Restricted accessResearch articleFirst published online 2020-8
Validation of Microsimulation Models against Alternative Model Predictions and Long-Term Colorectal Cancer Incidence and Mortality Outcomes of Randomized Controlled Trials
Background. This study aimed to assess the validity of 2 microsimulation models of colorectal cancer (CRC), Policy1-Bowel and ASCCA.Methods. The model-estimated CRC risk in population subgroups with different health statuses, “dwell time” (time from incident precancerous polyp to symptomatically detected CRC), and reduction in symptomatically detected CRC incidence after a one-time complete removal of polyps and/or undetected CRC were compared with published findings from 3 well-established models (MISCAN, CRC-SPIN, and SimCRC). Furthermore, 6 randomized controlled trials (RCTs) that provided screening using a guaiac fecal occult blood test (Funen trial, Burgundy trial, and Minnesota Colon Cancer Control Study [MCCCS]) or flexible sigmoidoscopy (NORCCAP, SCORE, and UKFSST) with long-term follow-up were simulated. Model-estimated long-term relative reductions of CRC incidence (RRinc) and mortality (RRmort) were compared with the RCTs’ findings. Results. The Policy1-Bowel and ASCCA estimates showed more similarities to CRC-SPIN and SimCRC. For example, overall dwell times estimated by Policy1-Bowel (24.0 years) and ASCCA (25.3) were comparable to CRC-SPIN (25.8) and SimCRC (25.2) but higher than MISCAN (10.6). In addition, ∼86% of Policy1-Bowel’s and ∼74% of ASCCA’s estimated RRinc and RRmort were consistent with the RCTs’ long-term follow-up findings. For example, at 17 to 18 years of follow-up, the MCCCS reported RRmort of 0.67 (95% confidence interval [CI], 0.51–0.83) and 0.79 (95% CI, 0.62–0.97) for the annual and biennial screening arm, respectively, and the UKFSST reported RRmort of 0.70 (95% CI, 0.62–0.79) for CRC at all sites and 0.54 (95% CI, 0.46–0.65) for distal CRC. The corresponding model estimates were 0.65, 0.74, 0.81, and 0.61, respectively, for Policy1-Bowel and 0.65, 0.70, 0.75, and 0.58, respectively, for ASCCA. Conclusion. Policy1-Bowel and ASCCA’s estimates are largely consistent with the data included for comparisons, which indicates good model validity.
KnudsenABZauberAGRutterCM, et al. Estimation of benefits, burden, and harms of colorectal cancer screening strategies: modeling study for the US Preventive Services Task Force. JAMA. 2016;315(23):2595–609.
2.
LewJBSimmsKTSmithMA, et al. National Cervical Screening Program Renewal: effectiveness modelling and economic evaluation in the Australia setting report. MSAC application no. 1276. 2013.
3.
LewJBSt JohnDJBXuXM, et al. Long-term evaluation of benefits, harms, and cost-effectiveness of the National Bowel Cancer Screening Program in Australia: a modelling study. Lancet Public Health. 2017;2(7):e331–e40.
4.
KitchenerHCCanfellKGilhamC, et al. The clinical effectiveness and cost-effectiveness of primary human papillomavirus cervical screening in England: extended follow-up of the ARTISTIC randomised trial cohort through three screening rounds. Health Technol Assess. 2014;18(23):1–196.
5.
LewJBSimmsKSmithMLewisHNealHCanfellK.Effectiveness modelling and economic evaluation of primary HPV screening for cervical cancer prevention in New Zealand. PLoS One. 2016;11(5):e0151619.
6.
ZauberAGLansdorp-VogelaarIKnudsenABWilschutJvanBMKuntzKM.Evaluating test strategies for colorectal cancer screening: a decision analysis for the U.S. Preventive Services Task Force. Ann Intern Med. 2008;149(9):659–69.
7.
EddyDMHollingworthWCaroJJTsevatJMcDonaldKMWongJB.Model transparency and validation: a report of the ISPOR-SMDM Modeling Good Research Practices Task Force—7. Value Health. 2012;15(6):843–50.
8.
GreuterMJXuXMLewJB, et al. Modeling the Adenoma and Serrated pathway to Colorectal CAncer (ASCCA). Risk Anal. 2014;34(5):889–910.
