Intervention Review

Ursodeoxycholic acid for cystic fibrosis-related liver disease

  1. Katharine Cheng1,*,
  2. Deborah Ashby2,
  3. Rosalind L Smyth3

Editorial Group: Cochrane Cystic Fibrosis and Genetic Disorders Group

Published Online: 15 APR 2009

Assessed as up-to-date: 11 AUG 2010

DOI: 10.1002/14651858.CD000222

Database Title

Additional Information

How to Cite

Cheng K, Ashby D, Smyth RL. Ursodeoxycholic acid for cystic fibrosis-related liver disease. Cochrane Database of Systematic Reviews 1999, Issue 3. Art. No.: CD000222. DOI: 10.1002/14651858.CD000222.

Author Information

  1. 1

    Institute of Child Health, University of Liverpool, c/o Cochrane Cystic Fibrosis & Genetic Disorders Review Group, Liverpool, UK

  2. 2

    Imperial College London, Division of Epidemiology, Public Health and Primary Care, London, UK

  3. 3

    Alder Hey Children's NHS Foundation Trust, Institute of Child Health, University of Liverpool, Liverpool, Merseyside, UK

*Katharine Cheng, c/o Cochrane Cystic Fibrosis & Genetic Disorders Review Group, Institute of Child Health, University of Liverpool, Alder Hey Children's NHS Foundation Trust, Eaton Road, Liverpool, L12 2AP, UK. cfgd@liv.ac.uk.

Publication History

  1. Publication Status: New search for studies and content updated (no change to conclusions)
  2. Published Online: 15 APR 2009

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Abstract

  1. Top of page
  2. Abstract
  3. Plain language summary
  4. 摘要

Background

Cystic fibrosis-related liver disease peaks in adolescence with up to 20% of people with cystic fibrosis developing chronic liver disease. Early changes in the liver may ultimately result in end-stage liver disease with people needing transplantation. One therapeutic option currently used is ursodeoxycholic acid.

Objectives

To analyse evidence that ursodeoxycholic acid improves indices of liver function, reduces the risk of developing chronic liver disease and improves outcomes in general in cystic fibrosis.

Search methods

We searched the Cochrane CF and Genetic Disorders Group Trials Register comprising references identified from comprehensive electronic database searches, handsearches of relevant journals and abstract books of conference proceedings. We also contacted drug companies.

Date of the most recent search of the Group's trials register: 02 August 2010.

Selection criteria

Randomised controlled trials of the use of ursodeoxycholic acid for at least three months compared with placebo or no additional treatment in people with cystic fibrosis.

Data collection and analysis

Two authors independently assessed trial eligibility and quality.

Main results

Ten trials have been identified, of which three trials involving 118 participants were included. The complex design used in two trials meant that data could only be analysed for subsets of participants. There was no significant difference in weight change, mean difference -0.90 kg (95% confidence interval -1.94 to 0.14) based on 30 participants from two trials. Improvement in biliary excretion was reported in only one trial and no significant change after treatment was shown. Long-term outcomes such as death or need for liver transplantation were not reported.

Authors' conclusions

There are few trials assessing the effectiveness of ursodeoxycholic acid. There is insufficient evidence to justify its routine use in cystic fibrosis.

 

Plain language summary

  1. Top of page
  2. Abstract
  3. Plain language summary
  4. 摘要

Ursodeoxycholic acid for liver disease related to cystic fibrosis

Problems of bile composition and flow cause liver disease for up to 20% of young people with cystic fibrosis. Ursodeoxycholic acid is a naturally occurring bile acid given to try and prevent liver disease in cystic fibrosis. We searched for trials of this treatment which lasted for at least three months. We were able to include three trials with 118 participants. The complex design of two trials meant data could not be analysed for all the participants. There were no significant differences in any of the outcomes measured. Only one trial measured long-term outcomes such as the need for liver transplant or death. The results were not published per treatment group, only for all participants combined, so we can not draw any firm conclusions from the data. The review did not find enough evidence from trials about the effects of using ursodeoxycholic acid. Current research shows that adverse effects are rare, but there are not enough data about long-term use. As there is no other preventive treatment, more research on ursodeoxycholic acid is needed.

 

摘要

  1. Top of page
  2. Abstract
  3. Plain language summary
  4. 摘要

背景

Ursodeoxycholic acid應用於囊狀纖維化性肝臟病變

囊狀纖維化性肝臟病變的發生，到患者青少年時達到高峰，約20%囊狀纖維化患者將發展為慢性肝臟病變。早期的肝臟病變，進展到晚期將可能需要接受肝臟移植。目前內科的治療選擇之一為使用Ursodeoxycholic acid。

目標

探討Ursodeoxycholic acid應用於改善囊狀纖維化患者的肝臟功能、減少進展為慢性肝臟病變的風險，以及增進囊狀纖維化患者整體的預後情形。

搜尋策略

我們搜尋Cochrane囊狀纖維化及遺傳疾病群組試驗登記資料庫；包括了各廣泛的電子資料庫搜索系統，並實際查詢相關的期刊文獻以及會議議程的摘要手冊。最新進入先天代謝異常疾病試驗登記資料庫的搜索日期為2006年11月。

選擇標準

採隨機對照試驗法，以比較使用Ursodeoxycholic acid治療三個月以上、使用placebo及未接受相關治療的各組囊狀纖維化患者的治療情形。

資料收集與分析

兩位作者各自獨立的評估這些研究的適切性，及其研究方法上的品質，以及接下來資料的選用及分析。

主要結論

發現了10篇相關研究，並納入其中包括118位受試者的3篇研究。此複合的研究採用2項研究數據，意指研究數據只適用於分析個別研究下的受試者。將2項研究中的30位受試者經過加權調整後，並無顯著差異，其加權平均變化數(weighted mean difference, WMD)為 −0.90 公斤(95%的信心區間為 −1.94至0.14)。只有一篇研究提到在治療後改善膽汁分泌，但未有顯著差異。未有研究提及像是死亡率、肝臟移植需求等等長期的預後情形。

作者結論

只有少數研究針對ursodeoxycholic acid的效果進行評估，因此目前還沒有足以作為囊狀纖維化患者常規用藥的證據。

翻譯人

本摘要由臺灣大學附設醫院蕭雅慧翻譯。

此翻譯計畫由臺灣國家衛生研究院(National Health Research Institutes, Taiwan)統籌。

總結

目前對於佔20%以上青少年期後的囊狀纖維化患者，因疾病所致的膽汁合成及引流問題，所造成的肝臟病變，ursodeoxycholic acid還沒有足以作為囊狀纖維化患者常規用藥的證據。 ursodeoxycholic acid來自膽汁酸(bile acid)，給予目的在於試圖避免上述問題的發生。本評論從探討使用ursodeoxycholic acid預防成效的研究試驗中，還未發現足夠的證據。 目前還沒有其他預防的治療方式，更多對於ursodeoxycholic acid的研究仍有其需要。目前研究顯示其副作用相當少見，但對於長期使用結果還沒有足夠的評估資料。

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