (400, 202.73) | 37 |
| Probability of AZA + VEN only after relapse† | 0.096 | Beta-PERT (0.077, 0.115) | 0 0.096‡ | N/A Beta-PERT (0.077, 0.115)‡ | 6,18,19,37 |
| Cost of FLT3i only after relapse∗ | $45 200 | Gamma (400, 112.95) | $45 200 | Gamma (400, 112.95) | 37 |
| Probability of FLT3i only after relapse† | 0.24 | Beta-PERT (0.179, 0.268) | 0 0.24‡ | N/A Beta-PERT (0.179, 0.268)† | 6 |
| Cost of BSC only after relapse∗ | $74 300 | Gamma (400, 185.76) | $74 300 | Gamma (400, 185.76) | 38 |
| Probability of BSC only after relapse† | 0.134 | Beta-PERT (0.120, 0.180) | 0 0.134‡ | N/A Beta-PERT (0.120, 0.180)† | 6,18,19,37 |
| Cost of supportive care | |||||
| Cost of terminal care∗ | $178 400 | Gamma (400, 446.04) | $178 400 | Gamma (400, 446.04) | 14 |
| GVHD | |||||
| Probability of cGVHD after 2 years | 0.42 | Beta-PERT (0.34, 0.50) | N/A | N/A | 26 |
| Survival parameter | |||||
| HR OS | 1.74 (1.39-2.09) | N/A | Ref | N/A | 6 |
| HR DFS | 0.45 (0.36-0.54) | N/A | Ref | N/A | 6 |
| Utilities | |||||
| During CCT | N/A | N/A | 0.58 | Beta-PERT (0.464, 0.696) | 27 |
| After CCT | N/A | N/A | 0.85 | Beta-PERT (0.688, 1) | 27 |
| During HSCT | 0.613 | Beta-PERT (0.49, 0.736) | 0.597 | Beta-PERT (0.478, 0.716) | 26 |
| After 6 months form HSCT until 1 year | 0.81 | Beta-PERT (0.648, 0.972) | 0.81 | Beta-PERT (0.648, 0.972) | 26 |
| Relapse | 0.53 | Beta-PERT (0.424, 0.636) | 0.53 | Beta-PERT (0.424, 0.636) | 26 |
| Model variable . | Upfront HSCT . | Delayed HSCT . | Reference . | ||
|---|---|---|---|---|---|
| Base-case scenario . | Distribution used in PSAs . | Base-case scenario . | Distribution used in PSAs . | ||
| Primary treatment | |||||
| Cost of HSCT∗ | $1 012 100 | Gamma (400, 2739.36) | N/A | N/A | |
| Cost of CCT∗ | N/A | N/A | $82 300 | Gamma (400, 205.72) | 13 |
| Cost of 1 cycle of HiDAC before HSCT∗ | $27 300 | Gamma (400, 67.16) | N/A | N/A | 36 |
| Probability of HiDAC before HSCT∗ | 0.16 | Beta-PERT (0.13, 0.19) | N/A | N/A | 6 |
| Salvage treatment | |||||
| Overall probability of HSCT after relapse† | 0.139 | N/A | 1 | Beta-PERT (0.8, 1) | 6,18,19 |
| Probability of only HSCT after relapse† | 0 | Beta-PERT (0.12, 0.18) | 0.49 0.0513‡ | Beta-PERT (0.39, 0.59) Beta-PERT (0.04, 0.06)‡ | 6,18 |
| Cost of HIC plus HSCT after relapse∗ | $1 191 900 | NA | $1 191 900 | NA | 14,37 |
| Probability of HIC plus HSCT after relapse† | 0.532 | Beta-PERT (0.425, 0.638) | 0.43 0.532‡ | Beta-PERT (0.34, 0.51) Beta-PERT (0.425, 0.638)‡ | 6,19 |
| Cost of LIC plus HSCT after relapse∗ | $1 075 400 | NA | $1 075 400 | NA | 14,37 |
| Probability of LIC plus HSCT after relapse† | 0.468 | Beta-PERT (0.374,0.562) | 0.08 0.468‡ | Beta-PERT (0.07, 0.1) Beta-PERT (0.374, 0.562) | 6,19 |
