Enzyme replacement therapy for Anderson-Fabry disease.

BACKGROUND: Anderson-Fabry disease is an X-linked defect of glycosphingolipid metabolism. Progressive renal insufficiency is a major source of morbidity, additional complications result from cardio- and cerebro-vascular involvement. Survival is reduced among affected males and symptomatic female carriers. OBJECTIVES: To evaluate the effectiveness and safety of enzyme replacement therapy compared to other interventions, placebo or no interventions, for treating Anderson-Fabry disease. SEARCH METHODS: We searched 'Clinical Trials' on The Cochrane Library, MEDLINE, EMBASE, LILACS and the Cystic Fibrosis and Genetic Disorders Group's Inborn Errors of Metabolism Trials Register (date of the most recent search: 11 September 2012). The original search was performed in September 2008.Date of the most recent search of the Cystic Fibrosis and Genetic Disorders Group's Inborn Errors of Metabolism Trials Register: 11 September 2012. SELECTION CRITERIA: Randomized controlled trials of agalsidase alfa or beta in participants diagnosed with Anderson-Fabry disease. DATA COLLECTION AND ANALYSIS: Two authors selected relevant trials, assessed methodological quality and extracted data. MAIN RESULTS: Six trials comparing either agalsidase alfa or beta in 223 participants fulfilled the selection criteria.Both trials comparing agalsidase alfa to placebo reported on globotriaosylceramide concentration in plasma and tissue; aggregate results were non-significant. One trial reported pain scores, there was a statistically significant improvement for participants receiving treatment at up to three months, mean difference -2.10 (95% confidence interval (CI) -3.79 to -0.41); at up to five months, mean difference -1.90 (95% CI -3.65 to -0.15); and at up to six months, mean difference -2.00 (95% CI -3.66 to -0.34). There was a significant difference in pain-related quality of life at over five months and up to six months, mean difference -2.10 (95% CI -3.92 to -0.28) but not at other time-points. Neither trial reported deaths.One of the three trials comparing agalsidase beta to placebo reported on globotriaosylceramide concentration in plasma and tissue and showed significant improvement: kidney, mean difference -1.70 (95% CI -2.09 to -1.31); heart, mean difference -0.90 (95% CI -1.18 to -0.62); and composite results (renal, cardiac, and cerebrovascular complications and death), mean difference -4.80 (95% CI -5.45 to -4.15). There was no significant difference between groups for death; no trials reported on pain.Only one trial compared agalsidase alfa to agalsidase beta. There was no significant difference between the groups for any adverse events, risk ratio 0.36 (95% CI 0.08 to 1.59), or any serious adverse events; risk ratio 0.30; 95% CI 0.03 to 2.57). AUTHORS' CONCLUSIONS: Six small, poor quality randomised controlled trials provide no robust evidence for use of either agalsidase alfa and beta to treat Anderson-Fabry disease.

Early light reduction for preventing retinopathy of prematurity in very low birth weight infants.

BACKGROUND: Retinopathy of prematurity (ROP) is a complex condition of the developing retinal blood vessels and is one of the leading causes of preventable childhood blindness. Several risk factors for ROP have been studied over the past 50 years. Among them, general immaturity (low birth weight and low gestational age) and prolonged oxygen therapy have been consistently related to disease onset. However, it is understood that the progression of the disease is multifactorial and may be associated with others risk factors, such as multiple gestation, apnoea, intracranial haemorrhage, anaemia, sepsis, prolonged mechanical ventilation, multiple transfusions and light exposure. Furthermore, the precise role of these individual factors in the development of the disease has not yet been well established. OBJECTIVES: To determine whether the reduction of early environmental light exposure reduces the incidence of retinopathy of prematurity (ROP) or poor ROP outcomes among very low birth weight infants. SEARCH METHODS: We searched the following databases: the Cochrane Neonatal Group Specialised Register, CENTRAL (The Cochrane Library), MEDLINE, EMBASE, CINAHL, HealthSTAR, Science Citation Index Database, CANCERLIT, the Oxford Database of Perinatal Trials and www.clinicaltrials.gov. We also searched previous reviews including cross-references, abstracts, conference and symposia proceedings, and contacted expert informants. This search was updated in October 2012. SELECTION CRITERIA: Randomised or quasi-randomised controlled trials that reduced light exposure to premature infants within the first seven days following birth were considered for this review. We also considered cluster-randomised controlled trials. DATA COLLECTION AND ANALYSIS: Data on clinical outcomes including any acute ROP and poor ROP outcome were extracted by both review authors independently and consensus reached. We conducted data analysis according to the standards of the Cochrane Neonatal Review Group. MAIN RESULTS: Data from four randomised trials with a total of 897 participants failed to show any reduction in acute ROP or poor ROP outcome with the reduction of ambient light to premature infants' retinas. The overall methodological quality of the included studies was about evenly split between those in which the classification was unclear and those in which the studies were categorised as low risk of bias. There was no report on the secondary outcomes considered in this review: quality of life measures; and time of exposure to oxygen. AUTHORS' CONCLUSIONS: The evidence shows that bright light is not the cause of retinopathy of prematurity and that the reduction of exposure of the retinas of premature infants to light has no effect on the incidence of the disease.

