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Azathioprine for primary biliary cirrhosis

  • Review
  • Intervention




Azathioprine is used for patients with primary biliary cirrhosis, but the therapeutic responses in randomised clinical trials have been conflicting.


To assess the benefits and harms of azathioprine for patients with primary biliary cirrhosis.

Search methods

Randomised clinical trials were identified by searching The Cochrane Hepato-Biliary Group Controlled Trials Register, the Cochrane Central Register of Controlled Trials (CENTRAL) in The Cochrane Library, MEDLINE, EMBASE, Science Citation Index Expanded, The Chinese Biomedical Database, and LILACS, and manual searches of bibliographies to September 2005.

Selection criteria

Randomised clinical trials comparing azathioprine versus placebo, no intervention, or another drug were included irrespective of blinding, language, year of publication, and publication status.

Data collection and analysis

Our primary outcomes were mortality, and mortality or liver transplantation. Dichotomous outcomes were reported as relative risk (RR) with 95% confidence interval (CI). Continuous outcomes were reported as weighted mean difference (WMD) or standardised mean difference (SMD). We examined the intervention effects by random-effects and fixed-effect models.

Main results

We identified two randomised clinical trials with 293 patients. Only one of the trials was regarded as having low bias risk. Azathioprine did not significantly decrease mortality (RR 0.80, 95% CI 0.49 to 1.31, 2 trials). Azathioprine did not improve pruritus at one-year intervention (RR 0.71, 95% CI 0.28 to 1.84, 1 trial), cirrhosis development, or quality of life. Patients given azathioprine experienced significantly more adverse events than patients given no intervention or placebo (RR 2.44, 95% CI 1.14 to 5.20, 2 trials). The common adverse events were rash, severe diarrhoea, and bone marrow depression.

Authors' conclusions

There is no evidence to support the use of azathioprine for patients with primary biliary cirrhosis. Researchers who are interested in performing further randomised clinical trials should be aware of the risks of adverse events.








透過搜索The Cochrane HepatoBiliary Group Controlled Trials Register, CochraneLibrary的Cochrane Central Register of Controlled Trials (CENTRAL)、MEDLINE、EMBASE、Science Citation Index Expanded,The Chinese Biomedical Database、LILACS、手動檢索至2005年9月前的參考書目,尋找隨機臨床試驗。




主要結果是死亡率、死亡率或肝移植。以相對風險(relative risk,RR)及其95% 信賴區間(CI)呈現二分法結果。連續變數結果以加權均數差(WMD)或標準平均差(SMD)報告呈現。我們使用隨機效果和固定效果模型檢驗處置的影響。


找到2項隨機臨床試驗,共293位病人。只有一項試驗被認為偏誤風險較低。Azathioprine沒有顯著降低死亡率 (RR 0.80, 95% CI 0.49 – 1.31, 2項試驗)。Azathioprine治療一年不能改善瘙癢 (RR 0.71, 95% CI 0.28 1.84, 1項試驗)、形成肝硬化或生活品質。 接受Azathioprine的病人明顯比無處置或接受安慰劑的病人有更多不良事件(RR 2.44, 95% CI 1.14 5.20, 2項試驗)。常見的不良事件有皮疹、嚴重腹瀉、骨髓抑制。




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


沒有證據支持Azathioprine用於治療原發性膽汁性肝硬化的病人。原發性膽汁性肝硬化(PBC) 是一種以膽管受損為特徵的肝臟慢性病。每年估計發病率為每100萬人口中有2至24人,而預估盛行率為每100萬人口中19 – 240人。 PBC 主要影響中年婦女。 預測診斷後病人的症狀將持續10年−15年。PBC的病因未明,但是疾病動態呈現像‘自身免疫疾病’。 因此期待免疫抑制藥物能產生顯著作用。本回顧評估所有和PBC有關的免疫抑制劑Azathioprine的臨床資料。本回顧的發現以收納292位病人的2項試驗為依據。Azathioprine與安慰劑或無處置的結果相比。本回顧主要發現Azathioprine對存活率、瘙癢、病程(肝硬化形成)或生活品質沒有影響。接受Azathioprine的病人比接受安慰劑的病人有更多不良事件。

Plain language summary

There is no evidence to support azathioprine for patients with primary biliary cirrhosis

Primary biliary cirrhosis (PBC) is a chronic disease of the liver that is characterised by destruction of bile ducts. Estimates of annual incidence range from 2 to 24 patients per million population, and estimates of prevalence range from 19 to 240 patients per million population. PBC primarily affects middle-aged women. The forecast for the symptomatic patient after diagnosis is between 10 and 15 years. The cause of PBC is unknown, but the dynamics of the disease resemble the group 'autoimmune disease'. Therefore, one might expect a noticeable effect of administering an immune repressing drug (immunosuppressant). This review evaluates all clinical data on the immunosuppressant azathioprine in relation to PBC.

The findings of this review are based on two clinical trials with 293 patients. The drug azathioprine was tested versus placebo or no intervention. The primary findings of the review are that azathioprine has no effect on survival, itching, progression of the disease (cirrhosis development), or quality of life. Patients given azathioprine experienced more adverse events than patients given placebo.