Table 3.

Cost-saving strategies

Dose reduction strategyNo. of instances calculatedNo. of saved dosesCost savings, €Cost savings, US$
Real-world data: early successful termination (platelet count guided/ADAMTS13 guided) of caplacizumab treatment 26/60 440 2 490 400 3 388 000 
Real-world data: nondaily treatment regimens* 10/60 386 2 184 760 2 972 200 
Hypothetical model: ADAMTS13 guidance of caplacizumab treatment duration 29/60 419 2 371 540 3 226 300 
Dose reduction strategyNo. of instances calculatedNo. of saved dosesCost savings, €Cost savings, US$
Real-world data: early successful termination (platelet count guided/ADAMTS13 guided) of caplacizumab treatment 26/60 440 2 490 400 3 388 000 
Real-world data: nondaily treatment regimens* 10/60 386 2 184 760 2 972 200 
Hypothetical model: ADAMTS13 guidance of caplacizumab treatment duration 29/60 419 2 371 540 3 226 300 

Real-world data and hypothetical cost-saving analysis investigating different dose reduction strategies in the German cohort in the post-PEX treatment period. Real-world data were based on the dose reduction strategies used by the treating physicians. The hypothetical model investigates the potential savings if this strategy had been applied to all patients in the cohort. Please note that these strategies are off-label and must be based on shared decision-making with the patient. Cost calculations are estimations based on the market price (January 2020) of €5660 per dose and US$7700 per dose. Dose reduction strategies overlap in parts and cannot be considered complementary.

*

Nondaily regimens ranged from alternate-day to once a week and may not be suitable for every individual or situation. VWF measurements may help identify suitable patients.

The hypothetical model assumes that no exacerbation/relapse occur after ADAMTS13-guided termination of caplacizumab treatment. Although this might be inferred from the cohort presented herein (no exacerbation/relapse in 14 of 14 attempts to stop caplacizumab treatment when ADAMTS13 activity has risen >10%), no prospective randomized data to support this idea are available.

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