{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,2,14]],"date-time":"2026-02-14T15:17:58Z","timestamp":1771082278805,"version":"3.50.1"},"reference-count":23,"publisher":"Wiley","issue":"3","license":[{"start":{"date-parts":[[2020,11,9]],"date-time":"2020-11-09T00:00:00Z","timestamp":1604880000000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Pharmacoepidemiology and Drug"],"published-print":{"date-parts":[[2021,3]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Objectives<\/jats:title><jats:p>To evaluate the effectiveness and safety of pembrolizumab use in advanced melanoma in a real\u2010life context; and to explore the existence of an efficacy\u2010effectiveness gap, comparing registry data with the reference clinical trial.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>This study followed the guidelines for good pharmacoepidemology practice. An ambispective cohort was constituted, initiating the observation upon drug approval (17\/07\/2015) and following exposed patients until death or cut\u2010off date (15\/11\/2019). The primary outcome was overall survival (OS); secondary outcomes comprised progression\u2010free survival (PFS), overall response rate (ORR) and the occurrence of adverse events (AE). For all survival analyses, the Kaplan\u2010Meier estimator was used, considering a 95% confidence interval (CI), aside with one\u2010year survival rates.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>A total of 125 patients constituted the cohort, originating from 16 hospitals in Portugal. Median OS was estimated to be 16.9 months (CI<jats:sub>95%<\/jats:sub> 11.3\u201025.5) and the probability of survival after 1\u2009year was 57.5% (CI<jats:sub>95%<\/jats:sub> 48.4%\u201065.6%). Median PFS was estimated to be 4.8 months (CI<jats:sub>95%<\/jats:sub> 3.9\u20106.7) and the probability of remaining progression\u2010free after 1\u2009year was 32.8% (CI<jats:sub>95%<\/jats:sub> 24.8\u201041.1). ORR was 30.4% (CI<jats:sub>95%<\/jats:sub> 22.5%\u201039.3%). AEs were experienced by 82% of patients, and 27% experienced AE\u2265 grade 3.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusions<\/jats:title><jats:p>Our data suggest lower effectiveness in a real\u2010life context than the efficacy reported in the clinical trial. Safety data seems, however, quite comparable to KEYNOTE\u2010006.<\/jats:p><\/jats:sec>","DOI":"10.1002\/pds.5163","type":"journal-article","created":{"date-parts":[[2020,10,26]],"date-time":"2020-10-26T14:04:16Z","timestamp":1603721056000},"page":"342-349","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":4,"title":["Monitoring real\u2010life utilization of pembrolizumab in advanced melanoma using the Portuguese National Cancer Registry"],"prefix":"10.1002","volume":"30","author":[{"ORCID":"https:\/\/orcid.org\/0000-0001-8496-9211","authenticated-orcid":false,"given":"F\u00e1bio C.","family":"Borges","sequence":"first","affiliation":[{"name":"Registo Oncol\u00f3gico Nacional (RON), Instituto Portugu\u00eas de Oncologia de Lisboa Francisco Gentil  Lisboa Portugal"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Catarina","family":"Ramos","sequence":"additional","affiliation":[{"name":"Registo Oncol\u00f3gico Nacional (RON), Instituto Portugu\u00eas de Oncologia de Lisboa Francisco Gentil  Lisboa Portugal"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Adriana","family":"Ramos","sequence":"additional","affiliation":[{"name":"Registo Oncol\u00f3gico Nacional (RON), Instituto Portugu\u00eas de Oncologia de Lisboa Francisco Gentil  Lisboa Portugal"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Gon\u00e7alo P.","family":"Mendes","sequence":"additional","affiliation":[{"name":"Registo Oncol\u00f3gico Nacional (RON), Instituto Portugu\u00eas de Oncologia de Lisboa Francisco Gentil  Lisboa Portugal"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Rodrigo","family":"Murteira","sequence":"additional","affiliation":[{"name":"Registo Oncol\u00f3gico Nacional (RON), Instituto Portugu\u00eas de Oncologia de Lisboa Francisco Gentil  Lisboa Portugal"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Patr\u00edcia","family":"Soares","sequence":"additional","affiliation":[{"name":"Registo Oncol\u00f3gico Nacional (RON), Instituto Portugu\u00eas de Oncologia de Lisboa Francisco Gentil  Lisboa Portugal"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Claudia","family":"Furtado","sequence":"additional","affiliation":[{"name":"Health Technology Assessment, Pricing and Reimbursement Division (DATS), INFARMED, Autoridade Nacional do Medicamento e Produtos de Sa\u00fade, I.P.  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