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Organ-sparing treatment strategy appears promising in early-stage rectal cancer

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January 11, 2019

Neoadjuvant chemoradiotherpy followed by transanal endoscopic microsurgery may be a viable alternative to conventional total mesorectal excision for patients with early-stage rectal cancer, according to results of a phase 2 feasibility study.

“Treatment of rectal cancer is shifting toward organ preservation aiming to reduce surgery-related morbidity,” Rutger C. H. Stijns, MD, researcher in the department of surgery of Radboud University Medical Centre in Nijmegen, the Netherlands, and colleagues wrote. “Short-term outcomes of organ-preserving strategies are promising, but long-term outcomes are scarce in the literature.”

Researchers sought to examine long-term outcomes and health-related quality of life among patients with early-stage rectal cancer who underwent neoadjuvant chemoradiotherapy followed by transanal endoscopic microsurgery.

The study included 55 patients (55% men; mean age, 64 years, range, 39-82) admitted to referral centers in the Netherlands between February 2011 and September 2012.

Minimal residual disease served as the primary endpoint. Locoregional recurrence and health-related quality of life served as secondary endpoints.

Median follow-up was 53 months.

Two patient deaths occurred during chemoradiotherapy treatment; one patient discontinued chemoradiotherapy; and one patient was lost to follow-up.

Eighty-five percent of patients (n = 47) underwent transanal endoscopic microsurgery after chemoradiotherapy, of whom 74% (n = 35) were treated successfully with local excision alone.

Sixteen patients underwent total mesorectal excision, including four patients who had insufficient responses to chemoradiotherapy, eight patients who underwent completion with total mesorectal excision after transanal endoscopic microsurgery, and four patients who developed an intraluminal local recurrence after local excision.

At 5 years, DFS was 81.6%, OS was 82.8% and the actuarial local recurrence rate was 7.7%.

Sixty-four percent of patients achieved successful organ preservation with acceptable long-term outcomes and health-related quality of life.

Researchers observed improvements in emotional well-being among those treated with local excision. Mean baseline score was 72 (95% CI, 67.1-80.1) compared with a mean follow-up score of 86.9 (95% CI, 79.2-94.7; P =.001).

Among those with successful organ preservation, half had major anterior resection syndrome, 28% had minor anterior resection syndrome and 22% had low anterior resection syndrome.

“In early-stage rectal cancer, chemoradiotherapy enables organ preservation with additional transanal endoscopic microsurgery in approximately two-thirds of patients with good long-term oncological outcome and health-related quality of life,” Stijns and colleagues wrote. “This multimodality treatment triggers a certain degree of bowel dysfunction, and one-third of patients still undergo radical surgery and are overtreated by chemoradiotherapy.”


Based on these findings, the “logical next step” is to evaluate neoadjuvant chemotherapy and a watch-and-wait approach or selective salvage surgery based on tumor response, Julio Garcia-Aguilar, MD, PhD, chief of colorectal service and Benno C. Schmidt chair in the department of surgical oncology at Memorial Sloan Kettering Cancer Center, wrote in an accompanying editorial.

“Evidence for the safety of a watch-and-wait approach in patients with a clinical complete response to chemoradiotherapy is accumulating, and it seems reasonable to speculate whether such patients require surgery at all,” Garcia-Aguilar wrote. “Since local excision is associated with some morbidity and impairment of bowel function, patients who can avoid it are likely to benefit from a higher quality of life.” – by Jennifer Southall

Disclosures: Stijns and all other authors report no relevant financial disclosures. Garcia-Aguilar reports serving as a paid consultant for Intuitive Surgical, Johnson & Johnson and Medtronic.

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