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PubMed2006

Where next after SPACE and EVA-3S: 'the good, the bad and the ugly!'.

SPACE and EVA-3S are the latest to publish outcomes in recently symptomatic patients who were randomised to carotid endarterectomy (CEA) or angioplasty and stenting (CAS). Contrary to expectations, both found that CAS was not 'as good as' CEA, while EVA-3S found CEA to be statistically superior. Not surprisingly, these trials have aroused considerable controversy and many in the pro-CAS lobby have simply dismissed them as being methodologically flawed and unrepresentative of contemporary CAS practice. However, to simply dismiss SPACE and EVA-3S as maverick trials is unacceptable. Unlike the landmark 'symptomatic' and 'asymptomatic' studies, the history of randomised trials comparing CAS and CEA has been characterised by repeated trial suspension (because of excess risk in the CAs cohort) and a systematic failure to achieve randomisation targets (thereby preventing any prospect of a statistically meaningful outcome) amid a milieu of corporate and individual conflicts of interest. SPACE and EVA-3S have certainly informed the dabate but they have not resolved it. Two trials are actively randomising recently symptomatic patients (ICSS in Europe and CREST in North America). Both require our support so that future guidelines are 'evidence based' rather than 'marker driven'.

Angioplasty↗
PubMed2002

Prophylaxis for venous thromboembolic disease in pregnancy and the early postnatal period.

BACKGROUND: Venous thromboembolic disease (TED), although very rare, is a major cause of maternal mortality and morbidity, hence methods of prophylaxis are often used for women at risk. This may include women delivered by caesarean section, those with a personal or family history of TED and women with inherited or acquired thrombophilias (conditions that predispose people to thrombosis). Many methods of prophylaxis carry a risk of side effects, and as the risk of thromboembolic disease is low, it is possible that the benefits of thromboprophylaxis may be outweighed by harm. Current guidelines for clinical practice are based on expert opinion only, rather than high quality evidence from randomised trials. OBJECTIVES: To determine the effects of thromboprophylaxis in association with pregnancy in women who are pregnant or have recently delivered on the incidence of venous thromboembolic disease and side effects. SEARCH STRATEGY: The register of trials maintained by the Cochrane Pregnancy and Childbirth Group, the Cochrane Controlled Trials Register, MEDLINE, EMBASE and bibliographies of reviews. Date of last search: January 2002. SELECTION CRITERIA: Randomised trials comparing one method of thromboprophylaxis with placebo or no treatment, and randomised trials comparing two (or more) methods of thromboprophylaxis. DATA COLLECTION AND ANALYSIS: Data were extracted independently by all reviewers. Discrepancies were resolved by discussion. MAIN RESULTS: Eight trials involving 649 women were included. Four of them compared methods of antenatal prophylaxis; low molecular weight versus unfractionated heparin (two studies), aspirin plus heparin versus aspirin alone (one study), and unfractionated heparin versus no treatment (one study). Four studies assessed postnatal prophylaxis after caesarean section; one compared hydroxyethyl starch with unfractionated heparin, two compared heparin with placebo (one low molecular weight heparin, one unfractionated heparin) and the other compared unfractionated heparin with low molecular weight heparin. It was not possible to assess the effects of any of these interventions on most outcomes, especially rare outcomes such as death, thromboembolic disease and osteoporosis, because of small sample sizes and the small number of trials making the same comparisons. REVIEWER'S CONCLUSIONS: There is insufficient evidence on which to base recommendations for thromboprophylaxis during pregnancy and the early postnatal period. Large scale randomised trials of currently-used interventions should be conducted.

Female↗
PubMed2002

The effectiveness and relative effectiveness of intravenous inotropic drugs acting through the adrenergic pathway in patients with heart failure-a meta-regression analysis.

