Background
Portal hypertension is defined as an increase in the blood pressure within a system of veins (a type of blood vessel) called the portal venous system, which drains blood from the gastrointestinal tract (gut) and spleen into the liver. Portal hypertension commonly accompanies advanced liver disease and often gives rise to life-threatening complications, including bleeding (haemorrhage) from oesophageal (food pipe) and gastrointestinal varices (enlarged or swollen veins).
Following numerous randomised clinical trials (studies where people are randomly put into one of two or more treatment groups) demonstrating the effectiveness of medicines called non-selective beta-blockers and endoscopic variceal ligation (where an enlarged vein is tied off or ligated by a rubber band using an illuminated optical, slender, and tubular instrument used to look into the body) in decreasing the incidence of variceal bleeding, treatment to prevent first variceal bleeding (called primary prophylaxis) has become the standard of care in adults. However, it is unknown whether these interventions are of benefit or harm when used in children. Because of the size of the endoscopic ligator, sclerotherapy (the endoscopic injection of tissue irritants that cause obliteration of blood vessels) is the only endoscopic prophylactic option currently available in infants weighing less than 10 kg of bodyweight.
Aims
We aimed to conduct a systematic review of randomised clinical trials to assess the benefits and harms of sclerotherapy versus beta-blockers for prevention of oesophageal variceal bleeding in children with chronic liver disease or portal vein thrombosis (blockage or narrowing of the portal vein (the blood vessel that brings blood to the liver from the intestines) by a blood clot). We searched for studies to February 2019.
Key results
We found no randomised clinical trials for inclusion in this review. Accordingly, we lack study results from randomised clinical trials to conclude if sclerotherapy versus beta-blocker may be beneficial or not when administered as primary prophylaxis of oesophageal varices in children and adolescents with a liver disease or portal vein thrombosis. There is a need for well-designed trials that should include important clinical outcomes such as death, failure to control bleeding, and side effects.
Randomised clinical trials assessing the benefits or harms of sclerotherapy versus beta-blockers for primary prophylaxis of oesophageal variceal bleeding in children and adolescents with chronic liver disease or portal vein thrombosis are lacking. Therefore, trials with adequate power and proper design, assessing the benefits and harms of sclerotherapy versus beta-blockers on patient-relevant clinical outcomes such as mortality, failure to control bleeding, and adverse events are needed. Unless such trials are conducted and the results become published, we cannot make any conclusions regarding the benefits or harms of the two interventions.
Portal hypertension commonly accompanies advanced liver disease and often gives rise to life-threatening complications, including bleeding (haemorrhage) from oesophageal and gastrointestinal varices. Variceal bleeding commonly occurs in children with chronic liver disease or portal vein obstruction. Prevention is therefore important. Primary prophylaxis of variceal bleeding in adults is the established standard of care because of the results of numerous randomised clinical trials demonstrating the efficacy of non-selective beta-blockers or endoscopic variceal ligation in decreasing the incidence of variceal bleeding. However, sclerotherapy is the only endoscopic prophylactic option currently available in infants weighing less than 10 kg of bodyweight due to the size of the endoscopic ligator.
To assess the benefits and harms of sclerotherapy versus any type of beta-blocker for primary prophylaxis of oesophageal variceal bleeding in children and adolescents with chronic liver disease or portal vein thrombosis.
We searched The Cochrane Hepato-Biliary Group Controlled Trials Register, CENTRAL, PubMed, Embase Elsevier, LILACS (Bireme), and Science Citation Index Expanded (Web of Science) in February 2019. We scrutinised the reference lists of the retrieved publications and performed a manual search from the main paediatric gastroenterology and hepatology conferences (NASPGHAN and ESPGHAN) abstract books from January 2008 to December 2018. We searched ClinicalTrials.gov, FDA, EMA, and WHO, for ongoing clinical trials. There were no language or document type restrictions.
We planned to include randomised clinical trials irrespective of blinding, language, or publication status, assessing sclerotherapy versus any type of beta-blocker for primary prophylaxis of oesophageal variceal bleeding in children and adolescents with chronic liver disease or portal vein thrombosis. We planned to include quasi-randomised and other observational studies retrieved with the searches for randomised clinical trials for report of harm.
We planned to collect and summarise data from randomised clinical trials as described in our protocol, using standard Cochrane methodologies.
We found no randomised clinical trials assessing sclerotherapy versus beta-blockers for primary prophylaxis of oesophageal variceal bleeding in children and adolescents with chronic liver disease or portal vein thrombosis.