Intravenous versus inhalation anaesthesia for one-lung ventilation.

BACKGROUND: This is an update of a Cochrane Review first published in The Cochrane Library, Issue 2, 2008.The technique called one-lung ventilation can confine bleeding or infection to one lung, prevent rupture of a lung cyst or, more commonly, facilitate surgical exposure of the unventilated lung. During one-lung ventilation, anaesthesia is maintained either by delivering an inhalation anaesthetic to the ventilated lung or by infusing an intravenous anaesthetic. It is possible that the method chosen to maintain anaesthesia may affect patient outcomes. Inhalation anaesthetics may impair hypoxic pulmonary vasoconstriction (HPV) and increase intrapulmonary shunt and hypoxaemia. OBJECTIVES: The objective of this review was to evaluate the effectiveness and safety of intravenous versus inhalation anaesthesia for one-lung ventilation. SEARCH METHODS: We searched the Cochrane Central Register of Controlled Trials (CENTRAL); The Cochrane Library (2012, Issue 11); MEDLINE (1966 to November 2012); EMBASE (1980 to November 2012); Literatura Latino-Americana e do Caribe em Ciências da Saúde (LILACS, 1982 to November 2012) and ISI web of Science (1945 to November 2012), reference lists of identified trials and bibliographies of published reviews. We also contacted researchers in the field. No language restrictions were applied. The date of the most recent search was 19 November 2012. The original search was performed in June 2006. SELECTION CRITERIA: We included randomized controlled trials and quasi-randomized controlled trials of intravenous (e.g. propofol) versus inhalation (e.g. isoflurane, sevoflurane, desflurane) anaesthesia for one-lung ventilation in both surgical and intensive care participants. We excluded studies of participants who had only one lung (i.e. pneumonectomy or congenital absence of one lung). DATA COLLECTION AND ANALYSIS: Two review authors independently assessed trial quality and extracted data. We contacted study authors for additional information. MAIN RESULTS: We included in this updated review 20 studies that enrolled 850 participants, all of which assessed surgical participants－no studies investigated one-lung ventilation performed outside the operating theatre. No evidence indicated that the drug used to maintain anaesthesia during one-lung ventilation affected participant outcomes. The methodological quality of the included studies was difficult to assess as it was reported poorly, so the predominant classification of bias was 'unclear'. AUTHORS' CONCLUSIONS: Very little evidence from randomized controlled trials suggests differences in participant outcomes with anaesthesia maintained by intravenous versus inhalational anaesthesia during one-lung ventilation. If researchers believe that the type of drug used to maintain anaesthesia during one-lung ventilation is important, they should design randomized controlled trials with appropriate participant outcomes, rather than report temporary fluctuations in physiological variables.

Interventions to promote the wearing of hearing protection.

