Treatment with everolimus may be associated with durable disease control in patients with advanced or recurrent thymoma (T) or thymic carcinoma (TC), according to a recent study published in the Journal of Clinical Oncology (online December 14, 2017; doi:10.1200/JCO.2017.74.4078).
However, this treatment also carries a potentially high risk for fatal pneumonitis in this patient population.
Currently, there are no effective salvage treatments available for patients with advanced/recurrent T or TC.
For their study, the researchers administered 10 mg oral everolimus per day to 51 participants with either T (n = 32) or TC (n = 19) who had been previously treated with cisplatin-containing chemotherapy. Patients were treated until disease progression, unacceptable toxicity, or patient refusal. Median follow-up lasted 25.7 months.
The researchers tested the null hypothesis of a true disease control rate (DCR) of 40% against a 1-sided alternative of a true DCR of 60%. They noted that everolimus could be recommended for further study if at least 21 of the first 41 evaluable patients achieved disease control. Factors including progression-free survival, overall survival, and safety were also examined.
Results of the study indicated that 1 participant with TC achieved complete remission with everolimus treatment. In addition, 3 participants with T and 2 participants with TC achieved partial response, and 27 participants with T and 11 patients with TC achieved stable disease (DCR: 88%).
Over the course of follow-up, median progression-free survival was found to be 10.1 months (T: 16.6 months; TC: 5.6 months), and median overall survival was found to be 25.7 months (T: not reached; TC: 14.7 months).
Serious adverse events associated with the study drug occurred in 14 participants, of whom 9 permanently discontinued treatment. Additionally, fatal pneumonitis occurred in 3 participants during the study.
“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,” the researchers concluded.—Christina Vogt