Evidence-Based Medicine
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World J Gastroenterol. Aug 14, 2014; 20(30): 10464-10469
Published online Aug 14, 2014. doi: 10.3748/wjg.v20.i30.10464
Cost-effectiveness analysis of beta-blockers vs endoscopic surveillance in patients with cirrhosis and small varices
Lorenza Di Pascoli, Alessandra Buja, Massimo Bolognesi, Sara Montagnese, Angelo Gatta, Dario Gregori, Carlo Merkel
Lorenza Di Pascoli, Massimo Bolognesi, Sara Montagnese, Angelo Gatta, Carlo Merkel, Hepatic Hemodynamic Laboratory and Clinica Medica 5, Department of Medicine, University of Padua, 35128 Padova, Italy
Alessandra Buja, Dario Gregori, Laboratory of Epidemiological Methodology and Biostatistics, Department of Environmental Medicine and Public Health, University of Padua, 35128 Padova, Italy
Author contributions: Di Pascoli L coordinated the work and participated in the analysis and writing of the manuscript; Buja A and Gregori D performed the cost-effective analysis; Bolognesi M, Montagnese S and Gatta A performed the follow up of the patients and controlled the adeguacy of the data set; Merkel C conceived the study, participated in the analysis and revised the manuscript.
Correspondence to: Carlo Merkel, MD, Hepatic Hemodynamic Laboratory and Clinica Medica 5, Department of Medicine, University of Padua, via Giustiniani 2, 35128 Padova, Italy. carlo.merkel@unipd.it
Telephone: +39-49-8212282 Fax: +39-49-8754179
Received: December 15, 2013
Revised: February 9, 2014
Accepted: April 30, 2014
Published online: August 14, 2014
Processing time: 245 Days and 18.1 Hours
Abstract

AIM: To evaluate the most cost-effectiveness strategy for preventing variceal growth and bleeding in patients with cirrhosis and small esophageal varices.

METHODS: A stochastic analysis based on decision trees was performed to compare the cost-effectiveness of beta-blockers therapy starting from a diagnosis of small varices (Strategy 1) with that of endoscopic surveillance followed by beta-blockers treatment when large varices are demonstrated (Strategy 2), for preventing variceal growth, bleeding and death in patients with cirrhosis and small esophageal varices. The basic nodes of the tree were gastrointestinal endoscopy, inpatient admission and treatment for bleeding, as required. All estimates were performed using a Monte Carlo microsimulation technique, consisting in simulating observations from known probability distributions depicted in the model. Eight-hundred-thousand simulations were performed to obtain the final estimates. All estimates were then subjected to Monte Carlo Probabilistic sensitivity analysis, to assess the impact of the variability of such estimates on the outcome distributions.

RESULTS: The event rate (considered as progression of varices or bleeding or death) in Strategy 1 [24.09% (95%CI: 14.89%-33.29%)] was significantly lower than in Strategy 2 [60.00% (95%CI: 48.91%-71.08%)]. The mean cost (up to the first event) associated with Strategy 1 [823 £ (95%CI: 106 £-2036 £)] was not significantly different from that of Strategy 2 [799 £ (95%CI: 0 £-3498 £)]. The cost-effectiveness ratio with respect to this endpoint was equal to 50.26 £ (95%CI: -504.37 £-604.89 £) per event avoided over the four-year follow-up. When bleeding episodes/deaths in subjects whose varices had grown were included, the mean cost associated with Strategy 1 was 1028 £ (95%CI: 122 £-2581 £), while 1699 £ (95%CI: 171 £-4674 £) in Strategy 2.

CONCLUSION: Beta-blocker therapy turn out to be more effective and less expensive than endoscopic surveillance for primary prophylaxis of bleeding in patients with cirrhosis and small varices.

Keywords: Pharmaco-economical analysis; Cirrhosis; Esophageal varices; Variceal bleeding; Primary prophylaxis

Core tip: In patients with cirrhosis and small esophageal varices no study so far has evaluated the economical consequences of replacing traditional endoscopic surveillance with primary prophylaxis with beta-blockers from this stage. In this study, a decision analysis based on stochastic regression trees and Markov models compared cost-effectiveness of primary prophylaxis with beta-blockers (starting from the diagnosis of small varices) and endoscopic surveillance (and beta-blockers administration when large varices develop). Beta-blocker therapy from the beginning turned out to be more effective and less expensive. This result demonstrate that an early beta-blocker therapy besides being clinically effective, is also also cost-effective.