Evaluating comparative effectiveness with observational data: endoscopic ultrasound and survival in pancreatic cancer

Cancer. 2013 Nov 1;119(21):3861-9. doi: 10.1002/cncr.28295. Epub 2013 Aug 6.

Abstract

Background: A previous observational study reported that endoscopic ultrasound (EUS) is associated with improved survival in older patients with pancreatic cancer. The objective of this study was to reevaluate this association using different statistical methods to control for confounding and selection bias.

Methods: Surveillance, Epidemiology, and End Results (SEER)-Medicare linked data (1992-2007) was used to identify patients with locoregional pancreatic cancer. Two-year survival in patients who did and did not receive EUS was compared by using standard Cox proportional hazards models, propensity score methodology, and instrumental variable analysis.

Results: EUS was associated with improved survival in both unadjusted (hazard ratio [HR] = 0.67, 95% confidence interval [CI] = 0.63-0.72) and standard regression analyses (HR = 0.78, 95% CI = 0.73-0.84) which controlled for age, sex, race, marital status, tumor stage, SEER region, Charlson comorbidity, year of diagnosis, education, preoperative biliary stenting, chemotherapy, radiation, and pancreatic resection. Propensity score adjustment, matching, and stratification did not attenuate this survival benefit. In an instrumental variable analysis, the survival benefit was no longer observed (HR = 1.00, 95% CI = 0.73-1.36).

Conclusions: These results demonstrate the need to exercise caution in using administrative data to infer causal mortality benefits with diagnostic and/or treatment interventions in cancer research.

Keywords: endoscopic ultrasound; instrumental variable; pancreatic cancer; propensity score; selection bias.

Publication types

  • Comparative Study
  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adenocarcinoma / diagnostic imaging*
  • Adenocarcinoma / mortality*
  • Aged
  • Aged, 80 and over
  • Confounding Factors, Epidemiologic
  • Data Interpretation, Statistical
  • Endosonography / statistics & numerical data
  • Female
  • Humans
  • Male
  • Observational Studies as Topic*
  • Pancreatic Neoplasms / diagnostic imaging*
  • Pancreatic Neoplasms / mortality*
  • SEER Program
  • Selection Bias
  • Survival Analysis
  • Treatment Outcome