LewJBSt JohnDJBMacraeFA, et al. Evaluation of the benefits, harms and cost-effectiveness of potential alternatives to iFOBT testing for colorectal cancer screening in Australia. Int J Cancer. 2018;143(2):269–82.
11.
GreuterMJBerkhofJCanfellKLewJBDekkerECoupeVM.Resilience of a FIT screening programme against screening fatigue: a modelling study. BMC Public Health. 2016;16(1):1009.
12.
GreuterMJBerkhofJFijnemanRJ, et al. The potential of imaging techniques as a screening tool for colorectal cancer: a cost-effectiveness analysis. Br J Radiol. 2016;89(1063):20150910.
13.
GreuterMJDemirelELewJB, et al. Long-term impact of the Dutch colorectal cancer screening program on cancer incidence and mortality-model-based exploration of the serrated pathway. Cancer Epidemiol Biomarkers Prev. 2016;25(1):135–44.
14.
GreuterMJEde KlerkCMMeijerGADekkerECoupeVMH. Screening for colorectal cancer with fecal immunochemical testing with and without postpolypectomy surveillance colonoscopy: a cost-effectiveness analysis. Ann Intern Med. 2017;167(8):544–54.
15.
LewJBSt JohnDJBMacraeFA, et al. Benefits, harms and cost-effectiveness of potential age-extensions to the National Bowel Cancer Screening Program in Australia [published online September6, 2018]. Cancer Epidemiol Biomarkers Prev.
16.
WorthingtonJFelettoELewJB, et al. Evaluating health benefits and cost-effectiveness of a mass-media campaign for improving participation in the National Bowel Cancer Screening Program in Australia. Public Health. 2019;179:90–9.
17.
BronzwaerMESGreuterMJEBleijenbergAGCIJspeertJEGDekkerECoupeVMH. Impact of differences in adenoma and proximal serrated polyp detection rate on the long-term effectiveness of FIT-based colorectal cancer screening. BMC Cancer. 2018;18(1):465.
18.
VleugelsJLAGreuterMJEHazewinkelYCoupeVMHDekkerE. Implementation of an optical diagnosis strategy saves costs and does not impair clinical outcomes of a fecal immunochemical test-based colorectal cancer screening program. Endosc Int Open. 2017;5(12):E1197–207.
19.
KuntzKMLansdorp-VogelaarIRutterCM, et al. A systematic comparison of microsimulation models of colorectal cancer: the role of assumptions about adenoma progression. Med Decis Making. 2011;31(4):530–9.
20.
van BallegooijenMRutterCMKnudsenAB, et al. Clarifying differences in natural history between models of screening: the case of colorectal cancer. Med Decis Making. 2011;31(4):540–9.
21.
ParenteFVailatiCBoemoC, et al. Improved 5-year survival of patients with immunochemical faecal blood test-screen-detected colorectal cancer versus non-screening cancers in northern Italy. Dig Liver Disease. 2015;47(1):68–72.
22.
GillMDBrambleMGHullMA, et al. Screen-detected colorectal cancers are associated with an improved outcome compared with stage-matched interval cancers. Br J Cancer. 2014;111(11):2076–81.
23.
PandeRFroggattPBaragwanathPHarmstonC.Survival outcome of patients with screening versus symptomatically detected colorectal cancers. Colorectal Dis. 2013;15(1):74–9.
24.
StoopEMde HaanMCde WijkersloothTR, et al. Participation and yield of colonoscopy versus non-cathartic CT colonography in population-based screening for colorectal cancer: a randomised controlled trial. Lancet Oncol. 2012;13(1):55–64.
25.
RutterCMKnudsenABMarshTL, et al. Validation of models used to inform colorectal cancer screening guidelines: accuracy and implications. Med Decis Making. 2016;36(5):604–14.
26.
LinJSPiperMAPerdueLA, et al. Screening for Colorectal Cancer: A Systematic Review for the US Preventive Services Task Force. Rockville, MD: Agency for Healthcare Research and Quality; 2016.
27.
HamzaSCottetVTouillonN, et al. Long-term effect of faecal occult blood screening on incidence and mortality from colorectal cancer. Dig Liver Dis. 2014;46(12):1121–5.
