Fifty-three years' experience with randomized clinical trials of emergency portacaval shunt for bleeding esophageal varices in Cirrhosis: 1958-2011.

IMPORTANCE: Emergency treatment of bleeding esophageal varices (BEV) consists mainly of endoscopic and pharmacologic measures, with transjugular intrahepatic portal-systemic shunt (TIPS) performed when bleeding is not controlled. Surgical shunt has been relegated to salvage. At the University of California, San Diego, Medical Center, our group has conducted 10 studies of emergency portacaval shunt (EPCS) during 46 years.

OBJECTIVE: To describe 2 randomized clinical trials (RCTs) conducted from 1988 to 2011 in unselected consecutive patients who received emergency treatment for BEV.

DESIGN, SETTING, AND PARTICIPANTS: In RCT No. 1, a total of 211 unselected consecutive patients with cirrhosis and acute BEV were randomized to emergency endoscopic sclerotherapy (EEST) (n=106) or EPCS (n=105). In RCT No. 2, a total of 154 unselected consecutive patients with cirrhosis and acute BEV were randomized to TIPS (n=78) or EPCS (n=76). Diagnostic workup was completed within 6 hours of initial contact, and primary treatment was initiated within 8 to 12 hours. Regular follow-up for up to 10 years was accomplished in 100% of the patients.

INTERVENTIONS: In RCT No. 1, EEST or EPCS; in RCT No. 2, TIPS or EPCS.

MAIN OUTCOMES AND MEASURES: The 2 groups were compared with regard to survival, control of bleeding, portal-systemic encephalopathy, and direct cost of care. RESULTS Distribution in Child risk classes was almost identical. One-third of patients were in Child class C. Permanent control of bleeding was achieved by EEST in only 20% of the patients and by TIPS in only 22%. In contrast, EPCS permanently controlled bleeding in 97% and 100% of the patients in RCT No. 2 and RCT No. 1, respectively (P<.001). Survival was significantly greater following EPCS than after EEST and TIPS (P<.001). Median survival was more than 10 years following EPCS compared with 1.99 years after TIPS. Occlusion of TIPS was demonstrated in 84% of the patients, 63% of whom underwent TIPS revision, which failed in 80% of the cases. Recurrent portal-systemic encephalopathy developed in 35% of the patients who underwent EEST and 61% of those who received TIPS. In contrast, portal-systemic encephalopathy occurred in 15% of the patients who received EPCS in RCT No. 1 and 21% of those in RCT No. 2. Direct costs of care were 5 to 7 times greater in the EEST ($168100) and TIPS ($264800) groups than in the EPCS ($39000) group (P<.001).

CONCLUSIONS AND RELEVANCE: Emergency portacaval shunt permanently stopped variceal bleeding, almost never became occluded, accomplished 5 times the long-term survival than EEST or TIPS, and was much less costly than EEST or TIPS. The widespread practice of using EPCS mainly as salvage for failure of endoscopic therapy or TIPS is not supported by the definitive results of these long-term RCTs in unselected patients with cirrhosis. TRIAL REGISTRATION clinicaltrials.gov Identifier: NCT00690027 and NCT00734227.