Endoscopic variceal ligation as primary prophylaxis for upper GI bleeding in children.

BACKGROUND AND AIMS: Variceal hemorrhage can be a life-threatening adverse event of chronic liver disease. In contrast to the well-described guidelines for the management of portal hypertension (PH) in adults, there is limited evidence about the optimal prophylactic management of variceal bleeding in children. This study was carried out to assess the efficacy of endoscopic variceal ligation (EVL) as primary prophylaxis to prevent upper GI bleeding in children with PH. METHODS: From January 2014 to April 2018, all pediatric patients with PH disease and medium to large esophageal varices or reddish spots, regardless of the grade of the varix, were prospectively included in the protocol of primary prophylaxis with EVL. A second retrospective group of patients was made after reviewing medical records of 32 pediatric patients with PH that presented esophageal varices in the upper endoscopy and had received propranolol as primary prophylaxis. RESULTS: Twenty-four patients (75%) reached varices eradication in the EVL group, with a median of 2 procedures (range, 1-4) before eradication and a median time to eradication of 3.40 months (range, 1.10-13.33). No EVL-related adverse events were observed. Statistically significant differences were observed in the bleeding rate at 3 years between propranolol and EVL groups (6/32 [21.9%] vs 1/32 [3.2%], P < .02). The hazard ratio for bleeding for patients treated with propranolol compared with those treated with EVL was 2.6 (95% confidence interval, 1.53-3.67). CONCLUSIONS: EVL is a safe and effective treatment to prevent upper GI bleeding in pediatric patients with PH. (Clinical trial registration number: NCT03943784.).

Long-term safety and effectiveness of levodopa-carbidopa intestinal gel infusion.

INTRODUCTION: Levodopa-carbidopa intestinal gel (LCIG) infusion has demonstrated to improve motor fluctuations. The aim of this study is to assess the long-term safety and effectiveness of LCIG infusion in advanced Parkinson's disease (PD) patients with motor fluctuations and its effect in nonmotor symptoms. METHODS: Adverse events (AE) and their management, clinical motor, and nonmotor aspects were assessed up to 10 years. Thirty-seven patients were treated with LGIC; in three subsets of patients, specific batteries of tests were used to assess cognitive and behavior assessment for 6 months, quality of sleep for 6 months, and quality of life and caregiver burden for 1 year. RESULTS: There was a high number of AE, but manageable, most of mild and moderate severity. All patients experienced significant improvement in motor fluctuations with a reduction in mean daily off time of 4.87 hr after 3 months (n = 37) to 6.25 hr after 9 years (n = 2). Diskynesias remained stables in 28 patients (75.7%) and improved in 5 patients (13.5%). There was no neuropsychological deterioration, but an improvement in attentional functions, voluntary motor control, and semantic fluency. Quality of sleep did not worsen, and there was an improvement in the subjective parameters, although overnight polysomnography did not change. There was a significant sustained improvement of 37% in PD-Q39 after 3 months and to 1 year, and a significant reduction in caregiver burden of 10% after 3 months. CONCLUSION: LCIG infusion is a safe and efficacious treatment for the control of motor fluctuations, and for improvement or nonworsening of nonmotor aspects, long-term sustained, and feasible for use in routine care.

Adding banding ligation is effective as rescue therapy to prevent variceal rebleeding in haemodynamic non-responders to pharmacological therapy.

BACKGROUND: It is unknown which is the best therapy to treat haemodynamic non-responders to pharmacological therapy after variceal bleeding. AIM: To evaluate the efficacy of adding banding ligation to drugs to prevent variceal rebleeding in haemodynamic non-responders to drugs. METHODS: Fifty-three cirrhotic patients with variceal bleeding underwent a hepatic venous pressure gradient (HVPG) measurement 5 days after the episode. Nadolol and nitrates were then titrated to maximum tolerated doses. A second HVPG was taken 14 days later. Responders (HVPG ≤12 mm Hg or ≥20% decrease from baseline) were maintained on drugs and non-responders had banding ligation added to drugs. RESULTS: Mean follow-up was 28 months. In 5 patients the second HVPG could not be performed because of early rebleeding. The remaining 48 patients were classified as responders (n=24) and non-responders (n=24), who had banding added. No baseline differences were observed between groups. Variceal rebleeding occurred in 12% of the 48 patients whose haemodynamic response was assessed. Responders on drug therapy presented a 16% rebleeding rate, whilst non-responders rescued with banding showed an 8% rebleeding rate. Rebleeding-related mortality was not different between groups. CONCLUSION: In a HVPG-guided strategy, adding banding ligation to drugs is an effective rescue strategy to prevent rebleeding in haemodynamic non-responders to drug therapy.