AIMS: To review systematically the use of intravenous (IV) inotropic agents acting through the adrenergic signalling pathway, compared with placebo or an active agent, in patients with heart failure. METHODS: Studies investigating the use of intravenous inotropes in patients with heart failure published between 1966 and 2000 were identified using MEDLINE, the Cochrane register and Embase databases. Reference lists from relevant papers and reviews were hand searched for further papers. In total, 21 trials, that included 632 patients receiving IV inotropic drugs, placebo or non-treatment control, were identified. Drugs of the following classes were included, the beta-agonists; dobutamine, high-dose (>2.5 microg/kg/min) dopamine, dopexamine and the phosphodiesterase (PDE) inhibitors; amrinone, milrinone, enoximone and toborinone. Sixteen trials (474 patients) contributed data from acute invasive haemodynamic studies of symptomatically severe heart failure. Five trials (158 patients) were based on intermittent inotropic therapy in an outpatient context. RESULTS: With few exceptions, trials of intravenous inotropic agents were small and often failed to report clinically important outcomes. Compared to placebo, intravenous inotropic agents acting through the adrenergic system tended to increase mortality (odds ratio 1.50 (95% CI=0.51 to 3.92) but this did not reach significance and insufficient data were available to determine whether symptoms improved. There appeared to be little difference in the effect of beta-agonists compared to PDE inhibitors on patient outcomes but this could be attributed to the paucity of data. CONCLUSIONS: Intravenous inotropic agents acting through the adrenergic pathway are often used in patients with worsening heart failure to achieve arbitrary haemodynamic targets. Our analyses show that there is very little evidence that such treatment improves symptoms or patient outcomes and may not be safe. This highlights the need for further well designed randomised clinical trials.

Adrenergic beta-Agonists↗
PubMed1998

Heparin management in acute myocardial infarction (AMI).

Antithrombotic agents have been shown to be beneficial in the setting of acute coronary syndromes, and as an adjunct to thrombolysis for acute myocardial infarction (AMI). The optimal type and dosing of antithrombotic drug, however, remains elusive. Heparin, the agent most commonly used, has several limitations, the most important of which may be its inability to inhibit clot-bound thrombin. Newer, direct thrombin inhibitors (such as hirudin) provide potent and predictable thrombin inhibition and are able to inhibit clot-bound thrombin. Both heparin and hirudin can carry a substantial risk of haemorrhage, however, and thrombin activity is likely to rebound after discontinuation of either agent. Further, the relationships of antithrombotic/thrombolytic dosing, measures of anticoagulation (such as APTT), and clinical outcomes are not always clear. Nonetheless, from the data available from large, randomised trials, intravenous heparin should remain a standard adjunct to thrombolytic therapy for AMI.

Fibrinolytic Agents↗
PubMed2002

[Therapeutic advances in 2002 (1)].

The most important drugs registered and/or launched in Belgium during the last year in the various disciplines of internal medicine will be briefly described. The main characteristics of each molecule and its modalities of appropriate use in clinical practice will be emphasized. For all these molecules, both the efficacy and tolerance have been proven in randomised clinical trials according to the rules of Evidence-Based Medicine.

Belgium↗
PubMed2005

[Therapy research in times of patient preference].

The Declaration of Helsinki bears the consequence that in the future reference-controlled clinical trials will increasingly replace placebo-controlled trials. This has enormous implications on research in complementary and alternative medicine: strong preferences for or against the compared therapies will prevent patients to consent to randomisation. In these circumstances a number of alternative designs have been proposed, based on the combination of preference and randomisation, but some of them suffer from severe methodological problems. Others are internally valid but unfeasible in practice and difficult to interpret. Thus, in case of strong patient preferences, we suggest to conduct non-randomised trials but to give special interest to advanced statistical methods (e.g. structural equation models) which must replace simple two-group comparisons.

Controlled Clinical Trials as Topic↗
PubMed2006

[Management of gestational diabetes: what's new in 2005?].

Gestational diabetes is a complication of pregnancy associated with an increase in maternal and perinatal morbidity, as well as an increased risk of subsequent type II diabetes. Diagnosis can be made by the O'Sullivan test, followed by an oral glucose tolerance test. Management of women with gestational diabetes involves multidisciplinary collaboration. The treatment includes diet and, if necessary, insulin therapy. Until recently, there was no evidence that this approach is beneficial. This year, the results of a randomised trial showed a reduction of perinatal mortality and morbidity associated with management of gestational diabetes.

Diabetes, Gestational↗
PubMedDate not supplied

Using economics alongside clinical trials: why we cannot choose the evaluation technique in advance.

When drafting protocols for the use of economic evaluation alongside clinical trials, it is common to have to specify which type of economic evaluation is going to be carried out. Will it be a cost-benefit analysis (CBA), cost-effectiveness analysis (CEA) or a cost-utility analysis (CUA)? It is our contention that prior specification of the appropriate economic technique is not possible, in the majority of cases, until data on effectiveness and cost are actually available. In this letter, we aim to demonstrate the thinking behind our contention and to illustrate this with two case studies; one of a recent randomised trial, the other of a trial currently in progress.

Clinical Protocols↗
PubMed2003

Cooling for newborns with hypoxic ischaemic encephalopathy.

