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The Cochrane Database of Systematic... Apr 2016Mucopolysaccharidosis type I can be classified as three clinical sub-types; Hurler syndrome, Hurler-Scheie syndrome and Scheie syndrome, with the scale of severity being... (Review)
Review
BACKGROUND
Mucopolysaccharidosis type I can be classified as three clinical sub-types; Hurler syndrome, Hurler-Scheie syndrome and Scheie syndrome, with the scale of severity being such that Hurler syndrome is the most severe and Scheie syndrome the least severe. It is a rare, autosomal recessive disorder caused by a deficiency of alpha-L-iduronidase. Deficiency of this enzyme results in the accumulation of glycosaminoglycans within the tissues. The clinical manifestations are facial dysmorphism, hepatosplenomegaly, upper airway obstruction, skeletal deformity and cardiomyopathy. If Hurler syndrome is left untreated, death ensues by adolescence. There are more attenuated variants termed Hurler-Scheie or Scheie syndrome, with those affected potentially not presenting until adulthood. Enzyme replacement therapy has been used for a number of years in the treatment of Hurler syndrome, although the current gold standard would be a haemopoietic stem cell transplant in those diagnosed by 2.5 years of age. This is an updated version of the original Cochrane review published in 2013.
OBJECTIVES
To evaluate the effectiveness and safety of treating mucopolysaccharidosis type I with laronidase enzyme replacement therapy as compared to placebo.
SEARCH METHODS
We searched the Cochrane Cystic Fibrosis and Genetic Disorders Group's Inborn Errors of Metabolism Trials Register, MEDLINE via OVID and Embase.Date of most recent search: 05 October 2015.
SELECTION CRITERIA
Randomised and quasi-randomised controlled studies of laronidase enzyme replacement therapy compared to placebo.
DATA COLLECTION AND ANALYSIS
Two authors independently screened the identified studies. The authors then appraised and extracted data.
MAIN RESULTS
One study of 45 patients met the inclusion criteria. This double-blind, placebo-controlled, randomised, multinational study looked at laronidase at a dose of 0.58 mg/kg/week versus placebo in patients with mucopolysaccharidosis type I. All primary outcomes listed in this review were studied in this study. The laronidase group achieved statistically significant improvements in per cent predicted forced vital capacity compared to placebo, MD 5.60 (95% confidence intervals 1.24 to 9.96) and in the six-minute-walk test (mean improvement of 38.1 metres in the laronidase group; P = 0.039, when using a prospectively planned analysis of covariance). The levels of urinary glycoaminoglycans were also significantly reduced. In addition, there were improvements in hepatomegaly, sleep apnoea and hypopnoea. Laronidase antibodies were detected in nearly all patients in the treatment group with no apparent clinical effect and titres were reducing by the end of the study. Infusion-related adverse reactions occurred in both groups but all were mild and none necessitated medical intervention or infusion cessation.
AUTHORS' CONCLUSIONS
The current evidence demonstrates that laronidase is effective when compared to placebo in the treatment of mucopolysaccharidosis type I. The included study was comprehensive and of good quality, although there were few participants. The study included all of the key outcome measures we wished to look at. It demonstrated that laronidase is efficacious in relation to reducing biochemical parameters (reduced urine glycosaminoglycan excretion) and improved functional capacity as assessed by forced vital capacity and the six-minute-walk test. In addition glycosaminoglycan storage was reduced as ascertained by a reduction in liver volume. Laronidase appeared to be safe and, while antibodies were generated, these titres were reducing by the end of the study. More studies are required to determine long-term effectiveness and safety and to assess the impact upon quality of life. Enzyme replacement therapy with laronidase can be used pre- and peri-haemopoietic stem cell transplant, which is now the gold standard treatment in those patients diagnosed under 2.5 years of age.
Topics: Adolescent; Adult; Antibodies; Child; Enzyme Replacement Therapy; Female; Humans; Iduronidase; Male; Mucopolysaccharidosis I; Randomized Controlled Trials as Topic; Rare Diseases; Severity of Illness Index; Young Adult
PubMed: 27033167
DOI: 10.1002/14651858.CD009354.pub4 -
The Cochrane Database of Systematic... Mar 2016Levels of physical fitness are low after stroke. It is unknown whether improving physical fitness after stroke reduces disability. (Meta-Analysis)
Meta-Analysis Review
BACKGROUND
Levels of physical fitness are low after stroke. It is unknown whether improving physical fitness after stroke reduces disability.
