Phase II study of everolimus in patients with thymoma and thymic carcinoma previously treated with cisplatin-based chemotherapy

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 GiordanoMonica Bertossi, Annarita Destro, Luca Di Tommaso, Armando Santoro

Research output: Contribution to journalArticlepeer-review


Purpose: 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.

Original languageEnglish
Pages (from-to)342-349
Number of pages8
JournalJournal of Clinical Oncology
Issue number4
Publication statusPublished - Feb 1 2018

ASJC Scopus subject areas

  • Oncology
  • Cancer Research


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