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is a matter of concern. There are new drugs for several diseases, some of them very effective, but out of reach for most patients. One example is the treatment of thrombotic thrombocytopenic purpura (TTP). Plasma exchange is routinely employed, this procedure is effective but expensive and the addition of rituximab, which may also increase responses, also increases cost. Recently, caplacizumab has been proposed as a new therapeutic option and even hailed as a new standard by some. This drug is an antibody fragment that reduces von Willebrand factor-mediated platelet adhesion and platelet consumption and blocks the formation of blood clots. However, it is interesting that a recent study by Goshua, et al. published in Blood reported a Markov model analysis with an incremental cost effectiveness ratio (ICER) of $1,482,260 USD for adding caplacizumab to standard TTP management. They concluded that is not cost effective. Nevertheless, it is interesting to note that the antibody has been conditionally recommended by the International Society of Thrombosis and Hemostasis (TTP guidelines). The average cost of therapy is around USD $270,000. There are many new drugs and antibodies in hematology, but are we ready, in the real world, to afford these new therapeutic options?
Keep safe,
David Gómez-Almaguer MD
ISH Chair of Council
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