Phase II trial of thalidomide with chemotherapy and as maintenance therapy for patients with poor prognosis small-cell lung cancer

Background Despite the high response rates achieved following standard chemotherapy for small-cell lung cancer (SCLC), the majority of patients will subsequently die from disease progression. Materials and methods We examined the efficacy and toxicity of thalidomide, an anti-angiogenic agent, in combination with carboplatin and etoposide and as maintenance therapy in patients with untreated SCLC. Twenty-five chemotherapy-naive patients with extensive disease (ED) or limited disease (LD) SCLC were enrolled in a single-arm phase II study. Carboplatin and etoposide were given every 3 weeks for 6 cycles with concurrent thalidomide 100 mg orally daily. The treatment with thalidomide was continued as maintenance for up to 2 years. Results Median progression free and overall survival were 8.3 months and 10.1 months, respectively. One-year survival was 40% and the 1-year progression-free survival was 36%. The overall response rate was 68% (95% CI 46–85%) with four complete remissions (20%) and 13 partial remissions (48%). We observed no increase in chemotherapy related toxicity. Thalidomide was well-tolerated and median time on thalidomide treatment was 7.6 months. Conclusion Concurrent thalidomide with chemotherapy followed by maintenance thalidomide appears to be well tolerated. The results on tumour response rate and survival led us to initiate a randomised phase III trial in the United Kingdom.