Analytical decision model for sample size and effectiveness projections for use in planning a population-based randomized controlled trial of colorectal cancer screening

Sherry Y H Chiu, Nea Malila, Amy M F Yen, Ahti Anttila, Matti Hakama, H. H. Chen

Research output: Contribution to journalArticle

4 Citations (Scopus)

Abstract

Rationale, aims and objectives Population-based randomized controlled trials (RCTs) often involve enormous costs and long-term follow-up to evaluate primary end points. Analytical decision-simulated model for sample size and effectiveness projections based on primary and surrogate end points are necessary before planning a population-based RCT. Method Based on the study design similar to two previous RCTs, transition rates were estimated using a five-state natural history model [normal, preclinical detection phase (PCDP) Dukes' A/B, PCDP Dukes' C/D, Clinical Dukes' A/B and Clinical Dukes' C/D]. The Markov cycle tree was assigned transition parameters, variables related to screening and survival rate that simulated results of 10-year follow-up in the absence of screening for a hypothetical cohort aged 45-74 years. The corresponding screened arm was to simulate the results after the introduction of population-based screening for colorectal cancer with fecal occult blood test with stop screen design. Results The natural course of mean sojourn time for five-state Markov model were estimated as 2.75 years for preclinical Dukes' A/B and 1.38 years for preclinical Dukes' C/D. The expected reductions in mortality and Dukes' C/D were 13% (95% confidence intervals: 7-19%) and 26% (95% confidence intervals: 20-32%), respectively, given a 70% acceptance rate and a 90% colonoscopy referral rate. Sample sizes required were 86 150 and 65 592 subjects for the primary end point and the surrogate end point, respectively, given an incidence rate up to 0.0020 per year. Conclusions The sample sizes required for primary and surrogate end points and the projection of effectiveness of fecal occult blood test for colorectal cancer screening were developed. Both are very important to plan a population-based RCT.

Original languageEnglish
Pages (from-to)123-129
Number of pages7
JournalJournal of Evaluation in Clinical Practice
Volume17
Issue number1
DOIs
Publication statusPublished - Feb 2011
Externally publishedYes

Fingerprint

Community Health Planning
Early Detection of Cancer
Sample Size
Colorectal Neoplasms
Randomized Controlled Trials
Occult Blood
Biomarkers
Hematologic Tests
Confidence Intervals
Population
Colonoscopy
Natural History
Referral and Consultation
Survival Rate
Costs and Cost Analysis
Mortality
Incidence

Keywords

  • colorectal cancer
  • mortality reduction
  • natural history
  • randomized controlled trial
  • sample size
  • surrogate end point

ASJC Scopus subject areas

  • Public Health, Environmental and Occupational Health
  • Health Policy

Cite this

Analytical decision model for sample size and effectiveness projections for use in planning a population-based randomized controlled trial of colorectal cancer screening. / Chiu, Sherry Y H; Malila, Nea; Yen, Amy M F; Anttila, Ahti; Hakama, Matti; Chen, H. H.

In: Journal of Evaluation in Clinical Practice, Vol. 17, No. 1, 02.2011, p. 123-129.

Research output: Contribution to journalArticle

@article{10ecbb05f196433aab1a4c1c65fb5c2a,
title = "Analytical decision model for sample size and effectiveness projections for use in planning a population-based randomized controlled trial of colorectal cancer screening",
abstract = "Rationale, aims and objectives Population-based randomized controlled trials (RCTs) often involve enormous costs and long-term follow-up to evaluate primary end points. Analytical decision-simulated model for sample size and effectiveness projections based on primary and surrogate end points are necessary before planning a population-based RCT. Method Based on the study design similar to two previous RCTs, transition rates were estimated using a five-state natural history model [normal, preclinical detection phase (PCDP) Dukes' A/B, PCDP Dukes' C/D, Clinical Dukes' A/B and Clinical Dukes' C/D]. The Markov cycle tree was assigned transition parameters, variables related to screening and survival rate that simulated results of 10-year follow-up in the absence of screening for a hypothetical cohort aged 45-74 years. The corresponding screened arm was to simulate the results after the introduction of population-based screening for colorectal cancer with fecal occult blood test with stop screen design. Results The natural course of mean sojourn time for five-state Markov model were estimated as 2.75 years for preclinical Dukes' A/B and 1.38 years for preclinical Dukes' C/D. The expected reductions in mortality and Dukes' C/D were 13{\%} (95{\%} confidence intervals: 7-19{\%}) and 26{\%} (95{\%} confidence intervals: 20-32{\%}), respectively, given a 70{\%} acceptance rate and a 90{\%} colonoscopy referral rate. Sample sizes required were 86 150 and 65 592 subjects for the primary end point and the surrogate end point, respectively, given an incidence rate up to 0.0020 per year. Conclusions The sample sizes required for primary and surrogate end points and the projection of effectiveness of fecal occult blood test for colorectal cancer screening were developed. Both are very important to plan a population-based RCT.",
keywords = "colorectal cancer, mortality reduction, natural history, randomized controlled trial, sample size, surrogate end point",
author = "Chiu, {Sherry Y H} and Nea Malila and Yen, {Amy M F} and Ahti Anttila and Matti Hakama and Chen, {H. H.}",
year = "2011",
month = "2",
doi = "10.1111/j.1365-2753.2010.01378.x",
language = "English",
volume = "17",
pages = "123--129",
journal = "Journal of Evaluation in Clinical Practice",
issn = "1356-1294",
publisher = "Wiley-Blackwell",
number = "1",

