{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,5]],"date-time":"2026-09-05T14:10:46Z","timestamp":1788617446166,"version":"build-2803163510"},"reference-count":45,"publisher":"Wiley","issue":"16","license":[{"start":{"date-parts":[[2023,7,26]],"date-time":"2023-07-26T00:00:00Z","timestamp":1690329600000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Cancer Medicine"],"published-print":{"date-parts":[[2023,8]]},"abstract":"<jats:title>Abstract<\/jats:title>\n                  <jats:sec>\n                    <jats:title>Purpose<\/jats:title>\n                    <jats:p>Medicines in special situations (MSS) refer to off\u2010label or to unlicensed drugs under investigation (compassionate use). Our objectives were to evaluate characteristics and to estimate overall survival (OS), event\u2010free survival (EFS), and the duration of treatment (DT) of MSS used for cancer treatment at a multicentre comprehensive cancer institution.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods<\/jats:title>\n                    <jats:p>Retrospective cohort study on adult cancer patients for whom an MSS treatment was requested (January 2011\u2013December 2020). A descriptive analysis was performed and median OS and EFS and 95% confidence intervals (CIs) were estimated. Survival curves were stratified by type of tumor, ECOG (Eastern Cooperative Oncology Group) performance status (PS), age, sex, treatment stage and type of drug (mechanism of action and target).<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>Treatment was initiated in 2092 episodes (1930 patients) out of 2377 MSS episodes (2189 patients) requested, 33% for hematological treatment and 87% for advanced stage cancer. Median OS (months) was 21.1 (95% CI 19.4\u201322.7), median EFS was 5.6 (95% CI 5.1\u20136.0) months, and median DT was 4.5 [0.0; 115.3] months.<\/jats:p>\n                    <jats:p>\n                      OS and EFS statistically significantly favored female patients, ECOG PS \u22652 episodes showed worse OS and EFS outcomes (\n                      <jats:italic>p<\/jats:italic>\n                      \u2009&lt;\u20090.0001). Statistically significant differences in survival were found within solid and hematological cancer, disease stage, drug mechanism of action, and type of cancer (\n                      <jats:italic>p<\/jats:italic>\n                      \u2009&lt;\u20090.001) but not for age. Survival outcomes by tumor subtype and drug are presented both globally and separately based on disease stage.\n                    <\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusion<\/jats:title>\n                    <jats:p>MSS uses are practiced across almost all cancer types, mostly for advanced disease. ECOG PS \u22652, along with advanced disease, was related to worse survival. Information about real\u2010world outcomes is valuable and contributes to better decision\u2010making regarding MSS and our experience in this field could be of interest for other colleagues.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1002\/cam4.6360","type":"journal-article","created":{"date-parts":[[2023,7,27]],"date-time":"2023-07-27T02:49:27Z","timestamp":1690426167000},"page":"17112-17125","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":14,"title":["Real\u2010world treatment outcomes of medicines used in special situations (off\u2010label and compassionate use) in oncology and hematology: A retrospective study from a comprehensive cancer institution"],"prefix":"10.1002","volume":"12","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-4450-2227","authenticated-orcid":false,"given":"Sandra","family":"Fontanals","sequence":"first","affiliation":[{"name":"Pharmacy Department, Catalan Institute of Oncology (ICO)\u2010Hospitalet, Institut d\u2019Investigaci\u00f3 Biom\u00e8dica de Bellvitge (IDIBELL), School of Medicine Universitat de Barcelona (UB)  Barcelona Spain"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-1962-971X","authenticated-orcid":false,"given":"Anna","family":"Esteve","sequence":"additional","affiliation":[{"name":"Research Management Unit (Unitat de Gesti\u00f3 de la recerca: UGR), Medical Oncology Department, Catalan Institute of Oncology (ICO), Badalona\u2010Applied Research Group in Oncology (B\u2010ARGO) Germans Trias I Pujol Research Institute (IGTP)  Badalona Spain"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0009-0005-4236-0199","authenticated-orcid":false,"given":"Andrea","family":"Gonz\u00e1lez","sequence":"additional","affiliation":[{"name":"Medical Oncology Department Catalan Institute of Oncology (ICO), Badalona\u2010Applied Research