• Paolo Andrea Zucali, Tommaso De Pas, Giovannella Palmieri, Adolfo Favaretto, Antonio Chella, Marcello Tiseo, Michele Caruso, Matteo Simonelli, Matteo Perrino, Fabio De Vincenzo, Francesca Toffalorio, Vincenzo Damiano, Giulia Pasello, Erika Garbella, Marco Ali, Fabio Conforti, Margaret Ottaviano, Angela Cioffi, Sabino De Placido, Laura Giordano, Monica Bertossi, Annarita Destro, Luca Di Tommaso, and Armando Santoro.
• Paolo Andrea Zucali, Matteo Simonelli, Matteo Perrino, Fabio De Vincenzo, Laura Giordano, Monica Bertossi, Annarita Destro, Luca Di Tommaso, and Armando Santoro¸ Humanitas Clinical and Research Hospital; Tommaso De Pas, Francesca Toffalorio, Fabio Conforti, and Angela Cioffi, European Institute of Oncology; Paolo Andrea Zucali, Matteo Simonelli, Luca Di Tommaso, and Armando Santoro, Humanitas University, Milan; Giovannella Palmieri, Vincenzo Damiano, Margaret Ottaviano, and Sabino De Placido, Università Federico II, Naples; Adolfo Favaretto and Giulia Pasello, Istituto Oncologico Veneto, Padua; Antonio Chella and Erika Garbella, University Hospital, Pisa; Marcello Tiseo, Azienda Ospedaliero-Universitaria of Parma, Parma; and Michele Caruso and Marco Ali, Humanitas Centro Catanese di Oncologia, Catania, Italy.
• J. Clin. Oncol. 2018 Feb 1; 36 (4): 342-349.

AbstractPurpose No effective salvage treatments are available for patients with advanced/recurrent thymoma (T) or thymic carcinoma (TC) who have progressed after platinum-based chemotherapy. This study evaluated the activity of everolimus in patients with advanced/recurrent T or TC previously treated with cisplatin-containing chemotherapy. Patients and Methods This was a single-arm, single-stage, open-label, multicenter, phase II trial. Patients received oral everolimus 10 mg/d until disease progression, unacceptable toxicity, or patient refusal. A Fleming phase II trial was designed. The null hypothesis of a true disease control rate (DCR) of 40% was tested against a one-sided alternative of a true DCR of 60% (α = β = 0.10): If disease control were achieved in ≥ 21 of the first 41 evaluable patients, everolimus could be recommended for further evaluation. Progression-free survival, overall survival, and safety were also evaluated. Results From 2011 to 2013, 51 patients were enrolled (T, n = 32; TC, n = 19). Complete remission was observed in one patient with TC, partial response in five patients (T, n = 3; TC, n = 2), and stable disease in 38 patients (T, n = 27; TC, n= 11), with a DCR of 88% (T,: 93.8%; TC, 77.8%). With a median follow up of 25.7 months, median progression-free survival was 10.1 months (T,: 16.6 months; TC, 5.6 months), and median overall survival was 25.7 months (T, not reached; TC, 14.7 months). Fourteen patients had a serious drug-related adverse event; of these patients, nine permanently discontinued treatment. Three patients died of pneumonitis while in the study. Immunohistochemical positivity for p4E-BP1 or insulin-like growth factor-1 receptor was statistically significantly related to a shorter survival. Conclusion Everolimus may induce durable disease control in a high percentage of patients with T or TC, albeit with a potential high risk of fatal pneumonitis.

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