BACKGROUND: This is an update of a Cochrane Review first published in The Cochrane Library in Issue 2, 2006 and previously updated in 2009.Noise-induced hearing loss can be prevented by eliminating or lowering noise exposure levels. Where the source of the noise cannot be eliminated, workers have to rely on hearing protection equipment. Several trials have been conducted to study the effectiveness of interventions to influence the wearing of hearing protection. OBJECTIVES: To evaluate the effectiveness of interventions to enhance the wearing of hearing protection among persons regularly exposed to high noise levels. SEARCH METHODS: We searched the Cochrane Ear, Nose and Throat Disorders Group Trials Register; the Cochrane Central Register of Controlled Trials (CENTRAL) (The Cochrane Library 2011, Issue 1); PubMed; EMBASE; CINAHL; Web of Science; BIOSIS Previews; Cambridge Scientific Abstracts; ICTRP and additional sources for published and unpublished trials. The date of the most recent search was 8 April 2011. SELECTION CRITERIA: We included studies if they had a randomised design, if they were among noise exposed (> 80 dB(A)) persons, if they included an intervention to promote the wearing of hearing protection (compared to another intervention or no intervention), and if the outcome measured was the amount of use of hearing protection or a proxy measure thereof. DATA COLLECTION AND ANALYSIS: Two authors selected relevant trials, assessed risk of bias and extracted data. MAIN RESULTS: Seven studies, involving 4670 participants, were included.A computer-based intervention lasting 30 minutes, tailored to the risk of an individual worker, was not found to be more effective than a video providing general information among workers, around 80% of whom already used hearing protection.A four-year school-based hearing loss prevention programme found that the intervention group was twice as likely to wear some kind of hearing protection as the control group that received a baseline hearing test and two additional tests at years two and three.We conducted two meta-analyses for the comparisons 'tailored strategy (the use of communication or other types of interventions that are specific to an individual or a group and aim to change behaviour) versus non-tailored strategy' and 'tailored strategy versus a commercial video on the use of hearing protection' to look at mean percentage use of hearing protective devices (HPDs), that showed improvement in the mean use of HPDs for the tailored group. A meta-analysis of the comparison 'mixed interventions' (classroom instruction, distribution of HPDs, mailings, noise level assessments and audiometric testing) versus control (audiometric testing alone) also showed improvement in self reported use of HPDs when shooting firearms.Tailored education showed an improvement in HPD use of 8.3% versus targeted education (6.1%). AUTHORS' CONCLUSIONS: The evidence found in this review shows that some interventions improve the mean use of hearing protection devices compared to non-intervention. Future trials should have standard outcomes and interventions to allow the combining of results in meta-analysis.

Interventions to promote the wearing of hearing protection.

BACKGROUND: This is an update of a Cochrane Review first published in The Cochrane Library in Issue 2, 2006 and previously updated in 2009.Noise-induced hearing loss can be prevented by eliminating or lowering noise exposure levels. Where the source of the noise cannot be eliminated, workers have to rely on hearing protection equipment. Several trials have been conducted to study the effectiveness of interventions to influence the wearing of hearing protection. OBJECTIVES: To evaluate the effectiveness of interventions to enhance the wearing of hearing protection among persons regularly exposed to high noise levels. SEARCH STRATEGY: We searched the Cochrane Ear, Nose and Throat Disorders Group Trials Register; the Cochrane Central Register of Controlled Trials (CENTRAL) (The Cochrane Library 2011, Issue 1); PubMed; EMBASE; CINAHL; Web of Science; BIOSIS Previews; Cambridge Scientific Abstracts; ICTRP and additional sources for published and unpublished trials. The date of the most recent search was 8 April 2011. SELECTION CRITERIA: We included studies if they had a randomised design, if they were among noise exposed (> 80 dB(A)) persons, if they included an intervention to promote the wearing of hearing protection (compared to another intervention or no intervention), and if the outcome measured was the amount of use of hearing protection or a proxy measure thereof. DATA COLLECTION AND ANALYSIS: Two authors selected relevant trials, assessed risk of bias and extracted data. MAIN RESULTS: Seven studies, involving 4670 participants, were included.A computer-based intervention lasting 30 minutes, tailored to the risk of an individual worker, was not found to be more effective than a video providing general information among workers, around 80% of whom already used hearing protection.A four-year school-based hearing loss prevention programme found that the intervention group was twice as likely to wear some kind of hearing protection as the control group that received a baseline hearing test and two additional tests at years two and three.We conducted two meta-analyses for the comparisons 'tailored strategy (the use of communication or other types of interventions that are specific to an individual or a group and aim to change behaviour) versus non-tailored strategy' and 'tailored strategy versus a commercial video on the use of hearing protection' to look at mean percentage use of hearing protective devices (HPDs), that showed improvement in the mean use of HPDs for the tailored group. A meta-analysis of the comparison 'mixed interventions' (classroom instruction, distribution of HPDs, mailings, noise level assessments and audiometric testing) versus control (audiometric testing alone) also showed improvement in self reported use of HPDs when shooting firearms.Tailored education showed an improvement in HPD use of 8.3% versus targeted education (6.1%). AUTHORS' CONCLUSIONS: The evidence found in this review shows that some interventions improve the mean use of hearing protection devices compared to non-intervention. Future trials should have standard outcomes and interventions to allow the combining of results in meta-analysis.

