Faculty Collaboration Database

Utilizing Patient Input in Rectal Cancer Trial Design. J Natl Cancer Inst 2025 Jul 01;117(7):1421-1428

Date

03/20/2025

Pubmed ID

40108482

Pubmed Central ID

PMC12229457

DOI

10.1093/jnci/djaf074

Scopus ID

2-s2.0-105009908498 (requires institutional sign-in at Scopus site)   3 Citations

Abstract

BACKGROUND: The treatment of locally advanced rectal cancer now includes "watch-and-wait" (WW) management for patients who exhibit a clinical complete response (cCR) to total neoadjuvant therapy (TNT). We assessed patients' knowledge and preferences regarding WW with the goal of incorporating patient input into clinical trial design.

METHODS: Rectal cancer patients in advocacy groups (COLONTOWN/Rectalburgh and Fight CRC) were surveyed regarding perceptions about treatment strategies, tolerable risks of treatment failure, and preferences on clinical trial design. Surveys were initially distributed in 2019, with the shift toward WW prompting re-survey of select questions in 2023.

RESULTS: All respondents preferred enrollment in trials that incorporated patient input. Most respondents (76%) preferred treatment that incorporated chemotherapy escalation over radiation escalation. Furthermore, when presented with a hypothetical patient who developed a cCR after chemoradiation (without the option for WW), 58% of patients chose surgical treatment (with 100% risk of permanent ostomy and 80% chance of cure) over omission of surgery (with <80% chance of cure). In contrast, when the hypothetical patient developed a cCR after TNT (with an option for WW), 82% of patients opted for selective WW over surgery.

CONCLUSIONS: This work successfully integrated patient advocacy groups as a resource for obtaining patient input. Surveys revealed patients had a strong preference for chemotherapy escalation, for selective WW when offered, and for incorporation of input into future clinical trial design. These findings describe how well-informed patients approach complex medical decision-making and serve as a starting point for future studies seeking to incorporate patient voices.

Author List

Alvarez J, Zambare W, George M, Garcia R, Gallagher P, Kitchens C, Bercz A, Kim MJ, Romesser PB, Cercek A, Garcia-Aguilar J, Sanoff H, George TJ, Hong TS, Yothers G, Philip PA, Al Baghdadi T, Alese OB, Shergill A, O'Reilly EM, Meyerhardt JA, Horvat N, Dasari A, Hall WA, Shi Q, Schrag D, Smith JJ

Author

William Adrian Hall MD Chair, Professor in the Radiation Oncology department at Medical College of Wisconsin

MESH terms used to index this publication - Major topics in bold

Adult
Aged
Clinical Trials as Topic
Female
Humans
Male
Middle Aged
Neoadjuvant Therapy
Patient Participation
Patient Preference
Rectal Neoplasms
Research Design
Surveys and Questionnaires
Watchful Waiting