Citations 2
Outcomes of Cisplatin, Gemcitabine, and Durvalumab According to TOPAZ ‐1 Study Eligibility: A Large Global Analysis in a Real‐World Setting
Prinzi, Federica Lo , Rimini, Margherita , Ikeda, Masafumi, Antonuzzo, Lorenzo, Kim, Jin Won, Pressiani, Tiziana, Peeters, Frederik , Pinato, David J., Pircher, Chiara, Tamberi, Stefano, Couto, Nuno, Chon, Hong Jae, Martirena, A. , Lamarca, Angela, Perkhofer, Lukas , Kim, Il Hwan, Pinto, C. , Kang, Beodeul, Pastorino, Alessandro , Changhoon, Yoo, Rodriquenz, Maria Grazia , Scartozzi, Mario, Zumstein, Lucrezia , Ghidini, Michele, Aprile, Giuseppe , Spinelli, Gian Paolo , Yung‐Yeh, Su, Warmington, Emily , Lancianese, Alessia , Satake, Tomoyuki, Bekaii‐Saab, Tanios, Lavacchi, Daniele, Passeri, Laura , Kang, Minsu, Prete, Alessandra Anna, Bozzarelli, Silvia, Persano, Mara , Masi, Gianluca, Balsano, Rita , Lonardi, Sara, Salani, Francesca, Camera, Silvia, Vogel, Arndt, Rimassa, Lorenza, Rimassa, Lorenza , Vivaldi, Caterina , Farinea, Giovanni , Prager, Gerald W , Chan, Stephen L , Niger, Monica , Fornaro, Lorenzo , Casadei-Gardini, Andrea , Lo Prinzi, Federica , Rimassa, Lorenza , Saborowski, Anna , Rimini, Margherita , Ikeda, Masafumi , Abidoye, Oluseyi , Lucchetti, Jessica , Antonuzzo, Lorenzo , Kim, Jin Won , Nichetti, Federico , Pressiani, Tiziana , Vivaldi, Caterina , Rapposelli, Ilario Giovanni , Braconi, Chiara , Pinato, David J. , Tamburini, Emiliano , Pircher, Chiara , Tamberi, Stefano , Castet, Florian , Verrico, Monica , Parisi, Alessandro , Couto, Nuno, Chon, Hong Jae , Lamarca, Angela , Landriscina, Matteo , Finkelmeier, Fabian , Oneda, Ester , Avallone, Antonio , Dell’Aquila, Emanuela , Kim, Il Hwan, Formica, Vincenzo , Garajova, Ingrid , Kang, Beodeul , Corallo, Salvatore , Fenocchio, Elisabetta , Diana, Anna , Schirripa, Marta , Scartozzi, Mario , Ghidini, Michele , Prager, Gerald W. , Chan, Stephen L. , Warmington, Emily, Satake, Tomoyuki , Bekaii‐Saab, Tanios , Giordano, Guido , Ferrara, Michele , Kang, Minsu, Prete, Alessandra Anna , Bozzarelli, Silvia , Masi, Gianluca , Niger, Monica , Lonardi, Sara , Salani, Francesca , Camera, Silvia , Vogel, Arndt , Fornaro, Lorenzo , Casadei‐Gardini, Andrea
International Journal of Cancer | August 12, 2026 | Volume: No volume available | Issue: No issue available | Pages: No page available
ABSTRACT Many real‐world patients with advanced biliary tract cancer (BTC) are excluded by TOPAZ‐1 criteria; this study assessed cisplatin and gemcitabine plus durvalumab (CGD) efficacy and safety in these patients. Data from 1358 patients with advanced BTC treated with first‐line CGD across 55 international centers were retrospectively analyzed. Patients were classified as TOPAZ‐1‐in (meeting all inclusion criteria) or TOPAZ‐1‐out (meeting ≥ 1 exclusion criterion). Primary endpoints were overall survival (OS) and progression‐free survival (PFS); secondary endpoints included objective response rate (ORR), disease control rate (DCR), and safety. Furthermore, OS and PFS of the TOPAZ‐1‐out cohort were compared with reconstructed survival data from the phase III trial. 912 (67.1%) patients were classified as TOPAZ‐1‐in , and 446 (32.9%) as TOPAZ‐1‐out . After a median follow‐up of 14.5 months (95% CI: 13.7–36.5), OS was 16.1 months in TOPAZ‐1‐in and 12.5 months in TOPAZ‐1‐out (HR 0.69, 95% CI: 14.6–16.5, p = 0.0001); PFS was 8.2 versus 6.5 months (HR 0.73, 95% CI: 7.1–8.1, p < 0.0001). Median OS in the TOPAZ‐1‐out cohort (12.5 months) was nearly identical to the phase III trial experimental arm (12.9 months; HR 1.15, p = 0.13); for PFS the HR was 1.12 (95% CI 0.93–1.42; p = 0.09). Among TOPAZ‐1‐out patients, active infection, elevated bilirubin, and ECOG PS > 1 were linked to poorer OS, while no detrimental impact emerged for ALT/AST abnormalities, corticosteroid use, renal or hematologic parameters, or prior surgery within 6 months, suggesting treatment effectiveness was broadly maintained across clinical subgroups. Furthermore, TOPAZ‐1‐out patients experienced no significant increase in adverse events compared with the TOPAZ‐1‐in group. Real‐world data suggest CGD may be effective in TOPAZ‐1‐out patients, with no evident increase in toxicity, supporting the potential expanded use of the regimen in clinical practice.
