Acquired thrombotic thrombocytopenic purpura (TTP) is a life-threatening disease characterized by thrombotic microangiopathy leading to end-organ damage. The standard of care (SOC) treatment is therapeutic plasma exchange (TPE) alongside immunomodulation with steroids, with increasing use of rituximab +/- other immunomodulatory agents. The addition of caplacizumab, a nanobody targeting von Willebrand factor, was shown to accelerate platelet count recovery and reduce TPE treatments and hospital length of stay in TTP patients treated in the TITAN and HERCULES trials. The addition of caplacizumab to SOC also led to increased bleeding due to transient reductions in von Willebrand factor and increased relapse rates. Using data from TITAN and HERCULES on caplacizumab, we performed the first-ever cost effectiveness analysis in TTP. Over a 5-year period, the projected incremental cost effectiveness ratio (ICER) in our Markov model was $1,482,260, significantly above the accepted 2019 US willingness-to-pay of $195,300. One-way sensitivity analyses showed the utility of the well state and the cost of caplacizumab to have the largest effects on ICER, with a reduction in caplacizumab cost demonstrating the greatest impact on lowering the ICER. In a probabilistic sensitivity analysis, SOC was favored over caplacizumab in 100% of 10,000 iterations. Our data indicate that the addition of caplacizumab to SOC in treatment of acquired TTP is not cost effective due to the high cost of the medication and its failure to improve relapse rates. The potential impact of caplacizumab on health system cost using longer-term follow-up data merits further study.
Von Willebrand factor, ADAMTS13, and thrombotic thrombocytopenic purpura