OBJECTIVES
To determine whether fitness training after stroke reduces death, dependence, and disability and to assess the effects of training with regard to adverse events, risk factors, physical fitness, mobility, physical function, quality of life, mood, and cognitive function. Interventions to improve cognitive function have attracted increased attention after being identified as the highest rated research priority for life after stroke. Therefore we have added this class of outcomes to this updated review.
SEARCH METHODS
We searched the Cochrane Stroke Group Trials Register (last searched February 2015), the Cochrane Central Register of Controlled Trials (CENTRAL 2015, Issue 1: searched February 2015), MEDLINE (1966 to February 2015), EMBASE (1980 to February 2015), CINAHL (1982 to February 2015), SPORTDiscus (1949 to February 2015), and five additional databases (February 2015). We also searched ongoing trials registers, handsearched relevant journals and conference proceedings, screened reference lists, and contacted experts in the field.
SELECTION CRITERIA
Randomised trials comparing either cardiorespiratory training or resistance training, or both (mixed training), with usual care, no intervention, or a non-exercise intervention in stroke survivors.
DATA COLLECTION AND ANALYSIS
Two review authors independently selected trials, assessed quality and risk of bias, and extracted data. We analysed data using random-effects meta-analyses. Diverse outcome measures limited the intended analyses.
MAIN RESULTS
We included 58 trials, involving 2797 participants, which comprised cardiorespiratory interventions (28 trials, 1408 participants), resistance interventions (13 trials, 432 participants), and mixed training interventions (17 trials, 957 participants). Thirteen deaths occurred before the end of the intervention and a further nine before the end of follow-up. No dependence data were reported. Diverse outcome measures restricted pooling of data. Global indices of disability show moderate improvement after cardiorespiratory training (standardised mean difference (SMD) 0.52, 95% confidence interval (CI) 0.19 to 0.84; P value = 0.002) and by a small amount after mixed training (SMD 0.26, 95% CI 0.04 to 0.49; P value = 0.02); benefits at follow-up (i.e. after training had stopped) were unclear. There were too few data to assess the effects of resistance training.Cardiorespiratory training involving walking improved maximum walking speed (mean difference (MD) 6.71 metres per minute, 95% CI 2.73 to 10.69), preferred gait speed (MD 4.28 metres per minute, 95% CI 1.71 to 6.84), and walking capacity (MD 30.29 metres in six minutes, 95% CI 16.19 to 44.39) at the end of the intervention. Mixed training, involving walking, increased preferred walking speed (MD 4.54 metres per minute, 95% CI 0.95 to 8.14), and walking capacity (MD 41.60 metres per six minutes, 95% CI 25.25 to 57.95). Balance scores improved slightly after mixed training (SMD 0.27, 95% CI 0.07 to 0.47). Some mobility benefits also persisted at the end of follow-up. The variability, quality of the included trials, and lack of data prevents conclusions about other outcomes and limits generalisability of the observed results.
AUTHORS' CONCLUSIONS
Cardiorespiratory training and, to a lesser extent, mixed training reduce disability during or after usual stroke care; this could be mediated by improved mobility and balance. There is sufficient evidence to incorporate cardiorespiratory and mixed training, involving walking, within post-stroke rehabilitation programmes to improve the speed and tolerance of walking; some improvement in balance could also occur. There is insufficient evidence to support the use of resistance training. The effects of training on death and dependence after stroke are still unclear but these outcomes are rarely observed in physical fitness training trials. Cognitive function is under-investigated despite being a key outcome of interest for patients. Further well-designed randomised trials are needed to determine the optimal exercise prescription and identify long-term benefits.
