CLINICAL REPORTSReal-world use, safety, and survival of ipilimumab in metastatic cutaneous melanoma in The NetherlandsJochems, Anouka,b,*; Leeneman, Brendac,*; Franken, Margreet G.d; Schouwenburg, Maartje G.a,b; Aarts, Maureen J.B.f; van Akkooi, Alexander C.J.g; van den Berkmortel, Franchette W.P.J.j; van den Eertwegh, Alfonsus J.M.b,i; Groenewegen, Gerardk; de Groot, Jan Willem B.m; Haanen, John B.A.G.b,h; Hospers, Geke A.P.n; Kapiteijn, Ellena; Koornstra, Rutger H.o; Kruit, Wim H.J.e; Louwman, Marieke W.J.l; Piersma, Djurap; van Rijn, Rozemarijn S.q; ten Tije, Albert J.r; Vreugdenhil, Gerards; Wouters, Michel W.J.M.b,g; Uyl-de Groot, Carin A.c,d; van der Hoeven, Koos J.M.b,oAuthor Information aDepartment of Medical Oncology, Leiden University Medical Center bDutch Institute for Clinical Auditing, Leiden cDepartment of Health Technology Assessment, Erasmus School of Health Policy & Management dInstitute for Medical Technology Assessment, Erasmus University Rotterdam eDepartment for Medical Oncology, Erasmus MC Cancer Institute, Rotterdam fDepartment of Medical Oncology, Maastricht University Medical Center, Maastricht Departments of gSurgical Oncology hMedical Oncology, Netherlands Cancer Institute – Antoni van Leeuwenhoek Hospital iDepartment of Medical Oncology, VU University Medical Center, Amsterdam jDepartment of Internal Medicine, Zuyderland Medical Center Geleen, Heerlen kDepartment of Medical Oncology, University Medical Center Utrecht lNetherlands Comprehensive Cancer Organisation, Utrecht mDepartment of Medical Oncology, Isala, Zwolle nDepartment of Medical Oncology, University Medical Center Groningen, Groningen oDepartment of Medical Oncology, Radboud University Medical Center, Nijmegen pDepartment of Internal Medicine, Medical Spectrum Twente, Enschede qDepartment of Internal Medicine, Medical Center Leeuwarden, Leeuwarden rDepartment of Internal Medicine, Amphia Hospital, Breda sDepartment of Internal Medicine, Maxima Medical Center, Veldhoven, The Netherlands *Anouk Jochems and Brenda Leeneman contributed equally to the writing of this article. Correspondence to Anouk Jochems, MD, MSc, Department of Medical Oncology, Leiden University Medical Center, Albinusdreef 2, PO Box 9600, 2300 RC Leiden, The Netherlands Tel: +31 71 5263486; fax: +31 71 5264036; e-mail: [email protected] Received October 5, 2017 Accepted March 15, 2018 Anti-Cancer Drugs: July 2018 - Volume 29 - Issue 6 - p 572-578 doi: 10.1097/CAD.0000000000000629 Buy SDC Metrics Abstract Phase III trials with ipilimumab showed an improved survival in patients with metastatic melanoma. We evaluated the use and safety of ipilimumab, and the survival of all patients with metastatic cutaneous melanoma (N=807) receiving ipilimumab in real-world clinical practice in The Netherlands using data from the Dutch Melanoma Treatment Registry. Patients who were registered between July 2012 and July 2015 were included and analyzed according to their treatment status: treatment-naive (N=344) versus previously-treated (N=463). Overall, 70% of treatment-naive patients and 62% of previously-treated patients received all four planned doses of ipilimumab. Grade 3 and 4 immune-related adverse events occurred in 29% of treatment-naive patients and 21% of previously-treated patients. No treatment-related deaths occurred. Median time to first event was 5.4 months [95% confidence interval (CI): 4.7–6.5 months] in treatment-naive patients and 4.4 months (95% CI: 4.0–4.7 months) in previously-treated patients. Median overall survival was 14.3 months (95% CI: 11.6–16.7 months) in treatment-naive patients and 8.7 months (95% CI: 7.6–9.6 months) in previously-treated patients. In both patient groups, an elevated lactate dehydrogenase level (hazard ratio: 2.25 and 1.70 in treatment-naive and previously-treated patients, respectively) and American Joint Committee on Cancer M1c-stage disease (hazard ratio: 1.81 and 1.83, respectively) were negatively associated with overall survival. These real-world outcomes of ipilimumab slightly differed from outcomes in phase III trials. Although phase III trials are crucial for establishing efficacy, real-world data are of great added value enhancing the generalizability of outcomes of ipilimumab in clinical practice. Copyright © 2018 Wolters Kluwer Health, Inc. All rights reserved.