Exploring the Role of Surgery After Systemic Therapy in Locally Advanced Biliary Tract Cancer: Outcomes From a Large Retrospective Multinational Real‐World Cohort Treated With Cisplatin, Gemcitabine, and Durvalumab
Rimini, Margherita , Lamarca, Angela, Ikeda, Masafumi, Kim, Jin Won , Vogel, Arndt, Peeters, Frederik , Pinato, David J., Pircher, Chiara, Martirena, A. , Perkhofer, Lukas , Pinto, C. , Kang, Beodeul, Pastorino, Alessandro , Changhoon, Yoo, Rodriquenz, Maria Grazia , Scartozzi, Mario, Zumstein, Lucrezia , Ghidini, Michele, Aprile, Giuseppe , Spinelli, Gian Paolo , Yung‐Yeh, Su, Bolnykh, Iakov, Lancianese, Alessia , Satake, Tomoyuki, Lavacchi, Daniele, Passeri, Laura , Kang, Minsu, Viola, Massimo, Persano, Mara , Balsano, Rita , Ratti, Francesca, Rimassa, Lorenza , Kim, Jin Won , Vivaldi, Caterina , Formica, Vincenzo , Farinea, Giovanni , Prager, Gerald W , Chan, Stephen L , Niger, Monica , Fornaro, Lorenzo , Casadei-Gardini, Andrea , Rimassa, Lorenza , Saborowski, Anna , Rimini, Margherita , Lamarca, Angela , Ikeda, Masafumi , Abidoye, Oluseyi , Lucchetti, Jessica , Antonuzzo, Lorenzo , Vogel, Arndt , Nichetti, Federico , Pressiani, Tiziana , Vivaldi, Caterina , Rapposelli, Ilario Giovanni , Braconi, Chiara , Pinato, David J. , Tamburini, Emiliano , Pircher, Chiara , Tamberi, Stefano , Castet, Florian , Verrico, Monica , Parisi, Alessandro , Couto, Nuno , Chon, Hong Jae , Landriscina, Matteo , Finkelmeier, Fabian , Oneda, Ester , Avallone, Antonio , Dell’Aquila, Emanuela , Kim, Il Hwan , Garajova, Ingrid , Kang, Beodeul , Corallo, Salvatore , Fenocchio, Elisabetta , Diana, Anna , Schirripa, Marta , Scartozzi, Mario , Ghidini, Michele , Prager, Gerald W. , Chan, Stephen L. , Bekaii‐Saab, Tanios , Ferrara, Michele , Kang, Minsu, Bozzarelli, Silvia , Masi, Gianluca , Niger, Monica , Lonardi, Sara , Salani, Francesca , Camera, Silvia , Ratti, Francesca , Fornaro, Lorenzo , Casadei‐Gardini, Andrea
International Journal of Cancer | July 29, 2026 | Volume: No volume available | Issue: No issue available | Pages: No page available
ABSTRACT Surgery after response to first‐line chemoimmunotherapy may offer a potential curative option for patients with locally advanced biliary tract cancer (BTC) initially considered unresectable. However, robust real‐world evidence in this setting remains limited. We retrospectively analyzed patients with locally advanced BTC treated with first‐line cisplatin, gemcitabine, and durvalumab (CGD) across 55 centers in 12 countries. Endpoints included overall survival (OS), progression‐free survival (PFS), disease‐free survival (DFS) among resected patients, objective response rate (ORR), and the prognostic impact of surgery. A multivariable logistic regression model was developed to predict surgical conversion using baseline clinical and laboratory variables. Among 1358 screened patients, 219 had locally advanced disease and were included in the analysis. Median follow‐up was 14.5 months. ORR was 34.6%, with median PFS and OS of 10.0 and 20.7 months, respectively. Twenty‐four patients (10.9%) underwent surgical resection after systemic therapy. Median OS was 22.5 months in resected patients versus 19.9 months in those who did not undergo surgery. Median DFS after resection was 15.8 months. Pathological lymph node involvement was independently associated with shorter DFS. Larger baseline tumor size correlated with higher odds of radiological response but not with OS. An exploratory elastic‐net model retained four baseline variables and showed moderate discriminative ability for surgical conversion, with an apparent AUC of 0.72 and an optimism‐corrected AUC of 0.65. First‐line CGD enabled secondary resection in approximately 11% of patients with locally advanced BTC. A simple baseline model may support patient selection for conversion surgery, although prospective validation is needed.