BACKGROUND: Newborn animal and human pilot studies suggest that mild hypothermia following peripartum hypoxia-ischaemia in newborn infants may reduce neurological sequelae, without adverse effects. OBJECTIVES: To determine whether therapeutic hypothermia in encephalopathic asphyxiated newborn infants reduces mortality and long-term neurodevelopmental disability, without clinically important side effects. SEARCH STRATEGY: The standard search strategy of the Neonatal Review Group as outlined in the Cochrane Library (Issue 2, 2003) was used. Randomised controlled trials evaluating therapeutic hypothermia in term newborns with hypoxic ischaemic encephalopathy were identified by searching the Oxford Database of Perinatal Trials, the Cochrane Central Register of Controlled Trials (CENTRAL, The Cochrane Library Issue Issue 2, 2003), MEDLINE (1966 to July 2003), previous reviews including cross-references, abstracts, conferences, symposia proceedings, expert informants and journal hand searching. SELECTION CRITERIA: Randomised controlled trials comparing the use of therapeutic hypothermia with normothermia in encephalopathic newborn infants with evidence of peripartum asphyxia and without recognisable major congenital anomalies were included. The primary outcome measure was death or long-term major neurodevelopmental disability. Other outcomes included adverse effects of cooling and 'early' indicators of neurodevelopmental outcome. DATA COLLECTION AND ANALYSIS: Three reviewers independently selected, assessed the quality of and extracted data from the included studies. Authors were contacted for further information. Meta-analyses were performed using relative risk and risk difference for dichotomous data, and weighted mean difference for continuous data with 95% confidence intervals. MAIN RESULTS: Two randomised controlled trials were included in this review, comprising 50 term infants with moderate/ severe encephalopathy and evidence of intrapartum asphyxia. There was no significant effect of therapeutic hypothermia on the combined outcome of death or major neurodevelopmental disability in survivors followed. No adverse effects of hypothermia on short term medical outcomes or on some 'early' indicators of neurodevelopmental outcome were detected. REVIEWER'S CONCLUSIONS: Although two small randomised controlled trials demonstrated neither evidence of benefit or harm, current evidence is inadequate to assess either safety or efficacy of therapeutic hypothermia in newborn infants with hypoxic ischaemic encephalopathy. Therapeutic hypothermia for encephalopathic asphyxiated newborn infants should be further evaluated in well designed randomised controlled trials.

Asphyxia Neonatorum↗
PubMed2003

Steroid therapy for meconium aspiration syndrome in newborn infants.

BACKGROUND: Meconium aspiration syndrome may cause severe respiratory distress in the newborn infant, with an associated high morbidity and mortality. A chemical pneumonitis is believed to occur secondary to bile, bile acids and pancreatic secretions contained in meconium. It has therefore been hypothesised that corticosteroids may be of benefit in the management of this condition through their anti-inflammatory properties. OBJECTIVES: The objective of this review was to determine whether steroid therapy for meconium aspiration syndrome decreases the morbidity and mortality associated with this condition without adverse effects. SEARCH STRATEGY: Searches were made of PREMEDLINE and MEDLINE from 1966 to April 2003, CINAHL back to 1982, Current Contents back to 1998, The Cochrane Central Register of Controlled Trials (CENTRAL, The Cochrane Library, Issue 1, 2003) and Oxford Database of Perinatal Trials. The search included cross-referencing of previous reviews, and a review of abstracts, conference and symposia proceedings published in Pediatric Research from 1993 to 2003. SELECTION CRITERIA: Randomised controlled trials and quasi-randomised trials comparing steroid treatment to no steroid treatment for neonates with meconium aspiration syndrome were considered for this review. DATA COLLECTION AND ANALYSIS: The methodological quality of each trial was assessed independently by each author. Data were extracted, analysed and results reviewed independently by each author. Meta-analysis was performed with RevMan 4.2, using the fixed effects model. Mean difference (MD) and weighted mean differences (WMD) with 95% confidence intervals in brackets for continuous variables and Relative Risk (RR) with 95% confidence intervals for categorical data were reported. MAIN RESULTS: Three randomised controlled trials were identified. Two trials, by Wu 1999 (50 participants) and Yeh 1977 (35 participants), were included in the review. The trial by Davey 1995, as yet unpublished, was excluded from this review as insufficient information about methodology and results were available. On meta-analysis, there was no significant reduction in mortality [typical RR 0.95 (0.20, 4.58)]. A small but significant increase in duration of oxygen therapy was seen with the use of steroids [WMD 30.0 hours (8.4, 51.6)]. There was no significant difference in duration of hospital stay in the study by Wu 1999 [MD 0.00 days (-3.09, 3.09)]. Duration of mechanical ventilation was reported by Wu 1999 with no significant difference seen [MD -1.10 days (-2.79, 0.59)]. Incidence of air leak was reported by Yeh 1977 with no significant difference detected [RR 0.64 (0.18, 2.26)]. Long-term outcome was not reported in either of the two studies. REVIEWER'S CONCLUSIONS: At present, there is insufficient evidence to assess the effects of steroid therapy in the management of meconium aspiration syndrome. A further large randomised controlled trial assessing potential benefits and harm would be required to determine its role.

