1132P - More than 50% of patients with metastatic melanoma are not represented in pivotal phase 3 immunotherapy registration trials

Date 09 October 2016
Event ESMO 2016 Congress
Session Poster display
Topics Immunotherapy
Skin Cancers
Presenter Marco Donia
Citation Annals of Oncology (2016) 27 (6): 379-400. 10.1093/annonc/mdw379
Authors M. Donia1, M.L. Kimper-Karl2, L. Bastholt2, I.M. Svane1
  • 1Center For Cancer Immune Therapy And Department Of Oncology, University Hospital Herlev, 2730 - Herlev/DK
  • 2Dept. Of Oncology, Odense University Hospital, DK-5000 - Odense C/DK



Recent randomized trials with strict patient (pt) selection criteria led to the approval of several immune checkpoint inhibitors for unresectable or metastatic melanoma (MM). It is currently unknown how large is the proportion of real life pts with MM not represented in these trials


Data from all MM patients referred in 2014 to assessment for systemic treatment were retrieved from the Danish MM Database. Data were available from two of three reference centers, where all resident pts diagnosed with MM are referred. A total of 194 cases (uveal melanoma was excluded) were retrieved, and 183 pts with sufficient records were included in the analysis. Seven pre-defined enrolment eligibility criteria, all employed in five recent randomized phase 3 immunotherapy trials, were analyzed


At 1st visit, the majority of pts (82%, n = 150) had confirmed cutaneous melanoma, 15% melanoma of unknown primary origin and 3% had mucosal melanoma. 32% of the pts had PS ≥ 2; 22% active/untreated known brain metastases; 22% significant comorbidities; 10% other malignancies in the past 5 years; 6% autoimmune diseases and 19% were on treatment with immunosuppressive drugs. 4 additional pts were not eligible because of the absence of target lesions. In total, 59% of the total population did not fulfil at least one enrolment criteria (non-eligible group). Median survival of the non-eligible group was 5.2 months vs 17.3 months for the eligible (p 


At least half the patients evaluated for systemic treatment of MM are not represented in phase 3 registration immunotherapy trials. These data reveal a huge knowledge gap regarding the usefulness of new immunotherapies in the broader patient population, and urge additional testing of known regimens in selected poor prognosis cohorts

Clinical trial identification

Legal entity responsible for the study

Herlev Hospital


Herlev Hospital


All authors have declared no conflicts of interest.