Enzyme replacement therapy for Anderson-Fabry disease.

BACKGROUND: Anderson-Fabry disease is an X-linked defect of glycosphingolipid metabolism. Progressive renal insufficiency is a major source of morbidity, additional complications result from cardio- and cerebro-vascular involvement. Survival is reduced among affected males and symptomatic female carriers. OBJECTIVES: To evaluate the effectiveness and safety of enzyme replacement therapy compared to other interventions, placebo or no interventions, for treating Anderson-Fabry disease. SEARCH STRATEGY: We searched 'Clinical Trials' on The Cochrane Library, MEDLINE, EMBASE, LILACS and the Cystic Fibrosis and Genetic Disorders Group's Inborn Errors of Metabolism Trials Register (date of the most recent search: 07 April 2010). SELECTION CRITERIA: Randomized controlled trials of agalsidase alfa or beta in participants diagnosed with Anderson-Fabry disease. DATA COLLECTION AND ANALYSIS: Two authors selected relevant trials, assessed methodological quality and extracted data. MAIN RESULTS: Five studies comparing either agalsidase alfa or beta in 187 participants fulfilled the selection criteria.Both trials comparing agalsidase alfa to placebo reported on globotriaosylceramide concentration in plasma and tissue; aggregate results were non-significant. One study reported pain scores, there was a statistically significant improvement for participants receiving treatment at up to three months, mean difference -2.10 (95% confidence interval (CI) -3.79 to -0.41); at up to five months, mean difference -1.90 (95% CI -3.65 to -0.15); and at up to six months, mean difference -2.00 (95% CI -3.66 to -0.34). There was a significant difference in pain-related quality of life at over five months and up to six months, mean difference -2.10 (95% CI -3.92 to -0.28) but not at other time-points. Neither study reported deaths.One of the three trials comparing agalsidase beta to placebo reported on globotriaosylceramide concentration in plasma and tissue and showed significant improvement: kidney, mean difference -1.70 (95% CI -2.09 to -1.31); heart, mean difference -0.90 (95% CI -1.18 to -0.62); and composite results (renal, cardiac, and cerebrovascular complications and death), mean difference -4.80 (95% CI -5.45 to -4.15). There was no significant difference between groups for death; no studies reported on pain. AUTHORS' CONCLUSIONS: Five small, poor quality randomised controlled trials provide no robust evidence for use of either agalsidase alfa and beta to treat Anderson-Fabry disease.

Aerobic exercise training programmes for improving physical and psychosocial health in adults with Down syndrome.