Group in Oncology (B\u2010ARGO) Germans Trias I Pujol Research Institute (IGTP)  Badalona Spain"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-6712-5428","authenticated-orcid":false,"given":"Cristina","family":"Ib\u00e1\u00f1ez","sequence":"additional","affiliation":[{"name":"Pharmacy Department, Catalan Institute of Oncology (ICO)\u2010Hospitalet School of Health Sciences, Blanquerna Ramon Llull University  Barcelona Spain"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Javier","family":"Mart\u00ednez","sequence":"additional","affiliation":[{"name":"Pharmacy Department Catalan Institute of Oncology (ICO)\u2010Hospitalet  Barcelona Spain"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-3785-3563","authenticated-orcid":false,"given":"Ricard","family":"Mes\u00eda","sequence":"additional","affiliation":[{"name":"Medical Oncology Department Catalan Institute of Oncology (ICO), Badalona\u2010Applied Research Group in Oncology (B\u2010ARGO) Germans Trias I Pujol Research Institute (IGTP)  Badalona Spain"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-1857-362X","authenticated-orcid":false,"given":"Ana","family":"Clop\u00e9s","sequence":"additional","affiliation":[{"name":"Pharmacy Department, Catalan Institute of Oncology (ICO)\u2010Hospitalet, Institut d\u2019Investigaci\u00f3 Biom\u00e8dica de Bellvitge (IDIBELL) School of Health Sciences, Blanquerna Ramon Llull University  Barcelona Spain"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2023,7,26]]},"reference":[{"key":"e_1_2_12_2_1","unstructured":"2021 FDA approvals of drugs for cancer treatment\u2014the ASCO post.2021.https:\/\/ascopost.com\/issues\/december\u201025\u20102021\/2021\u2010fda\u2010approvals\u2010of\u2010drugs\u2010for\u2010cancer\u2010treatment\/"},{"key":"e_1_2_12_3_1","doi-asserted-by":"publisher","DOI":"10.1093\/annonc\/mdm517"},{"key":"e_1_2_12_4_1","first-page":"1","article-title":"Study on off\u2010label use of medicinal products in the European Union","author":"Weda M","year":"2017","journal-title":"Eur Union"},{"key":"e_1_2_12_5_1","doi-asserted-by":"publisher","DOI":"10.1200\/JCO.2010.34.1248"},{"issue":"8","key":"e_1_2_12_6_1","first-page":"428","article-title":"Off\u2010label drug information: regulation, distribution, evaluation, and related controversies","volume":"34","author":"Lee VC","year":"2009","journal-title":"P&T"},{"key":"e_1_2_12_7_1","doi-asserted-by":"publisher","DOI":"10.1001\/jamanetworkopen.2021.0380"},{"key":"e_1_2_12_8_1","unstructured":"Bolet\u00edn Oficial del Estado.https:\/\/www.boe.es\/boe\/dias\/2009\/07\/20\/pdfs\/BOE\u2010A\u20102009\u201012002.pdf"},{"key":"e_1_2_12_9_1","unstructured":"CatSalut.Resoluci\u00f3 per la qual es crea el Consell Assessor de Medicaments en Situacions Especials (CAMSE).2010; 1\u20104.https:\/\/catsalut.gencat.cat\/web\/.content\/minisite\/catsalut\/proveidors_professionals\/normatives_instruccions\/2019\/resolucio\u2010camse.pdf"},{"key":"e_1_2_12_10_1","doi-asserted-by":"publisher","DOI":"10.1016\/S1470-2045(08)70280-8"},{"issue":"1","key":"e_1_2_12_11_1","doi-asserted-by":"crossref","first-page":"1","DOI":"10.32471\/exp-oncology.2312-8852.vol-43-no-1.15583","article-title":"Experience and prospects for the use of off\u2010label drugs in oncology","volume":"43","author":"Drogovoz SM","year":"2021","journal-title":"Exp Oncol"},{"key":"e_1_2_12_12_1","doi-asserted-by":"publisher","DOI":"10.1111\/jcpt.12507"},{"issue":"5","key":"e_1_2_12_13_1","first-page":"275","article-title":"Off\u2010label use of oncology drugs: national survey results","volume":"39","author":"Pe\u00f1 EGH","year":"2015","journal-title":"Farm Hosp"},{"key":"e_1_2_12_14_1","doi-asserted-by":"publisher","DOI":"10.5306\/wjco.v7.i2.253"},{"key":"e_1_2_12_15_1","doi-asserted-by":"publisher","DOI":"10.1093\/annonc\/mdz445"},{"key":"e_1_2_12_16_1","doi-asserted-by":"publisher","DOI":"10.1002\/cam4.3349"},{"issue":"3","key":"e_1_2_12_17_1","first-page":"133","article-title":"Medicines in exceptional circumstances for solid tumours: focusing on evidence, effectiveness, and toxicity profiles","volume":"46","author":"Medina AA","year":"2022","journal-title":"Farm Hosp"},{"key":"e_1_2_12_18_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.esmoop.2022.100604"},{"key":"e_1_2_12_19_1","doi-asserted-by":"publisher","DOI":"10.6004\/jnccn.2016.0007"},{"key":"e_1_2_12_20_1","doi-asserted-by":"publisher","DOI":"10.1007\/s00228-014-1746-2"},{"key":"e_1_2_12_21_1","doi-asserted-by":"publisher","DOI":"10.1097\/MD.0000000000003623"},{"key":"e_1_2_12_22_1","first-page":"1","article-title":"Frequency and level of evidence used in recommendations by the