Neoadjuvant Therapy Versus Upfront Surgery for Patients With Clinical Stage 2 or 3 Esophageal Squamous Cell Carcinoma: A Cost-Effectiveness Analysis

Background Although neoadjuvant therapy followed by surgery (NT) is the standard of care for esophageal cancer in Western countries, upfront surgery (US) followed by adjuvant therapy (when indicated) still is commonly used in Asia to minimize overtreatment. This study investigated the cost-effectiveness of NT versus US for patients with esophageal squamous cell carcinoma (ESCC). Methods Patients with a diagnosis of ESCC between 2010 and 2015 were divided into NT or US according to the intention to treat. Two propensity score-matched groups of patients with clinical stage 2 (135 pairs) or stage 3 (194 pairs) disease were identified and compared in terms of overall survival (OS) and direct costs incurred within 3 years after diagnosis. Results The esophagectomy rates after NT were 82% for stage 2 and 88% for stage 3 disease. Compared with US, surgery after NT was associated with higher R0 resection rates, a lower number of dissected lymph nodes, and similar postoperative mortality. On an intention-to-treat analysis, stage 3 patients who received NT had a significantly better 3-year OS rate (45%) than those treated with US (37%) (p = 0.029) without significant cost increases (p = 0.89). However, NT for clinical stage 2 disease neither increased costs nor improved 3-year OS rates (47% vs 47%; p = 0.88). At a willingness-to-pay level of US$50,000 per life-year, the probability of NT being cost-effective was 92% for stage 3 versus 59% for stage 2 ESCC. Conclusion Because of its higher cost-effectiveness, NT is preferable to US for patients with clinical stage 3 ESCC, but US remains a viable option for stage 2 disease.