Table 1.

Cost, clinical variables, probabilities, and utilities (United States health care system model)

Model variableUpfront HSCTDelayed HSCTReference
Base-case scenarioDistribution used in PSAsBase-case scenarioDistribution 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 Beta-PERT (0.8, 1) 6,18,19  
Probability of only HSCT after relapse  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 variableUpfront HSCTDelayed HSCTReference
Base-case scenarioDistribution used in PSAsBase-case scenarioDistribution 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 Beta-PERT (0.8, 1) 6,18,19  
Probability of only HSCT after relapse  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.

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