National Comprehensive Cancer Network guidelines beyond approvals of the US Food and Drug Administration: retrospective observational study","volume":"360","author":"Wagner J","year":"2018","journal-title":"BMJ"},{"issue":"3","key":"e_1_2_12_23_1","first-page":"188.e1","article-title":"Off\u2010label pediatric medicines in Spain","volume":"94","author":"Pi\u00f1eiro P\u00e9rez R","year":"2021","journal-title":"An Pediatr (Engl Ed)"},{"issue":"4","key":"e_1_2_12_24_1","first-page":"458","article-title":"Assessment of off\u2010label prescribing: profile, evidence and evolution","volume":"41","author":"Blanco\u2010Reina E","year":"2017","journal-title":"Farm Hosp"},{"key":"e_1_2_12_25_1","doi-asserted-by":"publisher","DOI":"10.3389\/fphar.2019.01210"},{"key":"e_1_2_12_26_1","first-page":"178","article-title":"Quality of off\u2010label prescriptions in advanced oncologic diseases","volume":"145","author":"Mart\u00edn Gonz\u00e1lez A","year":"2015","journal-title":"Med Clin"},{"key":"e_1_2_12_27_1","doi-asserted-by":"publisher","DOI":"10.1093\/annonc\/mdv249"},{"key":"e_1_2_12_28_1","doi-asserted-by":"publisher","DOI":"10.1093\/annonc\/mdu353.6"},{"issue":"6","key":"e_1_2_12_29_1","first-page":"272","article-title":"Coste\u2010efectividad de los medicamentos en situaciones especiales en un hospital","volume":"44","author":"Gonz\u00e1lez\u2010Morcillo G","year":"2020","journal-title":"Farm Hosp"},{"key":"e_1_2_12_30_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.esmoop.2023.101212"},{"key":"e_1_2_12_31_1","volume-title":"Guies i protocols","year":"2023"},{"key":"e_1_2_12_32_1","doi-asserted-by":"publisher","DOI":"10.1136\/esmoopen-2019-000615"},{"key":"e_1_2_12_33_1","first-page":"1","volume-title":"The Root Cause of Unavailability and Delay to Innovative Medicines: reducing the Time Before Patients have Access to Innovative Medicines","author":"European Federation of Pharmaceutical Industries and Associations","year":"2020"},{"issue":"3","key":"e_1_2_12_34_1","first-page":"382","article-title":"Off\u2010label antineoplastic drugs. An effectiveness and safety study contribution to scientific literature","volume":"41","author":"Arroyo \u00c1lvarez C","year":"2017","journal-title":"Farm Hosp"},{"issue":"7","key":"e_1_2_12_35_1","first-page":"1","article-title":"Sex and gender influences on cancer immunotherapy response","volume":"8","author":"Irelli A","year":"2020","journal-title":"Biomedicine"},{"key":"e_1_2_12_36_1","doi-asserted-by":"publisher","DOI":"10.1016\/S1470-2045(18)30261-4"},{"key":"e_1_2_12_37_1","doi-asserted-by":"publisher","DOI":"10.1158\/1078-0432.CCR-21-2600"},{"key":"e_1_2_12_38_1","doi-asserted-by":"publisher","DOI":"10.1007\/s00408-020-00407-5"},{"key":"e_1_2_12_39_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ctrv.2022.102428"},{"key":"e_1_2_12_40_1","doi-asserted-by":"publisher","DOI":"10.1111\/bjh.17746"},{"key":"e_1_2_12_41_1","doi-asserted-by":"publisher","DOI":"10.3389\/fonc.2021.739006"},{"key":"e_1_2_12_42_1","doi-asserted-by":"publisher","DOI":"10.3390\/jcm10245845"},{"key":"e_1_2_12_43_1","doi-asserted-by":"publisher","DOI":"10.3390\/cancers12020482"},{"key":"e_1_2_12_44_1","doi-asserted-by":"publisher","DOI":"10.1001\/jamanetworkopen.2023.2002"},{"key":"e_1_2_12_45_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa1412690"},{"key":"e_1_2_12_46_1","doi-asserted-by":"publisher","DOI":"10.1200\/OP.21.00419"}],"container-title":["Cancer Medicine"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1002\/cam4.6360","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2024,3,27]],"date-time":"2024-03-27T05:42:51Z","timestamp":1711518171000},"score":1,"resource":{"primary":{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/10.1002\/cam4.6360"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2023,7,26]]},"references-count":45,"journal-issue":{"issue":"16","published-print":{"date-parts":[[2023,8]]}},"alternative-id":["10.1002\/cam4.6360"],"URL":"https:\/\/doi.org\/10.1002\/cam4.6360","archive":["Portico"],"relation":{},"ISSN":["2045-7634","2045-7634"],"issn-type":[{"value":"2045-7634","type":"print"},{"value":"2045-7634","type":"electronic"}],"subject":[],"published":{"date-parts":[[2023,7,26]]},"assertion":[{"value":"2023-03-13","order":0,"name":"received","label":"Received","group":{"name":"publication_history","label":"Publication History"}},{"value":"2023-07-09","order":1,"name":"accepted","label":"Accepted","group":{"name":"publication_history","label":"Publication History"}},{"value":"2023-07-26","order":2,"name":"published","label":"Published","group":{"name":"publication_history","label":"Publication History"}}]}}