Topics: Activities of Daily Living; Exercise Therapy; Humans; Physical Fitness; Randomized Controlled Trials as Topic; Resistance Training; Stroke; Stroke Rehabilitation; Walking
PubMed: 27010219
DOI: 10.1002/14651858.CD003316.pub6 -
The Cochrane Database of Systematic... Mar 2016Mucopolysaccharidosis type VI or Maroteaux-Lamy syndrome is a rare genetic disorder caused by the deficiency of arylsulphatase B. The resultant accumulation of dermatan... (Review)
Review
BACKGROUND
Mucopolysaccharidosis type VI or Maroteaux-Lamy syndrome is a rare genetic disorder caused by the deficiency of arylsulphatase B. The resultant accumulation of dermatan sulphate causes lysosomal damage.The clinical symptoms are related to skeletal dysplasia (i.e. short stature and degenerative joint disease). Other manifestations include cardiac disease, impaired pulmonary function, ophthalmological complications, hepatosplenomegaly, sinusitis, otitis, hearing loss and sleep apnea. Intellectual impairment is generally absent. Clinical manifestation is typically by two or three years of age; however, slowly progressive cases may not present until adulthood.Enzyme replacement therapy with galsulfase is considered a new approach for treating mucopolysaccharidosis type VI.
OBJECTIVES
To evaluate the effectiveness and safety of treating mucopolysaccharidosis VI by enzyme replacement therapy with galsulfase compared to other interventions, placebo or no intervention.
SEARCH METHODS
Eletronic searches were performed on the Cystic Fibrosis and Genetic Disorders Group's Inborn Errors of Metabolism Trials Register, in CENTRAL, MEDLINE, LILACS, the Journal of Inherited Metabolic Disease and ClinicalTrials.gov. Date of the last search of the Cystic Fibrosis and Genetic Disorders Group's Inborn Errors of Metabolism Trials Register: 05 February 2016.
SELECTION CRITERIA
Randomized and quasi-randomized controlled clinical studies of enzyme replacement therapy with galsulfase compared to other interventions or placebo.
DATA COLLECTION AND ANALYSIS
Two authors independently screened the studies, assessed the risk of bias and extracted data.
MAIN RESULTS
One study was included involving 39 participants who received either enzyme replacement therapy with galsulfase (recombinant human arylsulphatase B) or placebo. This small study was considered to be of overall unclear quality, since the authors did not report how both the allocation generation and concealment were performed.The key finding at 24 weeks in the 12-minute walk test was a statistically significant mean difference of 92.00 meters between the two groups in favour of the galsulfase group (95% confidence interval 11.00 to 172.00). While week 24 results for the three-minute stair climb demonstrated some improvement in the treatment group as compared to the placebo group, this was not significant, mean difference 5.70 (95% confidence interval -0.10 to 11.50).A significant decrease in the urinary glycosaminoglycan levels was observed in favour of the galsulfase group at 24 weeks, mean difference -227.00 (95% confidence interval -264.00 to -190.00).In general, the dose of galsulfase was well tolerated and there were no significant differences in relation to adverse events. These events include drug-related adverse events, serious and severe adverse events, those during infusion, drug-related adverse events during infusion, and deaths. More infusion-related reactions were observed in the galsulfase group and were managed with interruption or slowing of infusion rate or administration of antihistamines or corticosteroids drugs. No deaths occurred during the study.
AUTHORS' CONCLUSIONS
The results of one small study (based on 24-week randomised phase of the study and prior to the open-label extension) demonstrated that galsulfase is more effective than placebo in people with MPS VI, with significant improvements in the 12-minute walk test and a reduction in urinary glycosaminoglycans.There were no significant changes in cardiac or pulmonary functions, liver or spleen volume, overnight apnea-hypopnea, height and weight, quality of life and adverse effects.Further studies are needed to obtain more information on the long-term effectiveness and safety of enzyme replacement therapy with galsulfase.
Topics: Enzyme Replacement Therapy; Glycosaminoglycans; Humans; Mucopolysaccharidosis VI; N-Acetylgalactosamine-4-Sulfatase; Randomized Controlled Trials as Topic; Recombinant Proteins
PubMed: 26943923
DOI: 10.1002/14651858.CD009806.pub2 -
European Journal of Physical and... Oct 2016Treadmill training is used in rehabilitation might improve gait parameters of patients with Parkinson Disease. Aim of this study was to assess the effectiveness of... (Review)
Review
INTRODUCTION
Treadmill training is used in rehabilitation might improve gait parameters of patients with Parkinson Disease. Aim of this study was to assess the effectiveness of treadmill training in improving the gait of patients with Parkinson Disease and the acceptability and safety of this type of therapy.
