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. 2025 Sep 8;6(3):e602.
doi: 10.1097/AS9.0000000000000602. eCollection 2025 Sep.

Patient-Reported Outcomes After Neoadjuvant Therapy and Watch-and-Wait for Rectal Cancer: A Systematic Review and Meta-Analysis

Bailey K Hilty Chu  1 , Anthony Loria  1 , Totadri Dhimal  1 , Lydia Rafferty  2 , Marie Jacobs  1 , Megan Boyer  1 , Paula Cupertino  1 , Fernando Colugnati  3 , Erika Ramsdale  4 , Fergal Fleming  1
Affiliations

Patient-Reported Outcomes After Neoadjuvant Therapy and Watch-and-Wait for Rectal Cancer: A Systematic Review and Meta-Analysis

Bailey K Hilty Chu et al. Ann Surg Open. .

Abstract

Objective: To evaluate patient-reported outcomes (PROs) in rectal cancer patients who underwent neoadjuvant therapy followed by watch-and-wait.

Background: Advancements in rectal cancer therapies have improved oncologic outcomes and increased focus on long-term treatment effects. Watch-and-wait, which aims for organ preservation in patients with a clinical complete response to neoadjuvant therapy, is used more frequently. While watch-and-wait may reduce functional impairments associated with proctectomy, its association with PROs is poorly understood.

Methods: We performed a systematic review and meta-analysis of studies reporting PROs for patients with stage I-III rectal adenocarcinoma treated with neoadjuvant therapy and watch-and-wait. Random-effects model meta-analysis was performed when feasible; otherwise, data were synthesized through a narrative review.

Results: Twenty-five studies (3139 patients) reported PROs for patients receiving neoadjuvant therapy followed by watch-and-wait (n = 1283), proctectomy (n = 1583), local excision (n = 263), or declined surgery (n = 10). Most studies (64.0%) excluded patients with local regrowth, leaving PRO data for 103 such patients. Fourteen studies compared PROs between treatment groups after neoadjuvant therapy. Meta-analyses demonstrated fewer bowel symptoms and improved quality of life (QoL) in 1/6 subscales for watch-and-wait. Across 24 QoL comparisons, 14 showed improvement in some subscales for watch-and-wait, while 10 found no difference. For 24 symptom scale comparisons, 11 indicated some improvement with watch-and-wait, and 13 found no differences between groups.

Conclusions: Watch-and-wait offers the potential to avoid permanent ostomy and surgical morbidity. However, more robust data are needed to assess its association with PROs compared to proctectomy or local excision. Researchers should prioritize publishing QoL data to better inform shared decision-making.

Keywords: patient-reported outcomes; quality-of-life; rectal cancer; watch-and-wait.

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Conflict of interest statement

Disclosure: F.F. received royalties from UpToDate unrelated to this study. The other authors have no conflicts of interest to disclose.

Figures

FIGURE 1.
FIGURE 1.
Study selection. PRISMA diagram of article selection.
FIGURE 2.
FIGURE 2.
Results of meta-analyses. Forest plots from random-effects model for select EORTC-QLQ-C30 (A) and LARS (B) scores.
FIGURE 3.
FIGURE 3.
Differences in other QoL or function scales between treatment groups. Absolute difference in each measure reported by studies that demonstrated statistically significant differences between the watch-and-wait and comparator group, though with insufficient data for pooled analysis of treatment effect between groups. A indicates anxiety; AP, abdominal pain; BFI, Memorial Sloan Kettering Bowel Function Instrument; BI, body image; BM, blood & mucous in stool; BP, buttock pain; CCS, FACT-C colorectal cancer subscale; DM, dry mouth; DP, defecation problems; E, embarrassment; ERF, emotional role function; GH, general health; MH, mental health; P, pain; PF, physical function; PRF, physical role function; SF, sexual function; T, taste; TOI, FACT-C Trial outcome index; UF, urinary frequency; UI, urinary incontinence; V, vitality (energy/fatigue).
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References

    1. Sung H, Ferlay J, Siegel RL, et al. Global cancer statistics 2020: GLOBOCAN estimates of incidence and Mortality Worldwide for 36 cancers in 185 countries. CA Cancer J Clin. 2021;71:209–249. - PubMed
    1. Siegel RL, Giaquinto AN, Jemal A. Cancer statistics, 2024. CA Cancer J Clin. 2024;74:12–49. - PubMed
    1. United States Department of Health and Human Services, Food and Drug Administration. Clinical trial endpoints for the approval of cancer drugs and Biologics. 2018; 1–19. Available at: https://www.fda.gov/media/71195/download. Accessed January 15, 2025.
    1. CHMP CFMPFHU. Regulatory guidance for the use of health-related quality of life (HRQL) measures in the evaluation of medicinal products - Scientific guideline. REFLECTION PAPER ON THE REGULATORY GUIDANCE FOR THE USE OF HEALTHRELATED QUALITY OF LIFE (HRQL) MEASURES IN THE EVALUATION OF MEDICINAL PRODUCTS. Eurpean Medicines Agency; 2005. Available at: https://www.ema.europa.eu/en/regulatory-guidance-use-health-related-qual.... Accessed January 15, 2025.
    1. United States Department of Health and Human Services, Food and Drug Administration. Guidance for Industry Patient-Reported Outcome Measures: Use in Medical Product Development to Support Labeling Claims. 2009: 1–43. Available at: https://www.fda.gov/media/77832/download. Accessed Jaunary 15, 2025.

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