28.
KronborgOJorgensenODFengerCRasmussenM.Randomized study of biennial screening with a faecal occult blood test: results after nine screening rounds. Scand J Gastroenterol. 2004;39(9):846–51.
29.
ShaukatAMonginSJGeisserMS, et al. Long-term mortality after screening for colorectal cancer. N Engl J Med. 2013;369(12):1106–14.
30.
SegnanNArmaroliPBonelliL, et al. Once-only sigmoidoscopy in colorectal cancer screening: follow-up findings of the Italian Randomized Controlled Trial—SCORE. J Natl Cancer Inst. 2011;103(17):1310–22.
31.
AtkinWWooldrageKParkinDM, et al. Long term effects of once-only flexible sigmoidoscopy screening after 17 years of follow-up: the UK Flexible Sigmoidoscopy Screening randomised controlled trial. Lancet. 2017;389(10076):1299–311.
32.
HolmeOLobergMKalagerM.Colorectal cancer and the effect of flexible sigmoidoscopy screening—reply. JAMA. 2014;312(22):2411–2.
33.
BarclayK; Cancer Council Australia Surveillance Colonoscopy Guidelines Working Party. Algorithm for Colonoscopic Surveillance Intervals—Adenomas. New South Wales: Cancer Council Australia; 2013.
34.
LiebermanDARexDKWinawerSJGiardielloFMJohnsonDALevinTR.Guidelines for colonoscopy surveillance after screening and polypectomy: a consensus update by the US Multi-Society Task Force on Colorectal Cancer. Gastroenterology. 2012;143(3):844–57.
35.
HassanCQuinteroEDumonceauJM, et al. Post-polypectomy colonoscopy surveillance: European Society of Gastrointestinal Endoscopy (ESGE) guideline. Endoscopy. 2013;45(10):842–51.
36.
CairnsSRScholefieldJHSteeleRJ, et al. Guidelines for colorectal cancer screening and surveillance in moderate and high risk groups (update from 2002). Gut. 2010;59(5):666–89.
AtkinWSEdwardsRKralj-HansI, et al. Once-only flexible sigmoidoscopy screening in prevention of colorectal cancer: a multicentre randomised controlled trial. Lancet. 2010;375(9726):1624–33.
39.
KuntzKMLansdorp-VogelaarIRutterCM, et al. A systematic comparison of microsimulation models of colorectal cancer: the role of assumptions about adenoma progression. Med Decis Making. 2011;31(4):530–9.
40.
LewJBSt JohnDJXuXM, et al. Long-term evaluation of benefits, harms, and cost-effectiveness of the National Bowel Cancer Screening Program in Australia: a modelling study. Lancet Public Health. 2017;2:e331–e40.
41.
RutterCMSavarinoJE.An evidence-based microsimulation model for colorectal cancer: validation and application. Cancer Epidemiol Biomarkers Prev. 2010;19(8):1992–2002.
42.
CeninDRSt JohnDJLedgerMJSlevinTLansdorp-VogelaarI.Optimising the expansion of the National Bowel Cancer Screening Program. Med J Aust. 2014;201(8):456–61.
43.
LewJBSimmsKTSmithMA, et al. Primary HPV testing versus cytology-based cervical screening in women in Australia vaccinated for HPV and unvaccinated: effectiveness and economic assessment for the National Cervical Screening Program. Lancet Public Health. 2017;2(2):e96–e107.
44.
MurphyJHalloranSGrayA.Cost-effectiveness of the faecal immunochemical test at a range of positivity thresholds compared with the guaiac faecal occult blood test in the NHS Bowel Cancer Screening Programme in England. BMJ Open. 2017;7(10):e017186.
45.
BuskermolenMGiniANaberSKToes-ZoutendijkEde KoningHJLansdorp-VogelaarI.Modeling in colorectal cancer screening: assessing external and predictive validity of MISCAN-colon microsimulation model using NORCCAP trial results. Med Decis Making. 2018;38(8):917–29.
46.
ThomasCWhyteSKearnsBChilcottJB.External validation of a colorectal cancer model against screening trial long-term follow-up data. Value Health. 2019;22(10):1154–61.
