Expert Point of View: Philip B. Paty, MD

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The main thing that should not be ignored is the importance of surveillance. If detected early, local regrowth is often minimal and asymptomatic and can be treated successfully. If delayed, it’s dangerous.
— Philip B. Paty, MD

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Philip B. Paty, MD, a surgical oncologist at Memorial Sloan Kettering Cancer Center, New York, commented on the findings for The ASCO Post. Dr. Paty is leading studies of surveillance at his own institution, which is not part of the International Watch & Wait Database.

Consistency of Outcomes

“The data reported by the international consortium are very similar to our data, in almost all aspects,” he indicated. He was referring to a 73-patient study from Memorial Sloan Kettering, reported at the 2015 Gastrointestinal Cancers Symposium (and soon to be published), in which 74% of patients following the watch-and-wait approach were able to avoid rectal surgery completely, and 26% underwent surgery due to regrowth of tumor.1

Of the 19 patients managed nonoperatively who had tumor regrowth, 2 underwent successful salvage therapy with local excision resulting in rectal preservation. The remaining 17 patients underwent rectal resections. The overall survival rate at 4 years was 91% for the watch-and-wait cohort. There were 6 deaths in the watch-and-wait group and 4 in a comparison group of 72 patients who underwent standard rectal surgery.

Dr. Paty said the International Watch & Wait Database findings “are very important, because they are showing consistency of outcomes among many centers. I think that what we can draw from this analysis is that when the tumor disappears—when surgeons carefully examine and find no evidence of cancer remaining—the clinical outcomes of a nonoperative approach are very similar across institutions, surgeons, and patients—even when patients receive different neoadjuvant therapies. Clinical complete response is a favorable marker for overall survival.”

However, some have questioned the wisdom of nonoperative management, most recently Ellis et al, who suggested this approach may not be generalizable to a broad population.2 They noted that a nonoperative approach is often the result of reduced access to optimal surgical care—ie, systemic barriers—and can represent “a disparity in appropriate care rather than an innovative and intentional treatment strategy.” Under these circumstances, survival without surgery has proven to be worse, they indicated.

Important Principles

Dr. Paty acknowledged that outcomes in some series are not always “excellent,” and he attributed this to failure to adhere to important principles of watch-and-wait. While participants in the International Watch & Wait Database and his own center are adhering to a certain paradigm, other researchers are retrospectively assessing outcomes among a heterogeneously treated group of patients. With nonoperative management, he said, one must adhere to three key components:

  1. Delivery of adequate neoadjuvant therapy in an acceptable fashion
  2. Proper assessment of clinical complete response at the proper time—generally 8 to 12 weeks post treatment—using the proper diagnostic tools (eg, endoscopic imaging)
  3. Strict surveillance for local ­regrowth.

“If you have all three of these components, you will get excellent results,” Dr. Paty commented. “The vast majority of patients not only do well oncologically, but 80% save their rectums.”

Unfavorable outcomes, on the other hand, are the result of nonadherence to this paradigm. “There may be many reasons why those patients did not do well,” he said. “For studies to be comparable, others will have to show that their patients were treated with watch-and-wait in mind from the start. They will need to show these three components.”

Well-Informed Patients

At Memorial Sloan Kettering, appropriate candidates can opt for nonoperative management either on their phase II study or off protocol, but in all cases they must follow the treatment paradigm and be well informed. Patients opting for surveillance off-study understand that “this is not the universally accepted, historical standard,” he said. “We tell them we believe it’s an emerging new standard for which there is increasing evidence that the outcomes are good.”

Dr. Paty acknowledged the word “standard” is a “delicate” concept that some will question. “You will get a diversity of opinion on this,” he acknowledged. “But if patients give informed consent and the three principles are followed, I believe that watch-and-wait is a reasonable standard by which to treat patients.… The main thing that should not be ignored is the importance of surveillance. If detected early, local regrowth is often minimal and asymptomatic and can be treated successfully. If delayed, it’s dangerous.” ■

Disclosure: Dr. Paty reported no potential conflicts of interest.


1. Smith JJ, Chow O, Eaton A, et al: Organ preservation in patients with rectal cancer with clinical complete response after neoadjuvant therapy. 2015 Gastrointestinal Cancers Symposium. Abstract 509. Presented January 17, 2015.

2. Ellis CT, Dusetzina SB, Sanoff H, et al: Long-term survival after chemoradiotherapy without surgery for rectal adenocarcinoma: A word of caution. JAMA Oncol 3:123-125, 2017.

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