Effectiveness of combined pharmacologic and ligation therapy in high-risk patients with acute esophageal variceal bleeding.

OBJECTIVES: After an acute variceal bleeding, early decision for aggressive management of patients with worse prognosis may improve outcomes. The effectiveness of currently recommended standard therapy (drugs plus endoscopic ligation) for different risk subgroups and the validity of available risk criteria in clinical practice are unknown. METHODS: We analyzed data of 301 consecutive cirrhotic patients admitted with esophageal variceal bleeding. All patients received antibiotics, somatostatin, and in 263 early endoscopic therapy. A stratified 6-week mortality assessment according to risk (low-risk: Child-Pugh B without active bleeding or Child-Pugh A; high-risk: Child-Pugh B with active bleeding or Child-Pugh C) was performed. A multivariate analysis was conducted to elaborate a new risk classification rule. RESULTS: Among the 162 patients receiving emergency ligation, 14% rebled and 16% died. Standard therapy was very effective in all risk strata, even in high-risk patients, specially if eligible for therapeutic trials (child <14, age ≤75 years, creatinine ≤3.0 mg/dl, no hepatocellular carcinoma, or portal thrombosis), showing this stratum a 10% mortality. In patients receiving ligation, Child-Pugh C patients with baseline creatinine <1.0 mg/dl showed similar mortality to Child-Pugh A or B patients (8% vs. 7%, respectively). Only Child-Pugh C patients with creatinine ≥1.0 were at a significant higher risk (Child-Pugh C: 46% mortality if creatinine ≥1.0 vs. 8% if creatinine <1.0, P=0.006). CONCLUSIONS: The combination of somatostatin, antibiotics, and endoscopic ligation after an acute variceal bleeding in a real-life situation is associated with very low mortality. Child-Pugh C patients with baseline creatinine ≥1.0 mg/dl should be considered high-risk patients in this setting.

Hemodynamic response-guided therapy for prevention of variceal rebleeding: an uncontrolled pilot study.

The clinical usefulness of assessing hemodynamic response to drug therapy in the prophylaxis of variceal rebleeding is unknown. An open-labeled, uncontrolled pilot trial was performed to evaluate the feasibility and efficacy of using the hemodynamic response to pharmacological treatment to guide therapy in this setting. Fifty patients with acute variceal bleeding underwent a hepatic venous pressure gradient (HVPG) measurement 5 days after the episode. Nadolol and nitrates were initiated, and a second HVPG was measured 15 days later. Responder patients (> or =20% decrease in HVPG from baseline) were maintained on drugs, partial responders (> or =10% and <20%) had banding ligation added to the drugs, and nonresponders (<10%) received a transjugular intrahepatic portal-systemic shunt (TIPS). Mean follow-up was 22 months. Eight patients (16%) did not receive the second HVPG, 6 of them because of early variceal rebleeding. Of the other 42 patients, 24 were classified as responders (57%); 10 as partial responders (24%), who had banding added; and 8 as nonresponders (19%), who received a TIPS. Patients with cirrhosis of viral etiology compared to alcoholic cirrhosis tended to present more early rebleedings, less response to drugs and needed more TIPS. Variceal rebleeding occurred in 22% of all patients but only in 12% of patients whose hemodynamic response was assessed. The 3 therapeutic groups were not different. In conclusion, using hemodynamic response to pharmacological treatment to guide therapy in secondary prophylaxis to prevent variceal bleeding is feasible and effectively protects patients from rebleeding. In this context, viral cirrhosis seems to present a worse outcome than alcoholic cirrhosis.