47.
BrayFFerlayJSoerjomataramISiegelRLTorreLAJemalA.Global cancer statistics 2018: GLOBOCAN estimates of incidence and mortality worldwide for 36 cancers in 185 countries. CA Cancer J Clin. 2018;68(6):394–424.
48.
HardcastleJDThomasWMChamberlainJ, et al. Randomised, controlled trial of faecal occult blood screening for colorectal cancer: results for first 107,349 subjects. Lancet. 1989;1(8648):1160–4.
49.
MandelJSBondJHBradleyM, et al. Sensitivity, specificity, and positive predictivity of the Hemoccult test in screening for colorectal cancers. The University of Minnesota’s Colon Cancer Control Study. Gastroenterology. 1989;97(3):597–600.
50.
GilbertsenVAChurchTRGreweFJ, et al. The design of a study to assess occult-blood screening for colon cancer. J Chronic Dis. 1980;33(2):107–14.
51.
MandelJSBondJHChurchTR, et al. Reducing mortality from colorectal cancer by screening for fecal occult blood. Minnesota Colon Cancer Control Study. N Engl J Med. 1993;328(19):1365–71.
52.
MandelJSChurchTREdererFBondJH.Colorectal cancer mortality: effectiveness of biennial screening for fecal occult blood. J Natl Cancer Inst. 1999;91(5):434–7.
53.
MandelJSChurchTRBondJH, et al. The effect of fecal occult-blood screening on the incidence of colorectal cancer. N Engl J Med. 2000;343(22):1603–7.
54.
FaivreJDancourtVLejeuneC, et al. Reduction in colorectal cancer mortality by fecal occult blood screening in a French controlled study. Gastroenterology. 2004;126(7):1674–80.
55.
TaziMAFaivreJDassonvilleFLamourJMilanCDurandG.Participation in faecal occult blood screening for colorectal cancer in a well defined French population: results of five screening rounds from 1988 to 1996. J Med Screen. 1997;4(3):147–51.
56.
KlaaborgKMadsenMSSondergaardOKronborgO.Participation in mass screening for colorectal cancer with fecal occult blood test. Scand J Gastroenterol. 1986;21(10):1180–4.
57.
KronborgOFengerCOlsenJBechKSondergaardO.Repeated screening for colorectal cancer with fecal occult blood test: a prospective randomized study at Funen, Denmark. Scand J Gastroenterol. 1989;24(5):599–606.
58.
JorgensenODKronborgOFengerC.A randomised study of screening for colorectal cancer using faecal occult blood testing: results after 13 years and seven biennial screening rounds. Gut. 2002;50(1):29–32.
59.
KronborgOFengerCSondergaardOPedersenKMOlsenJ.Initial mass screening for colorectal cancer with fecal occult blood test: a prospective randomized study at Funen in Denmark. Scand J Gastroenterol. 1987;22(6):677–86.
60.
KronborgOFengerCOlsenJJorgensenODSondergaardO.Randomised study of screening for colorectal cancer with faecal-occult-blood test. Lancet. 1996;348(9040):1467–71.
61.
GondalGGrotmolTHofstadBBretthauerMEideTJHoffG.The Norwegian Colorectal Cancer Prevention (NORCCAP) screening study: baseline findings and implementations for clinical work-up in age groups 50-64 years. Scand J Gastroenterol. 2003;38(6):635–42.
62.
BretthauerMGondalGLarsenK, et al. Design, organization and management of a controlled population screening study for detection of colorectal neoplasia: attendance rates in the NORCCAP study (Norwegian Colorectal Cancer Prevention). Scand J Gastroenterol. 2002;37(5):568–73.
63.
SegnanNSenoreCAndreoniB, et al. Baseline findings of the Italian multicenter randomized controlled trial of “once-only sigmoidoscopy”—SCORE. J Natl Cancer Inst. 2002;94(23):1763–72.
64.
AtkinWSCookCFCuzickJEdwardsRNorthoverJMWardleJ.Single flexible sigmoidoscopy screening to prevent colorectal cancer: baseline findings of a UK multicentre randomised trial. Lancet. 2002;359 (9314):1291–300.
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