EVIDENCE ACQUISITION
We searched the Cochrane Movement Disorders Group Specialized Register (last searched September 2014), Cochrane Central Register of Controlled Trials (The Cochrane Library 2014, Issue 10), MEDLINE (1950 to September 2014), and EMBASE (1980 to September 2014). We also handsearched relevant conference proceedings, searched trials and research registers, and checked reference lists (last searched September 2014). We contacted trialists, experts and researchers in the field and manufacturers of commercial devices. We included all randomized controlled trials comparing treadmill training with no treadmill training in patients with Parkinson Disease. Two review authors independently selected trials for inclusion, assessed trial quality and extracted data.
EVIDENCE SYNTHESIS
Treadmill training improved gait speed (MD=0.09 m/s; 95% confidence interval (CI) 0.03 to 0.14; P=0.001; I2=24%; moderate quality of evidence), stride length (MD=0.05 meters; 95% CI 0.01 to 0.09; P=0.01; I2=0%; low quality of evidence), but walking distance (MD=48.9 meters; 95% CI -1.32 to 99.14; P=0.06; I2=91%; very low quality of evidence) and cadence did not improve (MD=2.16 steps/minute; 95% CI -0.13 to 4.46; P=0.07; I2=28%; low quality of evidence) at the end of study. Treadmill training did not increase the risk of patients dropping out from intervention (RD=-0.02; 95% CI -0.06 to 0.02; P=0.32; I2=13%; moderate quality of evidence) and adverse events were not reported.
CONCLUSIONS
This systematic review provides evidence from eighteen trials with moderate to low risk of bias that the use of treadmill training in patients with PD may improve clinically relevant gait parameters such as gait speed and stride length. This apparent benefit for patients is, however, not supported by all secondary variables (e.g. cadence and walking distance). The results must be interpreted with caution because the results were heterogeneous and it is not known how long improvements last and differences between the trials in terms of patient characteristics, duration and amount of training and types of treadmill training exists.
Topics: Aged; Disability Evaluation; Evidence-Based Medicine; Exercise Test; Exercise Therapy; Female; Gait; Gait Disorders, Neurologic; Humans; Male; Middle Aged; Parkinson Disease; Prognosis; Randomized Controlled Trials as Topic; Severity of Illness Index; Treatment Outcome; Walking Speed
PubMed: 26940123
DOI: No ID Found -
Health Technology Assessment... Feb 2016In recent years, meters for continuous monitoring of interstitial fluid glucose have been introduced to help people with type 1 diabetes mellitus (T1DM) to achieve... (Review)
Review
Integrated sensor-augmented pump therapy systems [the MiniMed® Paradigm™ Veo system and the Vibe™ and G4® PLATINUM CGM (continuous glucose monitoring) system] for managing blood glucose levels in type 1 diabetes: a systematic review and economic evaluation.
BACKGROUND
In recent years, meters for continuous monitoring of interstitial fluid glucose have been introduced to help people with type 1 diabetes mellitus (T1DM) to achieve better control of their disease.
OBJECTIVE
The objective of this project was to summarise the evidence on the clinical effectiveness and cost-effectiveness of the MiniMed(®) Paradigm™ Veo system (Medtronic Inc., Northridge, CA, USA) and the Vibe™ (Animas(®) Corporation, West Chester, PA, USA) and G4(®) PLATINUM CGM (continuous glucose monitoring) system (Dexcom Inc., San Diego, CA, USA) in comparison with multiple daily insulin injections (MDIs) or continuous subcutaneous insulin infusion (CSII), both with either self-monitoring of blood glucose (SMBG) or CGM, for the management of T1DM in adults and children.
DATA SOURCES
A systematic review was conducted in accordance with the principles of the Centre for Reviews and Dissemination guidance and the National Institute for Health and Care Excellence Diagnostic Assessment Programme manual. We searched 14 databases, three trial registries and two conference proceedings from study inception up to September 2014. In addition, reference lists of relevant systematic reviews were checked. In the absence of randomised controlled trials directly comparing Veo or an integrated CSII + CGM system, such as Vibe, with comparator interventions, indirect treatment comparisons were performed if possible.
