Sunitinib in patients with chemotherapy-refractory thymoma and thymic carcinoma: an open-label phase 2 trial

摘要: Summary Background No standard treatments are available for advanced thymic epithelial tumours after failure of platinum-based chemotherapy. We investigated the activity sunitinib, an orally administered tyrosine kinase inhibitor. Methods Between May 15, 2012, and Oct 2, 2013, we did open-label phase 2 trial in patients with histologically confirmed chemotherapy-refractory tumours. Patients were eligible if they had disease progression at least one previous regimen platinum-containing chemotherapy, Eastern Cooperative Oncology Group performance status two or lower, measurable disease, adequate organ function. received 50 mg sunitinib once a day, 6-week cycles (ie, 4 weeks treatment followed by without treatment), until tumour unacceptable toxic effects arose. The primary endpoint was investigator-assessed best response any point, which analysed separately thymoma carcinoma cohorts. who cycle their reassessed included analyses response. registered ClinicalTrials.gov, number NCT01621568. Findings 41 enrolled, 25 16 thymoma. One patient deemed ineligible enrolment not receive protocol treatment. Of treatment, individual assessable because she died. Median follow-up on 17 months (IQR 14·0–18·4). 23 carcinoma, six (26%, 90% CI 12·1–45·3, 95% 10·2–48·4) partial responses, 15 (65%, 42·7–83·6) achieved stable (9%, 1·1–28·0) progressive disease. thymoma, (6%, 0·2–30·2) response, 12 (75%, 47·6–92·7) three (19%, 4·1–45·7) most common grade 3 treatment-related adverse events lymphocytopenia (eight [20%] 40 patients), fatigue [20%]), oral mucositis [20%]). Five (13%) decreases left-ventricular ejection fraction, (8%) events. Three died during including cardiac arrest that possibly treatment-related. Interpretation Sunitinib is active previously treated carcinoma. Further studies needed to identify potential biomarkers activity. Funding National Cancer Institute (Cancer Therapy Evaluation Program).