Humans↗
PubMed2004

Gluten- and casein-free diets for autistic spectrum disorder.

BACKGROUND: It has been suggested that peptides from gluten and casein may have a role in the origins of autism and that the physiology and psychology of autism might be explained by excessive opioid activity linked to these peptides. Research has reported abnormal levels of peptides in the urine and cerebrospinal fluid of persons with autism. If this is the case, diets free of gluten and /or casein should reduce the symptoms associated with autism. OBJECTIVES: To determine the efficacy of gluten- and/or casein- free diets as an intervention to improve behaviour, cognitive and social functioning in individuals with autism. SEARCH STRATEGY: Electronic searching of abstracts from the Cochrane Library (Issue 3, 2003), PsycINFO (1971- May 2003), EMBASE (1974- May 2003), CINAHL (1982- May 2003), MEDLINE (1986- May 2003), ERIC (1965-2003), LILACS (to 2003) and the specialist register of the Cochrane Complementary Medicine Field (January 2004). Review bibliographies were also examined to identify potential trials. SELECTION CRITERIA: All randomised controlled trials involving programmes which eliminated gluten, casein or both gluten and casein from the diets of individuals diagnosed with autistic spectrum disorder. DATA COLLECTION AND ANALYSIS: Abstracts of studies identified in searches of electronic databases were read and assessed to determine whether they might meet the inclusion criteria. The authors independently selected the relevant studies from the reports identified in this way. As only one trial fitted the inclusion criteria, no meta-analysis is currently possible and data are presented in narrative form. MAIN RESULTS: The one trial included reported results on four outcomes. Unsurprisingly in such a small-scale study, the results for three of these outcomes (cognitive skills, linguistic ability and motor ability) had wide confidence intervals that spanned the line of nil effect. However, the fourth outcome, reduction in autistic traits, reported a significant beneficial treatment effect for the combined gluten- and casein- free diet. REVIEWERS' CONCLUSIONS: This is an important area of investigation and large scale, good quality randomised controlled trials are needed.

Autistic Disorder↗
PubMed2006

Functional endoscopic sinus surgery for chronic rhinosinusitis.

BACKGROUND: Rhinosinusitis is a well-recognised clinical syndrome affecting patients of all ages and gender. FESS has now become a well-established strategy, comprising several techniques, for the treatment of chronic rhinosinusitis refractory to medical treatment. OBJECTIVES: The aim of this review was to assess the effectiveness of functional endoscopic sinus surgery as a treatment for patients with chronic rhinosinusitis. SEARCH STRATEGY: The Cochrane Ear, Nose and Throat Disorders Group Trials Register, the Cochrane Central Register of Controlled Trials (CENTRAL), MEDLINE (1966 to January 2006) and EMBASE (1974 to January 2006) were searched. Reference lists were handsearched and experts in the field contacted to identify further studies. SELECTION CRITERIA: Randomised controlled trials. Inclusion criteria were any of the following criteria singly or in combination: patients with chronic rhinosinusitis diagnosed by a health professional; patients with sinusitis symptoms for more than 12 weeks; endoscopic evidence of sinusitis or radiological evidence of sinusitis. Exclusion criteria were immune suppression; cystic fibrosis; Wegener's disease; previous sinus surgery or sinonasal malignancy. DATA COLLECTION AND ANALYSIS: All studies meeting the inclusion criteria underwent validity assessment and the data were extracted independently by the two authors. Comparisons were:FESS versus medical treatmentFESS versus conventional sinus surgeryFESS + medical treatment versus medical treatmentFESS + medical treatment versus conventional sinus surgery + medical treatment MAIN RESULTS: The three included studies were randomised controlled trials. The evidence available does not demonstrate that FESS, as practiced in the included trials, is superior to medical treatment with or without sinus irrigation in patients with chronic rhinosinusitis. A middle meatal antrostomy fashioned by FESS was also not shown to be superior to an inferior meatal antrostomy formed by traditional sinus surgery techniques, although the small sample size in the study does not exclude a type II error. In one study there was a relapse rate of 2.4% in the FESS and sinus irrigation group compared to 5.6% in the sinus irrigation only group. The relapse rates were not mentioned in the other studies. There were no major complications such as orbital injury or cerebrospinal fluid leak reported in any of the included trials. AUTHORS' CONCLUSIONS: FESS as currently practiced is a safe surgical procedure. The limited evidence available suggests that FESS as practiced in the included trials does not confer additional benefit to that obtained by medical treatment (+/- sinus irrigation) in chronic rhinosinusitis. More randomised controlled trials comparing FESS with medical and other treatments, with long-term follow up, are required.

