Table 1.

Base-case input parameters and their probability distributions for PSA

Result or transitionInput parameterProbability distributionStudy or data source
Probability of iTTP based on PLASMIC (6-7) 0.81 β-PERT (0.41, 1) Bendapudi et al5  
Probability of iTTP based on PLASMIC (5) 0.045 β-PERT (0.023, 0.068) Bendapudi et al5  
Daily mortality in patients with iTTP with caplacizumab and TPE (converted to daily using a conservative median per-person treatment period of 35 d) 0.000266 β-PERT (0.00013, 0.00040) Peyvandi et al18  
Daily mortality in patients without iTTP patients with TPE 0.00411 β-PERT (0.0021,0.0.0062) Li et al19  
Daily probability of ISTH-defined major bleeding with caplacizumab (converted to daily using a conservative median per-person treatment period of 35 d across available real-world reports) .000592 β-PERT (0.000296, 0.000888) Coppo et al, Dutt et al, and Knobl et al20-22  
Daily probability of ICH with caplacizumab
(converted to daily using a conservative median per-person treatment period of 35 d across available real-world reports) 
.000423 β-PERT (0.000212, 0.000635) Dutt et al, Knobl et al, and Izquierdo et al21-23  
Mortality of (ISTH-defined) non-ICH major bleeding 0.00440 β-PERT (0.0022, 0.0066) Franco et al24  
Mortality of ICH 0.00758 β-PERT (0.0038, 0.011) Franco et al24  
Probability of TRALI 0.0000833 β-PERT (0.000042, 0.00013) Pandey et al25  
Probability of TACO 0.000639 β-PERT (0.00032, 0.00096) Narick et al26  
Daily probability of severe anaphylaxis 0.000000606 β-PERT (0.0000003, 0.0000009) Som et al27  
Daily probability of catheter thrombosis 0.0000106 β-PERT (0.0000053, 0.000016) Som et al27  
Daily probability of central line infection 0.0000218 β-PERT (0.000011, 0.000033) Som et al27  
Daily mortality of TRALI 0.00107 β-PERT (0.00054, 0.0016) Li et al28  
Daily mortality of TACO 0.000410 β-PERT (0.0002, 0.006) Li et al28  
Daily mortality of central line infection 0.0568 β-PERT (0.028, 0.085) Ziegler et al29  
Costs 
Caplacizumab dose (11 mg) 8 112.62 Fixed CMS 202330,31  
TPE, per session (professional and technical cost) 5 573.47 Gamma (36, 154.82) Average of all 3 US reports: White et al, Connell et al, Goshua et al, and Kim et al10,31-33  
ICU bed per day 1 291.3 Gamma (36, 35.87) Goshua et al31  
Rapid testing (HemosIL Acustar ADAMTS13 activity) 546.02 Gamma (36, 15.17) White et al32  
In-house testing 442.51 Gamma (36, 12.29) Kim et al9  
Send-out testing 582.85 Gamma (36, 16.19) Kim et al9  
iTTP hospitalization without complications 6 667.84 Gamma (36, 185.22) MS-DRG 81034  
iTTP hospitalization with a complication (nonmajor) 8 482.30 Gamma (36, 235.62) MS-DRG 80934  
iTTP hospitalization with a major complication (ie, ICH) 15 155.00 Gamma (36, 420.97) MS-DRG 80834  
Result or transitionInput parameterProbability distributionStudy or data source
Probability of iTTP based on PLASMIC (6-7) 0.81 β-PERT (0.41, 1) Bendapudi et al5  
Probability of iTTP based on PLASMIC (5) 0.045 β-PERT (0.023, 0.068) Bendapudi et al5  
Daily mortality in patients with iTTP with caplacizumab and TPE (converted to daily using a conservative median per-person treatment period of 35 d) 0.000266 β-PERT (0.00013, 0.00040) Peyvandi et al18  
Daily mortality in patients without iTTP patients with TPE 0.00411 β-PERT (0.0021,0.0.0062) Li et al19  
Daily probability of ISTH-defined major bleeding with caplacizumab (converted to daily using a conservative median per-person treatment period of 35 d across available real-world reports) .000592 β-PERT (0.000296, 0.000888) Coppo et al, Dutt et al, and Knobl et al20-22  
Daily probability of ICH with caplacizumab
(converted to daily using a conservative median per-person treatment period of 35 d across available real-world reports) 
.000423 β-PERT (0.000212, 0.000635) Dutt et al, Knobl et al, and Izquierdo et al21-23  
Mortality of (ISTH-defined) non-ICH major bleeding 0.00440 β-PERT (0.0022, 0.0066) Franco et al24  
Mortality of ICH 0.00758 β-PERT (0.0038, 0.011) Franco et al24  
Probability of TRALI 0.0000833 β-PERT (0.000042, 0.00013) Pandey et al25  
Probability of TACO 0.000639 β-PERT (0.00032, 0.00096) Narick et al26  
Daily probability of severe anaphylaxis 0.000000606 β-PERT (0.0000003, 0.0000009) Som et al27  
Daily probability of catheter thrombosis 0.0000106 β-PERT (0.0000053, 0.000016) Som et al27  
Daily probability of central line infection 0.0000218 β-PERT (0.000011, 0.000033) Som et al27  
Daily mortality of TRALI 0.00107 β-PERT (0.00054, 0.0016) Li et al28  
Daily mortality of TACO 0.000410 β-PERT (0.0002, 0.006) Li et al28  
Daily mortality of central line infection 0.0568 β-PERT (0.028, 0.085) Ziegler et al29  
Costs 
Caplacizumab dose (11 mg) 8 112.62 Fixed CMS 202330,31  
TPE, per session (professional and technical cost) 5 573.47 Gamma (36, 154.82) Average of all 3 US reports: White et al, Connell et al, Goshua et al, and Kim et al10,31-33  
ICU bed per day 1 291.3 Gamma (36, 35.87) Goshua et al31  
Rapid testing (HemosIL Acustar ADAMTS13 activity) 546.02 Gamma (36, 15.17) White et al32  
In-house testing 442.51 Gamma (36, 12.29) Kim et al9  
Send-out testing 582.85 Gamma (36, 16.19) Kim et al9  
iTTP hospitalization without complications 6 667.84 Gamma (36, 185.22) MS-DRG 81034  
iTTP hospitalization with a complication (nonmajor) 8 482.30 Gamma (36, 235.62) MS-DRG 80934  
iTTP hospitalization with a major complication (ie, ICH) 15 155.00 Gamma (36, 420.97) MS-DRG 80834  

CMS, Centers for Medicare and Medicaid Services; ICH, intracranial hemorrhage; ISTH, International Society on Thrombosis and Haemostasis; TRALI, transfusion-related acute lung injury; TACO, transfusion-associated cirulatory overload.

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