| Cost of HIC only after relapse∗ | $179 800 | Gamma (400, 449.51) | $179 800 | Gamma (400, 449.51) | 37 |
| Probability of HIC only after relapse† | 0.179 | Beta-PERT (0.143, 0.215) | 0 0.179‡ | N/A Beta-PERT (0.143, 0.215)‡ | 6,18,19,37 |
| Cost of LIC only after relapse∗ | $63 300 | Gamma (400, 158.16) | $63 300 | Gamma (400, 158.16) | 37 |
| Probability of LIC only after relapse† | 0.212 | Beta-PERT (0.170, 0.255) | 0 0.212‡ | N/A Beta-PERT (0.170, 0.255)‡ | 6,18,19,37 |
| Cost of AZA + VEN only after relapse∗ | $81 100 | Gamma (400, 202.73) | $81 100 | Gamma (400, 202.73) | 37 |
| Probability of AZA + VEN only after relapse† | 0.096 | Beta-PERT (0.077, 0.115) | 0 0.096‡ | N/A Beta-PERT (0.077, 0.115)‡ | 6,18,19,37 |
| Cost of FLT3i only after relapse∗ | $45 200 | Gamma (400, 112.95) | $45 200 | Gamma (400, 112.95) | 37 |
| Probability of FLT3i only after relapse† | 0.24 | Beta-PERT (0.179, 0.268) | 0 0.24‡ | N/A Beta-PERT (0.179, 0.268)† | 6 |
| Cost of BSC only after relapse∗ | $74 300 | Gamma (400, 185.76) | $74 300 | Gamma (400, 185.76) | 38 |
| Probability of BSC only after relapse† | 0.134 | Beta-PERT (0.120, 0.180) | 0 0.134‡ | N/A Beta-PERT (0.120, 0.180)† | 6,18,19,37 |
| Cost of supportive care | |||||
| Cost of terminal care∗ | $178 400 | Gamma (400, 446.04) | $178 400 | Gamma (400, 446.04) | 14 |
| GVHD | |||||
| Probability of cGVHD after 2 years | 0.42 | Beta-PERT (0.34, 0.50) | N/A | N/A | 26 |
| Survival parameter | |||||
| HR OS | 1.74 (1.39-2.09) | N/A | Ref | N/A | 6 |
| HR DFS | 0.45 (0.36-0.54) | N/A | Ref | N/A | 6 |
| Utilities | |||||
| During CCT | N/A | N/A | 0.58 | Beta-PERT (0.464, 0.696) | 27 |
| After CCT | N/A | N/A | 0.85 | Beta-PERT (0.688, 1) | 27 |
| During HSCT | 0.613 | Beta-PERT (0.49, 0.736) | 0.597 | Beta-PERT (0.478, 0.716) | 26 |
| After 6 months form HSCT until 1 year | 0.81 | Beta-PERT (0.648, 0.972) | 0.81 | Beta-PERT (0.648, 0.972) | 26 |
| Relapse | 0.53 | Beta-PERT (0.424, 0.636) | 0.53 | Beta-PERT (0.424, 0.636) | 26 |
AZA + VEN, azacytidine + venetoclax; BSC, best supportive care; cGVHD, chronic graft-versus-host disease; FLT3i, FLT3 inhibitor; HIC, high-intensity chemotherapy (cytarabine in combination with daunorubicin, doxorubicin, etoposide, idarubicin, mitoxantrone, cladribine, or clofarabine); HiDAC, high-dose cytarabine; HR, hazard ratio; LIC, low-intensity chemotherapy (monotherapy of azacitidine, cytarabine, decitabine, gemtuzumab, glasdegib, and ivosidenib); N/A, not applicable.
Adjusted for inflation until the end of 2022.
Calculated depending on data derived from the original trial and literature variables.
This value was used only in sensitivity analysis, and is a secondary value from the literature for probability of certain salvage therapy regimens for patients who did not undergo salvage.