{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,28]],"date-time":"2026-09-28T22:46:39Z","timestamp":1790635599499,"version":"4.1.0"},"reference-count":33,"publisher":"American Society of Hematology","issue":"7","content-domain":{"domain":["ashpublications.org"],"crossmark-restriction":true},"short-container-title":[],"published-print":{"date-parts":[[2021,2,18]]},"abstract":"<jats:title>Abstract<\/jats:title>\n               <jats:p>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 \u00b1 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 2 major randomized clinical trials. The addition of caplacizumab to SOC also led to increased bleeding from transient reductions in von Willebrand factor and increased relapse rates. Using data from the 2 clinical trials of 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\u2009482\u2009260, significantly above the accepted 2019 US willingness-to-pay threshold of $195\u2009300. 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 single greatest impact on lowering the ICER. In a probabilistic sensitivity analysis, SOC was favored over caplacizumab in 100% of 10\u2009000 iterations. Our data indicate that the addition of caplacizumab to SOC in treatment of acquired TTP is not cost effective because of 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.<\/jats:p>","DOI":"10.1182\/blood.2020006052","type":"journal-article","created":{"date-parts":[[2020,12,6]],"date-time":"2020-12-06T20:00:51Z","timestamp":1607284851000},"page":"969-976","update-policy":"https:\/\/doi.org\/10.1182\/blood.2019cm0000","source":"Crossref","is-referenced-by-count":77,"title":["Cost effectiveness of caplacizumab in acquired thrombotic thrombocytopenic purpura"],"prefix":"10.1182","volume":"137","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-1624-4427","authenticated-orcid":false,"given":"George","family":"Goshua","sequence":"first","affiliation":[{"name":"Division of Hematology, Yale University School of Medicine, New Haven, CT;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Pranay","family":"Sinha","sequence":"additional","affiliation":[{"name":"Division of Infectious Diseases, Boston Medical Center, Boston, MA;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-7928-3132","authenticated-orcid":false,"given":"Jeanne E.","family":"Hendrickson","sequence":"additional","affiliation":[{"name":"Department of Laboratory Medicine, Yale University School of Medicine, New Haven, CT;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Christopher","family":"Tormey","sequence":"additional","affiliation":[{"name":"Department of Laboratory Medicine, Yale University School of Medicine, New Haven, CT;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Pavan K.","family":"Bendapudi","sequence":"additional","affiliation":[{"name":"Division of Hematology and Blood Transfusion Service, Massachusetts General Hospital, Boston, MA;"},{"name":"Division of Hemostasis and Thrombosis, Beth Israel Deaconess Medical Center, Boston, MA; and"},{"name":"Harvard Medical School, Boston, MA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Alfred Ian","family":"Lee","sequence":"additional","affiliation":[{"name":"Division of Hematology, Yale University School of Medicine, New Haven, CT;"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"234","reference":[{"issue":"4","key":"2022041419424802200_B1","doi-asserted-by":"crossref","first-page":"841","DOI":"10.1111\/trf.15721","article-title":"Deaths and complications associated with the management of acute immune thrombotic thrombocytopenic purpura","volume":"60","author":"Colling","year":"2020","journal-title":"Transfusion"},{"key":"2022041419424802200_B2","doi-asserted-by":"crossref","first-page":"637","DOI":"10.1182\/asheducation-2015.1.637","article-title":"Treatment of thrombotic thrombocytopenic purpura beyond therapeutic plasma exchange","volume":"2015","author":"Coppo","year":"2015","journal-title":"Hematology Am Soc Hematol Educ Program"},{"issue":"3","key":"2022041419424802200_B3","doi-asserted-by":"crossref","first-page":"299","DOI":"10.1016\/j.transci.2010.09.018","article-title":"Efficacy and safety of rituximab in adult patients with idiopathic relapsing or refractory thrombotic thrombocytopenic purpura: results of a Spanish multicenter study","volume":"43","author":"Rubia","year":"2010","journal-title":"Transfus Apheresis Sci"},{"issue":"37-38","key":"2022041419424802200_B4","first-page":"518","article-title":"Rituximab for acute plasma-refractory thrombotic thrombocytopenic purpura. 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