Chronic Disease↗
PubMed2004

Aripiprazole for schizophrenia.

BACKGROUND: Treatment of people with schizophrenia using older typical antipsychotic drugs such as haloperidol can be problematic. Many fail to respond and more experience disabling adverse effects. Aripiprazole is said to be one of a new generation of atypical antipsychotics with good antipsychotic properties and minimal adverse effects. OBJECTIVES: To evaluate the effects of aripiprazole for people with schizophrenia and schizophrenia-like psychoses. SEARCH STRATEGY: The reviewers searched the Cochrane Schizophrenia Group's Register (May 2003) which is based on regular searches of BIOSIS, CENTRAL, CINAHL, EMBASE, MEDLINE and PsycINFO. References of all identified studies were inspected for further trials. The authors contacted relevant pharmaceutical companies, the FDA and authors of trials for additional information. SELECTION CRITERIA: All clinical randomised trials comparing aripiprazole with placebo, typical or atypical antipsychotic drugs for schizophrenia and schizophrenia-like psychoses. DATA COLLECTION AND ANALYSIS: We extracted data independently. For homogenous dichotomous data we calculated random effects, relative risk (RR), 95% confidence intervals (CI) and, where appropriate, numbers needed to treat (NNT) on an intention-to-treat basis. For continuous data, we calculated weighted mean differences (WMD). MAIN RESULTS: Despite the fact that 4125 people participated in ten randomised aripiprazole studies, we were unable to extract any usable data on death, service outcomes, general functioning, behaviour, engagement with services, satisfaction with treatment; economic outcomes or cognitive functioning. Study attrition was very large and data reporting poor. Compared with placebo, aripiprazole significantly decreased relapse in both the short and medium term (n=300, 1 RCT, RR 0.66 CI 0.53 to 0.81, NNT 5 CI 4 to 8). It also produced better compliance with study protocol (n=1348, 5 RCTs, RR 0.66 CI 0.49 to 0.88, NNT 15 CI 10 to 41). Aripiprazole may decrease prolactin levels below that expected from placebo (n=305, 1 RCT, RR 0.32 CI 0.13 to 0.81, NNT 14 CI 11 to 50). Compared with typical antipsychotics there were no significant benefits for aripiprazole with regards to global state, mental state, quality of life or leaving the study early. Both groups reported similar rates of adverse effects, including akathisia (RR 0.44 CI 0.17 to 1.12) and general extrapyramidal effects (RR 0.53 CI 0.18 to 1.53). Aripiprazole did however cause more insomnia than perphenazine (n=300, 1 RCT, RR 2.23 CI 1.57 to 3.18, NNH 4 CI 3 to 9) and less need for antiparkinson drugs than 10-20mg/day haloperidol (n=1854, 4 RCTs, RR 0.45 CI 0.33 to 0.60, NNT 4 CI 3 to 5). When compared with olanzapine and risperidone, aripiprazole was no better or worse on outcomes of global state and leaving the study early. The rates of adverse effects were also similar, with the exception of less elevation of prolactin (n=301, 1 RCT, RR 0.04 CI 0.02 to 0.08, NNT 2) and less prolongation of the average QTc (30mg/day) (n=200, 1 RCT, WMD -10.0, CI -16.99 to -3.01) compared with risperidone. REVIEWERS' CONCLUSIONS: Aripiprazole may be effective for the treatment of schizophrenia, but it is not much different from typical antipsychotics and atypical antipsychotics with respect to treatment response, efficacy or tolerability. In comparison with typical antipsychotics, aripiprazole may have a higher risk of insomnia, but in comparison to atypical antipsychotics, less risk of raised prolactin and prolongation of the QTc interval. Clearly reported pragmatic short, medium and long term randomised controlled trials should be carried out to determine its position in everyday clinical practice.