BACKGROUND: Although physical fitness has been suggested to improve physical and psychosocial health for a variety of population profiles, there is a lack of information about the safety and effectiveness of aerobic exercise for adults with Down syndrome. OBJECTIVES: To evaluate the effectiveness and safety of aerobic exercise training programmes for physiological and psychosocial outcomes in adults with Down syndrome. SEARCH STRATEGY: The following electronic databases were searched: The Cochrane Central Register of Controlled Trials (CENTRAL) (2009, Issue 1); MEDLINE (1966 to August 2009); EMBASE (1980 to August 2009); CINAHL (1982 to August 2009); LILACS (1982 to August 2009); PsycINFO (1887 to August 2009); ERIC (1966 to August 2009); Current Controlled Trials (August 2009); and Campbell Collaboration's Social, Psychological, Educational and Criminological Register (C2- SPECTR) (to August 2009). Information about ongoing clinical trials was sought by searching ClinicalTrials.gov (http://clinicaltrials.gov) (accessed August 2009), and the National Research Register (NRR) (2009 Issue 1). SELECTION CRITERIA: Randomised or quasi-randomised controlled trials using supervised aerobic exercise training programmes with behavioral components accepted as co-interventions. DATA COLLECTION AND ANALYSIS: Two reviewers selected relevant trials, assessed methodological quality and extracted data. Where appropriate, data were pooled using meta-analysis with a random-effects model. Positive values favour the intervention group, while negative values favour the control group. MAIN RESULTS: Three studies included in this systematic review used different kinds of aerobic activity: walking/jogging and rowing training and included participants with a broad age range (17 to 65 years). They were conducted in the USA, Portugal and Israel. In the meta-analyses, only maximal treadmill grade was improved after aerobic exercise training programmes (4.26 grades (%) [95% CI 2.06, 6.45]). Other variables relative to work performance that could not be combined in a meta-analysis were also improved in the intervention group (maximal test time P=0.0003), total turns of fan wheel (P=0.02), resistance of ergometer (p=0.003), power knee extension and flexion (p<0.00001), and timed up and go test (p=0.008). Thirty other outcomes measured in this review including, oxidative stress and body composition variables, could not be combined in the meta-analysis. Apart from work performance, trials reported no statistically significant improvements. AUTHORS' CONCLUSIONS: There is insufficient evidence to demonstrate that there is improvement in physical or psychosocial outcomes of aerobic exercise in adults with Down syndrome. Although evidence exists to support improvements in physiological and psychological aspects from strategies using mixed physical activity programmes, well-conducted research examining long-term physical outcomes, adverse effects, psychosocial outcomes and costs is required before informed practice decisions can be made.

Interventions to promote the wearing of hearing protection.

BACKGROUND: This is an update of a Cochrane Review first published in The Cochrane Library in Issue 2, 2006. The original review was republished in Issue 2, 2009 with feedback incorporated.Noise induced hearing loss can be prevented by eliminating or lowering noise exposure levels. Where the source of the noise cannot be eliminated, workers have to rely on hearing protection equipment. Several trials have been conducted to study the effectiveness of interventions to influence the wearing of hearing protection. OBJECTIVES: To evaluate the effectiveness of interventions to enhance the wearing of hearing protection among persons regularly exposed to high noise levels. SEARCH STRATEGY: We searched the Cochrane Ear, Nose and Throat Disorders Group Trials Register; the Cochrane Central Register of Controlled Trials (CENTRAL); PubMed; EMBASE; CINAHL; Web of Science; BIOSIS Previews; Cambridge Scientific Abstracts; mRCT and additional sources for published and unpublished trials. The date of the most recent search was 4 February 2009. SELECTION CRITERIA: We included studies if they had a randomised design, if they were among noise exposed (> 80 dB(A)) persons, if they included an intervention to promote the wearing of hearing protection (compared to another intervention or no intervention), and if the outcome measured was the amount of use of hearing protection or a proxy measure thereof. DATA COLLECTION AND ANALYSIS: Two authors selected relevant trials, assessed methodological quality and extracted data. MAIN RESULTS: Six studies, involving 3917 participants, were included.A computer-based intervention lasting 30 minutes, tailored to the risk of an individual worker, was not found to be more effective than a video providing general information among workers, around 80% of whom already used hearing protection.A four year school-based hearing loss prevention programme found that the intervention group was twice as likely to wear some kind of hearing protection as the control group that received baseline hearing test and two additional tests at years two and three.We conducted two meta-analyses for the comparisons 'tailored strategy (the use of communication or other types of interventions that are specific to an individual or a group and aim to change behaviour) versus non-tailored strategy' and 'tailored strategy versus a commercial video on the use of hearing protection' to look at mean percentage use of hearing protective devices (HPDs), that showed improvement in the mean use of HPDs for the tailored group.Tailored education showed an improvement in HPD use of 8.3% versus targeted education (6.1%). AUTHORS' CONCLUSIONS: The evidence found in this review shows that some interventions improve the mean use of hearing protection devices compared to non-intervention. Future trials should have standard outcomes and interventions to allow the combining of results in meta-analysis.

Prevalence of high frequency hearing loss consistent with noise exposure among people working with sound systems and general population in Brazil: a cross-sectional study.

