{Reference Type}: Journal Article
{Title}: Real-world effectiveness of pembrolizumab as first-line therapy for cisplatin-ineligible patients with advanced urothelial carcinoma: the ARON-2 study.
{Author}: Santoni M;Myint ZW;Büttner T;Takeshita H;Okada Y;Lam ET;Gilbert D;Küronya Z;Tural D;Pichler R;Grande E;Crabb SJ;Kemp R;Massari F;Scagliarini S;Iacovelli R;Vau N;Basso U;Maruzzo M;Molina-Cerrillo J;Galli L;Bamias A;De Giorgi U;Zucali PA;Rizzo M;Seront E;Popovic L;Caffo O;Buti S;Kanesvaran R;Kopecky J;Kucharz J;Zeppellini A;Fiala O;Landmesser J;Ansari J;Giannatempo P;Rizzo A;Zabalza IO;Monteiro FSM;Battelli N;Calabrò F;Porta C;
{Journal}: Cancer Immunol Immunother
{Volume}: 72
{Issue}: 9
{Year}: 2023 Sep 29
{Factor}: 6.63
{DOI}: 10.1007/s00262-023-03469-5
{Abstract}: <B>BACKGROUND: </B>The advent of immune-checkpoint inhibitors has challenged previous treatment paradigms for advanced urothelial carcinoma (UC) in the post-platinum setting as well as in the first-line setting for cisplatin-ineligible patients. In this study, we investigated the effectiveness of pembrolizumab as first-line treatment for cisplatin-ineligible UC.<BR><B>METHODS: </B>Data from patients aged ≥ 18 years with cisplatin-ineligible UC and receiving first-line pembrolizumab from January 1st 2017 to September 1st 2022 were collected. Cisplatin ineligibility was defined according to the Galsky criteria. Thirty-three Institutions from 18 countries were involved in the ARON-2 study.<BR><B>RESULTS: </B>Our analysis included 162 patients. The median follow-up time was 18.9 months (95%CI 15.3-76.9). In the overall study population, the median OS was 15.8 months (95%CI 11.3-32.4). The median OS was significantly longer in males versus females while no statistically significant differences were observed between patients aged < 65y versus ≥ 65y and between smokers and non-smokers. According to Recist 1.1 criteria, 26 patients (16%) experienced CR, 32 (20%) PR, 39 (24%) SD and 55 (34%) PD.<BR><B>CONCLUSIONS: </B>Our data confirm the role of pembrolizumab as first-line therapy for cisplatin-unfit patients. Further studies investigating the biological and immunological characteristics of UC patients are warranted in order to optimize the outcome of patients receiving immunotherapy in this setting.