Cost-Effectiveness and Quality-Adjusted Survival of Watch-and-Wait after Complete Response to Chemoradiotherapy for Rectal Cancer. Journal of the National Cancer Institute Miller, J. A., Wang, H. n., Chang, D. T., Pollom, E. L. 2020


Neoadjuvant chemoradiotherapy (CRT) followed by total mesorectal excision (TME) is the standard treatment for locally-advanced rectal cancer. There is interest in de-escalating local therapy after a clinical complete response (cCR) to CRT. We hypothesized that a watch-and-wait (WW) strategy offers comparable cancer-specific survival (CSS), superior quality-adjusted survival, and reduced cost compared to upfront TME.We developed a decision-analytic model to compare WW, low anterior resection (LAR), and abdominoperineal resection (APR) for patients achieving a cCR to CRT. Rates of local regrowth, pelvic recurrence, and distant metastasis were derived from series comparing WW to TME after pathologic complete response. Lifetime incremental costs and quality-adjusted life-years (QALYs) were calculated between strategies, and sensitivity analyses were performed to study model uncertainty.The base case 5-year CSS was 93.5% (95% confidence interval [CI] 91.5 to 94.9%) on a WW program, compared to 95.9% (95% CI 93.6 to 97.4%) after upfront TME. WW was dominant relative to LAR, with cost savings of $28,500 (95% CI $22,200 to $39,000) and incremental QALY of 0.527 (95% CI 0.138 to 1.125). WW was also dominant relative to APR, with cost savings of $32,100 (95% CI $21,800 to $49,200) and incremental QALY of 0.601 (95% CI 0.213 to 1.208). WW remained dominant in sensitivity analysis unless the rate of surgical salvage fell to 73.0%.Using current multi-institutional recurrence estimates, we observed comparable CSS, superior quality-adjusted survival, and decreased costs with WW compared to upfront TME. Upfront TME was preferred when surgical salvage rates were low.

View details for DOI 10.1093/jnci/djaa003

View details for PubMedID 31930400