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Observational Study
. 2024 Jan 1;10(1):79-86.
doi: 10.1001/jamaoncol.2023.4845.

Practice Patterns for Organ Preservation in US Patients With Rectal Cancer, 2006-2020

Affiliations
Observational Study

Practice Patterns for Organ Preservation in US Patients With Rectal Cancer, 2006-2020

Anthony Loria et al. JAMA Oncol. .

Abstract

Importance: In March 2023, the National Comprehensive Cancer Network endorsed watch and wait for those with complete clinical response to total neoadjuvant therapy. Neoadjuvant therapy is highly efficacious, so this recommendation may have broad implications, but the current trends in organ preservation in the US are unknown.

Objective: To describe organ preservation trends among patients with rectal cancer in the US from 2006 to 2020.

Design, setting, and participants: This retrospective, observational case series included adults (aged ≥18 years) with rectal adenocarcinoma managed with curative intent from 2006 to 2020 in the National Cancer Database.

Exposure: The year of treatment was the primary exposure. The type of therapy was chemotherapy, radiation, or surgery (proctectomy, transanal local excision, no tumor resection). The timing of therapy was classified as neoadjuvant or adjuvant.

Main outcomes and measures: The primary outcome was the absolute annual proportion of organ preservation after radical treatment, defined as chemotherapy and/or radiation without tumor resection, proctectomy, or transanal local excision. A secondary analysis examined complete pathologic responses among eligible patients.

Results: Of the 175 545 patients included, the mean (SD) age was 63 (13) years, 39.7% were female, 17.4% had clinical stage I disease, 24.7% had stage IIA to IIC disease, 32.1% had stage IIIA to IIIC disease, and 25.7% had unknown stage. The absolute annual proportion of organ preservation increased by 9.8 percentage points (from 18.4% in 2006 to 28.2% in 2020; P < .001). From 2006 to 2020, the absolute rate of organ preservation increased by 13.0 percentage points for patients with stage IIA to IIC disease (19.5% to 32.5%), 12.9 percentage points for patients with stage IIIA to IIC disease (16.2% to 29.1%), and 10.1 percentage points for unknown stages (16.5% to 26.6%; all P < .001). Conversely, patients with stage I disease experienced a 6.1-percentage point absolute decline in organ preservation (from 26.4% in 2006 to 20.3% in 2020; P < .001). The annual rate of transanal local excisions decreased for all stages. In the subgroup of 80 607 eligible patients, the proportion of complete pathologic responses increased from 6.5% in 2006 to 18.8% in 2020 (P < .001).

Conclusions and relevance: This case series shows that rectal cancer is increasingly being managed medically, especially among patients whose treatment historically relied on proctectomy. Given the National Comprehensive Cancer Network endorsement of watch and wait, the increasing trends in organ preservation, and the nearly 3-fold increase in complete pathologic responses, international professional societies should urgently develop multidisciplinary core outcome sets and care quality indicators to ensure high-quality rectal cancer research and care delivery accounting for organ preservation.

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Conflict of interest statement

Conflict of Interest Disclosures: Dr Temple reported grants from the Patient-Centered Outcomes Research Institute and the Agency for Healthcare Research and Quality outside the submitted work. Dr Fleming reported royalties from UpToDate outside the submitted work. No other disclosures were reported.

Figures

Figure 1.
Figure 1.. Treatment Trends Over the Study Period (N = 175 545)
A, Organ preservation (local excision or multimodal therapy without tumor resection) vs proctectomy. B, Overall rates of proctectomy, local excision, and no tumor resection.
Figure 2.
Figure 2.. Treatment Trends by Clinical Stage of Rectal Cancer
Figure 3.
Figure 3.. Patients With Complete Pathologic Response (ypT0N0/NX) After Neoadjuvant Therapy

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