METHODS
A commercially available cost-effectiveness model, the IMS Centre for Outcomes Research and Effectiveness diabetes model version 8.5 (IMS Health, Danbury, CT, USA), was used for this assessment. This model is an internet-based, interactive simulation model that predicts the long-term health outcomes and costs associated with the management of T1DM and type 2 diabetes. The model consists of 15 submodels designed to simulate diabetes-related complications, non-specific mortality and costs over time. As the model simulates individual patients over time, it updates risk factors and complications to account for disease progression.
RESULTS
Fifty-four publications resulting from 19 studies were included in the review. Overall, the evidence suggests that the Veo system reduces hypoglycaemic events more than other treatments, without any differences in other outcomes, including glycated haemoglobin (HbA1c) levels. We also found significant results in favour of the integrated CSII + CGM system over MDIs with SMBG with regard to HbA1c levels and quality of life. However, the evidence base was poor. The quality of the included studies was generally low, often with only one study comparing treatments in a specific population at a specific follow-up time. In particular, there was only one study comparing Veo with an integrated CSII + CGM system and only one study comparing Veo with a CSII + SMBG system in a mixed population. Cost-effectiveness analyses indicated that MDI + SMBG is the option most likely to be cost-effective, given the current threshold of £30,000 per quality-adjusted life-year gained, whereas integrated CSII + CGM systems and Veo are dominated and extendedly dominated, respectively, by stand-alone, non-integrated CSII with CGM. Scenario analyses did not alter these conclusions. No cost-effectiveness modelling was conducted for children or pregnant women.
CONCLUSIONS
The Veo system does appear to be better than the other systems considered at reducing hypoglycaemic events. However, in adults, it is unlikely to be cost-effective. Integrated systems are also generally unlikely to be cost-effective given that stand-alone systems are cheaper and, possibly, no less effective. However, evidence in this regard is generally lacking, in particular for children. Future trials in specific child, adolescent and adult populations should include longer term follow-up and ratings on the European Quality of Life-5 Dimensions scale at various time points with a view to informing improved cost-effectiveness modelling.
STUDY REGISTRATION
PROSPERO Registration Number CRD42014013764.
FUNDING
The National Institute for Health Research Health Technology Assessment programme.
Topics: Blood Glucose; Blood Glucose Self-Monitoring; Cost-Benefit Analysis; Diabetes Complications; Diabetes Mellitus, Type 1; Glycated Hemoglobin; Humans; Hypoglycemic Agents; Insulin; Quality of Life; Technology Assessment, Biomedical
PubMed: 26933827
DOI: 10.3310/hta20170 -
Archives of Physical Medicine and... Apr 2016To systematically review the evidence to determine energy expenditure (EE) in volume of oxygen uptake (V̇O2) (mL/kg/min) and energy cost in oxygen uptake per meter... (Review)
Review
OBJECTIVES
To systematically review the evidence to determine energy expenditure (EE) in volume of oxygen uptake (V̇O2) (mL/kg/min) and energy cost in oxygen uptake per meter walked (V̇O2/walking speed; mL/kg/m) during walking poststroke and how it compares with healthy controls; and to determine how applicable current exercise prescription guidelines are to stroke survivors.
DATA SOURCES
Cochrane Central Register of Controlled Trials, MEDLINE, Embase, and CINAHL were searched on October 9, 2014, using search terms related to stroke and EE. Additionally, we screened reference lists of eligible studies.
STUDY SELECTION
Two independent reviewers screened titles and abstracts of 2115 identified references. After screening the full text of 144 potentially eligible studies, we included 29 studies (stroke survivors: n=501, healthy controls: n=123), including participants with confirmed stroke and a measure of V̇O2 during walking using breath-by-breath analysis. Studies with (9 studies) and without (20 studies) a healthy control group were included.
DATA EXTRACTION
Two reviewers independently extracted data using a standard template, including patient characteristics, outcome data, and study methods.
