Table 2.

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

Model variableUpfront HSCTDelayed HSCTReference
Base-case scenarioDistribution used in PSAsBase-case scenarioDistribution used in PSAs
Primary treatment      
Cost of HSCT for the first 2 y  £78 800 ($97 700) Gamma (100, 977.30) N/A N/A 17  
Probability of cGVHD after 2 y 0.42 Beta-PERT (0.34, 0.50) N/A N/A 26  
Annual supportive care after 2 years from HSCT without cGVHD £11 800 ($14 700) Gamma (100, 146.64) N/A N/A 16  
Annual supportive care after 2 years from HSCT with cGVHD £24 200 ($30 000) Gamma (100, 299.90) N/A N/A 16  
Cost of CCT  N/A N/A £11 700 ($14 500) Gamma (100, 144.58) 15  
Cost of 1 cycle of HiDAC before HSCT  £2900 ($3600) Gamma (100, 36.14) N/A N/A 15  
Probability of HiDAC before HSCT  0.16 Beta-PERT (0.13, 0.19) N/A N/A 6  
Supportive care during CCT N/A N/A £7000 ($8700) (100, 86.65) 15  
Annual supportive care for CCT after 6 months N/A N/A £3900 ($4800) Gamma (100, 47.98) 15  
Salvage treatments      
Overall probability of HSCT after relapse  0.342 N/A Beta-PERT (0.8, 1) 19  
Probability of only HSCT after relapse  0.0513 Beta-PERT (0.04, 0.06) 0.49
0.0513  
Beta-PERT (0.39, 0.59)
Beta-PERT (0.04, 0.06)  
6,19  
Cost of HIC plus HSCT after relapse  £126 400 ($156 700) NA £126 400 ($156 700) NA 17,39  
Probability of HIC plus HSCT after relapse  0.2907 Beta-PERT (0.23, 0.35) 0.43
0.2907  
Beta-PERT (0.34, 0.51)
Beta-PERT (0.23, 0.35)  
6,19  
Cost of LIC plus HSCT after relapse  £115 500 ($143 200) NA £115 500 ($143 200) NA 17,39  
Probability of LIC plus HSCT after relapse  N/A 0.08
0  
Beta-PERT (0.07, 0.1)
N/A 
6,19  
Cost of HIC only after relapse  £47 500 ($59 000) Gamma (100, 589.58) £47 500 ($59 000) Gamma (100, 589.58) 39  
Probability of HIC only after relapse  0.23 Beta-PERT (0.18, 0.28) 0
0.23  
N/A
Beta-PERT (0.18, 0.28)  
6,19  
Cost of LIC only after relapse  £36 700 ($45 500) Gamma (100, 454.94) £36 700 ($45 500) Gamma (100, 454.94) 39  
Probability of LIC only after relapse  0.3 Beta-PERT (0.24, 0.36) 0
0.3  
N/A
Beta-PERT (0.24, 0.36)  
6,19  
Cost of BSC only after relapse  £4700 ($5800) Gamma (100, 57.92) £4700 ($5800) Gamma (100, 57.92) 39  
Probability of BSC only after relapse  0.128 Beta-PERT (0.03, 0.045) 0
0.128  
N/A
Beta-PERT (0.11, 0.17)  
6,19  
Cost of supportive care      
Cost of terminal care £16 700 ($20 700) Gamma (100, 206.79) £16 700 ($20 700) Gamma (100, 206.79) 15  
GVHD      
Probability of cGVHD after 2 y 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 from HSCT until 1 y 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 for the first 2 y  £78 800 ($97 700) Gamma (100, 977.30) N/A N/A 17  
Probability of cGVHD after 2 y 0.42 Beta-PERT (0.34, 0.50) N/A N/A 26  
Annual supportive care after 2 years from HSCT without cGVHD £11 800 ($14 700) Gamma (100, 146.64) N/A N/A 16  
Annual supportive care after 2 years from HSCT with cGVHD £24 200 ($30 000) Gamma (100, 299.90) N/A N/A 16  
Cost of CCT  N/A N/A £11 700 ($14 500) Gamma (100, 144.58) 15  
Cost of 1 cycle of HiDAC before HSCT  £2900 ($3600) Gamma (100, 36.14) N/A N/A 15  
Probability of HiDAC before HSCT  0.16 Beta-PERT (0.13, 0.19) N/A N/A 6  
Supportive care during CCT N/A N/A £7000 ($8700) (100, 86.65) 15  
Annual supportive care for CCT after 6 months N/A N/A £3900 ($4800) Gamma (100, 47.98) 15  
Salvage treatments      
Overall probability of HSCT after relapse  0.342 N/A Beta-PERT (0.8, 1) 19  
Probability of only HSCT after relapse  0.0513 Beta-PERT (0.04, 0.06) 0.49
0.0513  
Beta-PERT (0.39, 0.59)
Beta-PERT (0.04, 0.06)  
6,19  
Cost of HIC plus HSCT after relapse  £126 400 ($156 700) NA £126 400 ($156 700) NA 17,39  
Probability of HIC plus HSCT after relapse  0.2907 Beta-PERT (0.23, 0.35) 0.43
0.2907  
Beta-PERT (0.34, 0.51)
Beta-PERT (0.23, 0.35)  
6,19  
Cost of LIC plus HSCT after relapse  £115 500 ($143 200) NA £115 500 ($143 200) NA 17,39  
Probability of LIC plus HSCT after relapse  N/A 0.08
0  
Beta-PERT (0.07, 0.1)
N/A 
6,19  
Cost of HIC only after relapse  £47 500 ($59 000) Gamma (100, 589.58) £47 500 ($59 000) Gamma (100, 589.58) 39  
Probability of HIC only after relapse  0.23 Beta-PERT (0.18, 0.28) 0
0.23  
N/A
Beta-PERT (0.18, 0.28)  
6,19  
Cost of LIC only after relapse  £36 700 ($45 500) Gamma (100, 454.94) £36 700 ($45 500) Gamma (100, 454.94) 39  
Probability of LIC only after relapse  0.3 Beta-PERT (0.24, 0.36) 0
0.3  
N/A
Beta-PERT (0.24, 0.36)  
6,19  
Cost of BSC only after relapse  £4700 ($5800) Gamma (100, 57.92) £4700 ($5800) Gamma (100, 57.92) 39  
Probability of BSC only after relapse  0.128 Beta-PERT (0.03, 0.045) 0
0.128  
N/A
Beta-PERT (0.11, 0.17)  
6,19  
Cost of supportive care      
Cost of terminal care £16 700 ($20 700) Gamma (100, 206.79) £16 700 ($20 700) Gamma (100, 206.79) 15  
GVHD      
Probability of cGVHD after 2 y 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 from HSCT until 1 y 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  

HIC, high-intensity chemotherapy (cytarabine in combination with daunorubicin, doxorubicin, etoposide, idarubicin, mitoxantrone, cladribine, or clofarabine); HiDAC, high-dose cytarabine; LIC, low-intensity chemotherapy (monotherapy of azacitidine, cytarabine, decitabine, gemtuzumab, glasdegib, and ivosidenib).

Adjusted for inflation until the end of 2022.

Calculated depending on data derived from the original trial and literature variables.

This value is a secondary value from the literature for probability of patients who did not undergo salvage HSCT as documented in the ETAL-1 trail.

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