Everolimus in Previously Treated Thymoma and Thymic Carcinoma


Key Points

  • Everolimus was associated with a disease control rate of 88%.
  • Three patients (6%) died from pneumonitis on study.

In an Italian phase II study reported in the Journal of Clinical Oncology, Zucali et al found that everolimus was active in cisplatin-pretreated thymoma and thymic carcinoma; however, a high rate of fatal pneumonitis was observed.

Study Details

In the study, 51 patients with advanced/recurrent disease previously treated with cisplatin-based chemotherapy, including 32 with thymoma and 19 with thymic carcinoma, received everolimus 10 mg/d until disease progression, unacceptable toxicity, or patient refusal. The primary outcome measure was disease control rate.

Disease Control Rate

Median follow-up was 25.7 months. Complete remission was observed in 1 patient (with thymic carcinoma), partial response in 5 (3 with thymoma, 2 with thymic carcinoma), and stable disease in 38 (27 with thymoma, 11 with thymic carcinoma. The disease control rate among all patients was 88%, including 94% in patients with thymoma and 78% in those with thymic carcinoma. Median progression-free survival was 10.1 months, including 16.6 months in patients with thymoma and 5.6 months in patients with thymic carcinoma. Median overall survival was 25.7 months, including not reached in patients with thymoma and 14.7 months in thymic carcinoma patients. Positivity for p4E-BP1 or insulin-like growth factor-1 receptor on immunohistochemistry was significantly associated with shorter survival.

Adverse Events

The most common treatment-related adverse events of any grade were stomatitis (66%), fatigue (48%), mucositis (36%), and pneumonitis in (36%). Treatment-related grade ≥ 3 adverse events occurred in 28%, with the most common being liver toxicity (8%). Serious drug-related adverse events occurred in 28% and resulted in discontinuation of treatment in nine patients (18%). Three patients (6%) died from pneumonitis while on study.

The investigators concluded, “Everolimus may induce durable disease control in a high percentage of patients with [thymoma or thymic carcinoma], albeit with a potential high risk of fatal pneumonitis.”

Paolo Andrea Zucali, MD, of Humanitas Clinical and Research Center, Milan, Italy, is the corresponding author for the Journal of Clinical Oncology article. 

The content in this post has not been reviewed by the American Society of Clinical Oncology, Inc. (ASCO®) and does not necessarily reflect the ideas and opinions of ASCO®.