DATA SYNTHESIS
Mean age of stroke survivors was 57 years (range, 40-67y). Poststroke EE was highly variable across studies and could not be pooled because of high heterogeneity. EE during steady-state overground walking at matched speeds was significantly higher in stroke survivors than healthy controls (mean difference in V̇O2, 4.06 mL/kg/min; 95% confidence interval [CI], 2.21-5.91; 1 study; n=26); there was no significant group difference at self-selected speeds. Energy cost during steady-state overground walking was higher in stroke survivors at both self-selected (mean difference, .47 mL/kg/m; 95% CI, .29-.66; 2 studies; n=38) and matched speeds compared with healthy controls (mean difference, .27 mL/kg/m; 95% CI, .03-.51; 1 study; n=26).
CONCLUSIONS
Stroke survivors expend more energy during walking than healthy controls. Low-intensity exercise as described in guidelines might be at a moderate intensity level for stroke survivors; there is a need for stroke-specific exercise guidelines.
Topics: Adult; Aged; Case-Control Studies; Energy Metabolism; Exercise Therapy; Female; Healthy Volunteers; Humans; Male; Middle Aged; Oxygen Consumption; Stroke; Stroke Rehabilitation; Survivors; Walking
PubMed: 26686877
DOI: 10.1016/j.apmr.2015.11.007 -
American Journal of Physical Medicine &... Mar 2016The purpose of this systematic review was to examine studies that examined the effectiveness of aerobic exercise interventions on mobility in long-term stroke survivors. (Review)
Review
OBJECTIVE
The purpose of this systematic review was to examine studies that examined the effectiveness of aerobic exercise interventions on mobility in long-term stroke survivors.
DESIGN
The authors searched electronic databases for randomized control trials between January 1995 and December 2014 investigating aerobic exercise interventions and mobility in stroke survivors after the subacute phase (>6 mos). Mobility was measured using objective functional fitness tests: 6-minute walk, 10-meter walk, and up-n-go.
RESULTS
Nine randomized control trials that compared aerobic exercise with a control group among stroke survivors (mean age, 56.95-68 yrs) were identified. Aerobic interventions lasted between 2 and 6 mos and primarily involved walking. Using the Comprehensive Meta-analysis software, it was found that two of the three mobility outcomes showed small to moderate effect sizes favoring the aerobic exercise group: 6-minute walk (g = 0.366, P < 0.001) and 10-meter walk (g = 0.411, P = 0.002), while the up-n-go test was not significant (g = -0.150, P = 0.330).
CONCLUSION
These findings demonstrate that stroke survivors may continue to benefit from aerobic exercise after the subacute phase. Future research needs to examine the precise dose and recommendation for aerobic exercise, test other exercise modalities, and use larger samples to thoroughly determine long-term exercise effects on mobility in this population.
Topics: Exercise; Humans; Stroke; Stroke Rehabilitation; Walking
PubMed: 26544857
DOI: 10.1097/PHM.0000000000000416 -
Circulation. Cardiovascular Quality and... Oct 2015About half of survivors with stroke experience severe and significant long-term disability. The purpose of this article is to review the state of the science and to make... (Review)
Review
BACKGROUND
About half of survivors with stroke experience severe and significant long-term disability. The purpose of this article is to review the state of the science and to make recommendations for measuring patient-centric outcomes in interventions for motor improvement in the chronic stroke phase.
METHODS AND RESULTS
A 9-member expert panel reviewed evidence to identify measures of upper and lower extremity function used to date as outcomes in trials with patients who experienced a stroke ≥6 months before assessment. Outcome measures were screened using StrokEDGE consensus panel recommendations, and evaluated for availability of a published minimal clinically important difference. Measures meeting these criteria were further evaluated with regard to their level of measurement, psychometric properties, and ability of minimal clinically important difference to capture gains associated with improved function and clinical relevance to patients, to arrive at recommendations. A systematic literature review yielded 115 clinical trials of upper and lower extremity function in chronic stroke that used a total of 34 outcome measures. Seven of these had published minimal clinically important differences and were recommended or highly recommended by StrokEDGE. Those are the Fugl-Meyer Upper Extremity and Lower Extremity scales, Wolf Motor Function Test, Action Research Arm Test, Ten-Meter and Six-Minute Walk Tests, and the Stroke Impact Scale. All had evidence for their psychometric performance, although the strength of evidence for validity varied, especially in populations with chronic stroke Fugl-Meyer Upper and Lower Extremity scales showing the strongest evidence for validity.