BACKGROUND: Music is ever present in our daily lives, establishing a link between humans and the arts through the senses and pleasure. Sound technicians are the link between musicians and audiences or consumers. Recently, general concern has arisen regarding occurrences of hearing loss induced by noise from excessively amplified sound-producing activities within leisure and professional environments. Sound technicians' activities expose them to the risk of hearing loss, and consequently put at risk their quality of life, the quality of the musical product and consumers' hearing. The aim of this study was to measure the prevalence of high frequency hearing loss consistent with noise exposure among sound technicians in Brazil and compare this with a control group without occupational noise exposure. METHODS: This was a cross-sectional study comparing 177 participants in two groups: 82 sound technicians and 95 controls (non-sound technicians). A questionnaire on music listening habits and associated complaints was applied, and data were gathered regarding the professionals' numbers of working hours per day and both groups' hearing complaint and presence of tinnitus. The participants' ear canals were visually inspected using an otoscope. Hearing assessments were performed (tonal and speech audiometry) using a portable digital AD 229 E audiometer funded by FAPESP. RESULTS: There was no statistically significant difference between the sound technicians and controls regarding age and gender. Thus, the study sample was homogenous and would be unlikely to lead to bias in the results. A statistically significant difference in hearing loss was observed between the groups: 50% among the sound technicians and 10.5% among the controls. The difference could be addressed to high sound levels. CONCLUSION: The sound technicians presented a higher prevalence of high frequency hearing loss consistent with noise exposure than did the general population, although the possibility of residual confounding due to unmeasured factors such as socioeconomic status cannot be ruled out.

Mapping the Cochrane evidence for decision making in health care.

RATIONALE AND AIM: Over the past 12 years, thousands of authors working with the Cochrane Collaboration around the world have produced systematic reviews to reduce uncertainty in health care decision making. We evaluated the conclusions from Cochrane systematic reviews of randomized controlled trials in terms of their recommendations for clinical practice and research. METHODS: In our cross-sectional study of systematic reviews published in the Cochrane Library, we randomly selected and analysed completed systematic reviews published across all 50 Cochrane Collaborative Review Groups. RESULTS: We analysed 1016 completed systematic reviews. Of these, 44% concluded that the interventions studied were likely to be beneficial, of which 1% recommended no further research and 43% recommended additional research. Also, 7% of the reviews concluded that the interventions were likely to be harmful, of which 2% did not recommend further studies and 5% recommended additional studies. In total, 49% of the reviews reported that the evidence did not support either benefit or harm, of which 1% did not recommend further studies and 48% recommended additional studies. Overall, 96% of the reviews recommended further research. CONCLUSIONS: Cochrane systematic reviews were about evenly split between those in which the authors concluded that at least one of the interventions was beneficial and those in which the evidence neither supported nor refuted the intervention tested. The Cochrane Collaboration needs to include clinical trial protocol summaries with a study design optimized to answer the relevant research questions.

A systematic review of new advances in the management of mucopolysaccharidosis VI (Maroteaux-Lamy syndrome): focus on galsulfase.

INTRODUCTION: Mucopolysaccharidosis type VI (MPS VI, Maroteaux-Lamy syndrome) is an autosomal recessive lysosomal storage disorder, characterized primarily by skeletal dysplasia and joint contracture. It is caused by a deficiency of N-acetylgalactosamine-4-sulfatase (arylsulfatase B), for which a recombinant formulation (galsulfase) is available as replacement therapy. OBJECTIVE: To evaluate the effectiveness and safety of galsulfase compared to placebo or no interventions, for treating MPS VI. We also considered studies evaluating different doses of galsulfase. METHODS: A systematic review of the literature was conducted. A computerized electronic search in MEDLINE, EMBASE, CENTRAL, SciELO, and LILACS was carried on to identify any randomized trials that met our inclusion criteria. RESULTS: Two studies were included in the review. Because the number of studies was small, our analysis probably did not find any statistically significant difference. Long-term follow-up will be required to ascertain full clinical benefit, on both event-free survival and quality of life measures. CONCLUSIONS: There is some evidence to support the use of galsulfase in the treatment of MPS VI; however due to the very low quantity of included studies we could not analyze it in an appropriate way. This review highlights the need for continued research into the use of enzyme replacement therapy for MPS VI.