{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,7,8]],"date-time":"2026-07-08T00:26:10Z","timestamp":1783470370409,"version":"3.55.0"},"reference-count":40,"publisher":"Cambridge University Press (CUP)","issue":"1","license":[{"start":{"date-parts":[[2023,7,21]],"date-time":"2023-07-21T00:00:00Z","timestamp":1689897600000},"content-version":"unspecified","delay-in-days":201,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0"}],"funder":[{"DOI":"10.13039\/100004339","name":"Sanofi","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100004339","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Int J Technol Assess Health Care"],"published-print":{"date-parts":[[2023]]},"abstract":"<jats:title>Abstract<\/jats:title>\n\t  <jats:sec id=\"S0266462323000442_sec1\">\n\t    <jats:title>Objectives<\/jats:title>\n\t    <jats:p>Acquired thrombotic thrombocytopenic purpura (aTTP) is a rare hematological disease whose clinical management includes caplacizumab along with plasma exchange and immunosuppression, according to international guidelines. Caplacizumab has been available in Colombia since 2022. This study seeks to determine the therapeutic classification of caplacizumab according to the methodology of the Instituto de Evaluaci\u00f3n Tecnol\u00f3gica en Salud.<\/jats:p>\n\t  <\/jats:sec>\n\t  <jats:sec id=\"S0266462323000442_sec2\" sec-type=\"methods\">\n\t    <jats:title>Methods<\/jats:title>\n\t    <jats:p>The classification was carried out through a deliberative process following the modified Delphi technique, with a panel of experts, made up of four hemato-oncologists, a pharmaceutical chemist, and a patient. The results of effectiveness and safety obtained through a systematic review, therapeutic thresholds (clinical significance), and degree of acceptability (willingness to use the technology) were used for the classification.<\/jats:p>\n\t  <\/jats:sec>\n\t  <jats:sec id=\"S0266462323000442_sec3\" sec-type=\"results\">\n\t    <jats:title>Results<\/jats:title>\n\t    <jats:p>Fourteen effectiveness and safety outcomes were submitted for the classification process. Caplacizumab showed clinical significance for some effectiveness outcomes, was not considered inferior in terms of safety, and displayed acceptability of use. Through consensus, the panel determined that caplacizumab plus the standard regimen is superior to the standard regimen in terms of treatment response and composite outcome, and no different for the other effectiveness and safety outcomes. Likewise, in overall terms, the panel determined that caplacizumab together with the standard regimen is superior to the standard regimen.<\/jats:p>\n\t  <\/jats:sec>\n\t  <jats:sec id=\"S0266462323000442_sec4\" sec-type=\"conclusions\">\n\t    <jats:title>Conclusion<\/jats:title>\n\t    <jats:p>Treatment with caplacizumab together with the standard regimen was considered superior to the standard regimen for the treatment of patients with aTTP, as it showed clinically significant benefits in critical outcomes for decision making, and a safety profile no different to its comparator.<\/jats:p>\n\t  <\/jats:sec>","DOI":"10.1017\/s0266462323000442","type":"journal-article","created":{"date-parts":[[2023,7,21]],"date-time":"2023-07-21T08:45:11Z","timestamp":1689929111000},"source":"Crossref","is-referenced-by-count":4,"title":["Effectiveness and safety of caplacizumab in acquired thrombotic thrombocytopenic purpura: health technology assessment and classification according to the methodology established in Colombia"],"prefix":"10.1017","volume":"39","author":[{"ORCID":"https:\/\/orcid.org\/0009-0004-1205-0168","authenticated-orcid":false,"given":"Jahir A.","family":"Soto-Mora","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-2518-7513","authenticated-orcid":false,"given":"Lina M.","family":"G\u00f3mez-Espitia","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-8998-6608","authenticated-orcid":false,"given":"Pieralessandro","family":"Lasalvia","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-7330-3358","authenticated-orcid":false,"given":"Camilo A.","family":"Castellanos Moreno","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0009-0001-3637-6582","authenticated-orcid":false,"given":"Carol A.","family":"Casallas Vanegas","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0009-0004-2603-1451","authenticated-orcid":false,"given":"Sergio A.","family":"Londo\u00f1o Guti\u00e9rrez","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"56","published-online":{"date-parts":[[2023,7,21]]},"reference":[{"key":"S0266462323000442_r29","unstructured":"29. Bro\u017cek, J , Guyatt, G , Oxman, A. GRADE handbook; 2013."},{"key":"S0266462323000442_r18","doi-asserted-by":"publisher","DOI":"10.1111\/jth.15010"},{"key":"S0266462323000442_r21","unstructured":"21. European Medicines Agency (EMA). Anexo I ficha t\u00e9cnica o resumen de las caracter\u00edsticas del producto: Caplacizumab. European Medicines Agency (EMA). 2021. Available from: https:\/\/www.ema.europa.eu\/en\/documents\/product-information\/cablivi-epar-product-information_es.pdf."},{"key":"S0266462323000442_r35","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2019-123720"},{"key":"S0266462323000442_r39","doi-asserted-by":"publisher","DOI":"10.1080\/14712598.2019.1650908"},{"key":"S0266462323000442_r14","unstructured":"14. Stanley, M , Killeen, RB , Michalski, JM . Thrombotic thrombocytopenic purpura. StatPearls. 2022. 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Higgins, J , Thomas, J. Cochrane handbook for systematic reviews of interventions [Internet]. 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