Antipsychotic Agents↗
PubMed2005

Hyperbaric oxygen therapy for delayed onset muscle soreness and closed soft tissue injury.

BACKGROUND: Soft tissue injuries (including muscle damage after unaccustomed exercise) are common and are often associated with athletic activity. Hyperbaric oxygen therapy (HBOT) is the therapeutic administration of 100% oxygen at environmental pressures greater than one atmosphere. OBJECTIVES: To assess the benefits and harms of HBOT for treating soft tissue injury, including delayed onset muscle soreness (DOMS). SEARCH STRATEGY: We searched the following in July 2004: CENTRAL, MEDLINE, EMBASE, CINAHL, DORCTIHM and reference lists from relevant articles. Relevant journals were handsearched and researchers in the field contacted. SELECTION CRITERIA: Randomised trials comparing the effect on closed soft tissue injury (including DOMS) of therapeutic regimens which include HBOT with those that exclude HBOT (with or without sham therapy). DATA COLLECTION AND ANALYSIS: Four reviewers independently evaluated study quality and extracted data. Most of the data presented in the review were extracted from graphs in the trial reports. MAIN RESULTS: Nine small trials involving 219 participants were included. Two trials compared HBOT versus sham therapy on acute closed soft tissue injuries (ankle sprain and medial collateral knee ligament injury respectively). The other seven trials examined the effect of HBOT on DOMS following eccentric exercise in unconditioned volunteers. All 32 participants of the ankle sprain trial returned to their normal activities. There were no significant differences between the two groups in time to recovery, functional outcomes, pain, or swelling. There was no difference between the two groups in knee function scores in the second acute injury trial; however, intention-to-treat analysis was not possible for this trial. Pooling of data from the seven DOMS trials showed significantly and consistently higher pain at 48 and 72 hours in the HBOT group (mean difference in pain score at 48 hours [0 to 10 worst pain] 0.88, 95% CI 0.09 to 1.67, P = 0.03) in trials where HBOT was started immediately. There were no differences between the two groups in longer-term pain scores or in any measures of swelling or muscle strength. No trial reported complications of HBOT but careful selection of participants was evident in most trials. AUTHORS' CONCLUSIONS: There was insufficient evidence from comparisons tested within randomised controlled trials to establish the effects of HBOT on ankle sprain or acute knee ligament injury, or on experimentally induced DOMS. There was some evidence that HBOT may increase interim pain in DOMS. Any future use of HBOT for these injuries would need to have been preceded by carefully conducted randomised controlled trials which have demonstrated effectiveness.

Athletic Injuries↗
PubMed2000

URSO-panacea or placebo?

BACKGROUND: Ursodeoxycholic acid (UDCA) is the only approved treatment for primary biliary cirrhosis, but its effect on disease progression is uncertain. The aim of this study was to clarify the efficacy of UDCA in primary biliary cirrhosis. METHODS: A systematic review, including the use of meta-analysis, was done for the randomised and switch-over phases of trials comparing UDCA with placebo, obtained from Medline and Embase databases, and from manual searches derived from review articles and abstracts of major international meetings. All trials had more than a mean of 6 months' follow-up and only included patients with primary biliary cirrhosis (PBC) according to established diagnostic criteria. FINDINGS: 17 relevant articles were identified: 11 randomised controlled trials, including 1272 patients, and six reports of the switch-over phases. UCDA had a favourable effect on liver biochemistry in most of the studies but not on symptoms or the progression of histological stage; two studies did not assess survival, liver transplantation, or complications of liver disease. Meta-analysis showed no difference between UDCA and placebo in the incidence of death (odds ratio 1.21, 95% CI 0.71-2.04), liver related death (0.72, 0.22-2.32), liver transplantation (1.72, 0.78-2.07), death or transplantation (1.26, 0.87-1.82) and in the development of complications of liver disease (1.11, 0.64-1.92). With the primary end-point defined by the authors (a combined end-point in three studies, and death or liver transplantation in the others) an odds ratio of 1.53 (0.97-2.42) was obtained. Assessment of the switch-over phases, during which there was a longer follow-up, did not change the results of the meta-analysis. INTERPRETATION: Published randomised controlled trials of UDCA do not show evidence of therapeutic benefit in PBC and its use as standard therapy needs to be re-examined.

Controlled Clinical Trials as Topic↗