}

TY - JOUR

T1 - Analytical decision model for sample size and effectiveness projections for use in planning a population-based randomized controlled trial of colorectal cancer screening

AU - Chiu, Sherry Y H

AU - Malila, Nea

AU - Yen, Amy M F

AU - Anttila, Ahti

AU - Hakama, Matti

AU - Chen, H. H.

PY - 2011/2

Y1 - 2011/2

N2 - Rationale, aims and objectives Population-based randomized controlled trials (RCTs) often involve enormous costs and long-term follow-up to evaluate primary end points. Analytical decision-simulated model for sample size and effectiveness projections based on primary and surrogate end points are necessary before planning a population-based RCT. Method Based on the study design similar to two previous RCTs, transition rates were estimated using a five-state natural history model [normal, preclinical detection phase (PCDP) Dukes' A/B, PCDP Dukes' C/D, Clinical Dukes' A/B and Clinical Dukes' C/D]. The Markov cycle tree was assigned transition parameters, variables related to screening and survival rate that simulated results of 10-year follow-up in the absence of screening for a hypothetical cohort aged 45-74 years. The corresponding screened arm was to simulate the results after the introduction of population-based screening for colorectal cancer with fecal occult blood test with stop screen design. Results The natural course of mean sojourn time for five-state Markov model were estimated as 2.75 years for preclinical Dukes' A/B and 1.38 years for preclinical Dukes' C/D. The expected reductions in mortality and Dukes' C/D were 13% (95% confidence intervals: 7-19%) and 26% (95% confidence intervals: 20-32%), respectively, given a 70% acceptance rate and a 90% colonoscopy referral rate. Sample sizes required were 86 150 and 65 592 subjects for the primary end point and the surrogate end point, respectively, given an incidence rate up to 0.0020 per year. Conclusions The sample sizes required for primary and surrogate end points and the projection of effectiveness of fecal occult blood test for colorectal cancer screening were developed. Both are very important to plan a population-based RCT.

AB - Rationale, aims and objectives Population-based randomized controlled trials (RCTs) often involve enormous costs and long-term follow-up to evaluate primary end points. Analytical decision-simulated model for sample size and effectiveness projections based on primary and surrogate end points are necessary before planning a population-based RCT. Method Based on the study design similar to two previous RCTs, transition rates were estimated using a five-state natural history model [normal, preclinical detection phase (PCDP) Dukes' A/B, PCDP Dukes' C/D, Clinical Dukes' A/B and Clinical Dukes' C/D]. The Markov cycle tree was assigned transition parameters, variables related to screening and survival rate that simulated results of 10-year follow-up in the absence of screening for a hypothetical cohort aged 45-74 years. The corresponding screened arm was to simulate the results after the introduction of population-based screening for colorectal cancer with fecal occult blood test with stop screen design. Results The natural course of mean sojourn time for five-state Markov model were estimated as 2.75 years for preclinical Dukes' A/B and 1.38 years for preclinical Dukes' C/D. The expected reductions in mortality and Dukes' C/D were 13% (95% confidence intervals: 7-19%) and 26% (95% confidence intervals: 20-32%), respectively, given a 70% acceptance rate and a 90% colonoscopy referral rate. Sample sizes required were 86 150 and 65 592 subjects for the primary end point and the surrogate end point, respectively, given an incidence rate up to 0.0020 per year. Conclusions The sample sizes required for primary and surrogate end points and the projection of effectiveness of fecal occult blood test for colorectal cancer screening were developed. Both are very important to plan a population-based RCT.

KW - colorectal cancer

KW - mortality reduction

KW - natural history

KW - randomized controlled trial

KW - sample size

KW - surrogate end point

UR - http://www.scopus.com/inward/record.url?scp=78751672732&partnerID=8YFLogxK

UR - http://www.scopus.com/inward/citedby.url?scp=78751672732&partnerID=8YFLogxK

U2 - 10.1111/j.1365-2753.2010.01378.x

DO - 10.1111/j.1365-2753.2010.01378.x

M3 - Article

VL - 17

SP - 123

EP - 129

JO - Journal of Evaluation in Clinical Practice

JF - Journal of Evaluation in Clinical Practice

SN - 1356-1294

IS - 1

ER -