Surgery has long been the primary curative modality for localized rectal cancer. Neoadjuvant chemoradiation has significantly improved local control rates and, in a significant minority, eradicated all disease. Patients who achieve a pathologic complete response to neoadjuvant therapy have an excellent prognosis, although the combination treatment is associated with long-term morbidity. Because of this, a nonoperative management (NOM) strategy has been pursued to preserve sphincter function in select patients. Clinical and radiographic findings are used to identify patients achieving a clinical complete response to chemoradiation, and they are then followed with intensive surveillance. Incomplete, nonresponding and those demonstrating local progression are referred for salvage with standard surgery. Habr-Gama and colleagues have published extensively on this treatment strategy and have laid the groundwork for this approach. This watch-and-wait strategy has evolved over time, and several groups have now reported their results, including recent prospective experiences. Although initial results appear promising, several significant challenges remain for NOM of rectal cancer. Further study is warranted before routine implementation in the clinic. Cancer 2016;122:34-41. V C 2015 American Cancer Society.KEYWORDS: neoadjuvant therapy, nonoperative, radiation therapy, rectal cancer, watchful waiting.
INTRODUCTIONIn 2015, an estimated 40,000 cases of rectal cancer will be diagnosed in the United States.1 Surgical therapy, by either low anterior resection (LAR) or abdominoperineal resection (APR), has long been considered the primary curative modality for localized disease. In recent decades, a paradigm shift for locally advanced disease has occurred: postoperative chemoradiation therapy (CRT) has largely been supplanted by a neoadjuvant approach, either through short-course radiation therapy (RT) alone or long-course CRT. This shift has been paralleled by improvements in preoperative imaging and staging, including endoscopic ultrasound (EUS) and magnetic resonance imaging (MRI), as well as advancements in surgical techniques. Although the initial rationale for neoadjuvant treatment was to decrease local recurrence rates, improve treatment tolerability, and increase sphincter-preserving surgery rates, a small but significant percentage of patients were found to be complete pathologic responders upon examination of the surgical specimen. Investigators have since explored definitive CRT and careful follow up, also called nonoperative management (NOM), as a curative approach for select patients, with surgery reserved as salvage therapy. This review aims to critically evaluate NOM of rectal cancer in the modern era.
BACKGROUNDThe preferred surgical approach for rectal cancer depends on the location of the cancer in relation to the anal sphincter complex: a sphincter-preserving LAR is reserved for proximal and midrectal lesions, and an APR requiring a permanent stoma is reserved for distal lesions. Both types of resections now...