Treatment for patients with rectal cancer and a clinical complete response to neoadjuvant therapy: A decision analysis Journal Article


Authors: Neuman, H. B.; Elkin, E. B.; Guillem, J. G.; Paty, P. B.; Weiser, M. R.; Wong, W. D.; Temple, L. K.
Article Title: Treatment for patients with rectal cancer and a clinical complete response to neoadjuvant therapy: A decision analysis
Abstract: PURPOSE: A clinical complete response to neoadjuvant therapy occurs in a subset of patients with rectal cancer. Management of these patients is controversial and tension exists between the recurrence risk with observation, and the impact of surgery on quality-of-life. Therefore, the objective was to develop a decision-analytic model to evaluate the relative benefits of surgery vs. observation in rectal cancer patients who achieve clinical complete response after neoadjuvant chemoradiation. METHODS: Clinically relevant inputs and events, including the ability to identify complete responders, likelihood of relapse and of salvage surgery after relapse, and utilities for each health state, were simulated by use of a Markov state-transition model. Transition probabilities and health-state utilities were derived from the literature and expert consensus. One-way and two-way sensitivity analyses were performed to assess the robustness of model results to assumptions. The primary outcome was quality-adjusted life expectancy. RESULTS: In the base-case analysis, the quality-adjusted life expectancy with surgery exceeded observation (5.63 vs. 5.34 quality-adjusted life-years). Sensitivity analysis demonstrated that observation was preferred to surgery if the ability to correctly identify patients with true complete responses exceeded 58 percent, if quality-of-life after surgery was poor (utility <0.81), or if the relative reduction in recurrence risk with surgery was <43 percent when compared with observation. CONCLUSIONS: Our model outlines the issues associated with surgery vs. observation, and suggests that surgery is beneficial for the average patient with rectal cancer with a clinical complete response after neoadjuvant therapy. Current limitations in the clinical assessment of patient response to chemoradiation constitute an important factor influencing our results, and therefore warrant further investigation. ©The ASCRS 2009.
Keywords: survival; cancer chemotherapy; cancer survival; treatment outcome; treatment response; aged; cancer surgery; mortality; cancer recurrence; salvage therapy; multimodality cancer therapy; adjuvant therapy; cancer adjuvant therapy; cancer radiotherapy; chemotherapy, adjuvant; neoadjuvant therapy; radiotherapy, adjuvant; recurrence risk; antineoplastic agent; sensitivity analysis; neoplasm recurrence, local; radiation; markov chains; cancer mortality; distant metastasis; simulation; health status; probability; models, statistical; tumor recurrence; adjuvant chemotherapy; cancer relapse; clinical decision making; rectal neoplasms; rectum cancer; rectum tumor; statistical model; life expectancy; quality adjusted life year; clinical observation; medical expert; markov model; rectal cancer; quality-adjusted life years
Journal Title: Diseases of the Colon and Rectum
Volume: 52
Issue: 5
ISSN: 0012-3706
Publisher: Lippincott Williams & Wilkins  
Date Published: 2009-05-01
Start Page: 863
End Page: 871
Language: English
DOI: 10.1007/DCR.0b013e31819eefba
PUBMED: 19502849
PROVIDER: scopus
DOI/URL:
Notes: --- - "Cited By (since 1996): 1" - "Export Date: 30 November 2010" - "CODEN: DICRA" - "Source: Scopus"
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MSK Authors
  1. Philip B Paty
    499 Paty
  2. Jose Guillem
    414 Guillem
  3. Heather B Neuman
    18 Neuman
  4. Elena B Elkin
    163 Elkin
  5. Martin R Weiser
    538 Weiser
  6. Larissa Temple
    193 Temple
  7. Douglas W Wong
    178 Wong