CONCLUSIONS
The panel recommends that the Fugl-Meyer Upper and Lower Extremity scales be used as primary outcomes in intervention trials targeting motor function in populations with chronic stroke. The other 6 measures are recommended as secondary outcomes.
Topics: Chronic Disease; Clinical Trials as Topic; Disability Evaluation; Early Medical Intervention; Expert Testimony; Humans; Lower Extremity; Outcome Assessment, Health Care; Recovery of Function; Research Design; Stroke; Upper Extremity
PubMed: 26515205
DOI: 10.1161/CIRCOUTCOMES.115.002098 -
Archives of Physical Medicine and... Dec 2015To evaluate the characteristics and available evidence on the measurement properties of the gait speed (GS) test, timed Up and Go test (TUG), sit-to-stand (STS) test,... (Review)
Review
OBJECTIVES
To evaluate the characteristics and available evidence on the measurement properties of the gait speed (GS) test, timed Up and Go test (TUG), sit-to-stand (STS) test, and step test; to investigate their relation with clinical outcomes in chronic obstructive pulmonary disease (COPD); and to provide recommendations for clinical practice and future research.
DATA SOURCES
Studies were systematically identified from a literature search using PubMed, PEDro, CINAHL, and Cochrane Library databases and the reference lists of the included articles.
STUDY SELECTION
Studies including ≥1 of these 4 lower limb functional tests (GS test, TUG, STS test, and step test) as an outcome in patients with COPD were selected. No limits were applied for language and study design.
DATA EXTRACTION
Two researchers independently performed data extraction and, by using the COnsensus-based standards for the Selection of health status measurement INstruments, assessed the quality of those studies that described measurement properties.
DATA SYNTHESIS
Forty-two articles met the selection criteria. GS test, STS test, and step test are valid, reproducible, and responsive tests, especially the 4-meter GS, 5-repetition STS test, and 6-minute step test (6MST). The TUG is reliable; however, studies on other measurement properties of this test are lacking. Outcomes of these tests are correlated with mortality, physical activity in daily life, exercise capacity, dyspnea, and quality of life.
CONCLUSIONS
Simple and functional lower limb tests provide information about important clinical outcomes in patients with COPD. The 4-meter GS, 5-repetition STS test, and 6MST are tests with well-established psychometric properties, whereas the properties of the TUG need to be studied further.
Topics: Activities of Daily Living; Dyspnea; Exercise; Exercise Tolerance; Gait; Health Status; Humans; Lower Extremity; Physical Therapy Modalities; Pulmonary Disease, Chronic Obstructive; Quality of Life; Reproducibility of Results
PubMed: 26254951
DOI: 10.1016/j.apmr.2015.07.017 -
British Journal of Haematology Aug 2015Many studies report estimated pulmonary artery systolic pressure (ePASP) in patients with sickle cell disease (SCD) screened by echocardiography. To better understand... (Meta-Analysis)
Meta-Analysis Review
Many studies report estimated pulmonary artery systolic pressure (ePASP) in patients with sickle cell disease (SCD) screened by echocardiography. To better understand the prevalence and outcomes of elevated ePASP in clinically stable SCD patients, we conducted a random-effects meta-analysis. A total of 45 studies, representing 15 countries and including 6109 individuals, met our inclusion criteria. In most (70%) studies, elevated ePASP was defined by a tricuspid regurgitant velocity of 2.5 m/s. The prevalence of elevated ePASP was 21% (17-26%) in children and 30% (26-35%) in adults. After adjustment for sex, SCD genotype, haemoglobin, hydroxycarbamide (hydroxyurea) treatment, country and publication year, age remained associated with elevated ePASP, yielding a 12% (0.4-23%) higher adjusted prevalence in adults. Few studies reported 6-min walk tests or mortality outcomes, and estimates were highly heterogeneous. In random effects meta-analyses, patients with elevated ePASP walked an estimated 30.4 (6.9-53.9) metres less than those without elevated ePASP and had an associated mortality hazard ratio of 4.9 (2.4-9.7).
Topics: Adult; Anemia, Sickle Cell; Antisickling Agents; Arterial Pressure; Echocardiography; Female; Humans; Hydroxyurea; Male; Prevalence; Pulmonary Artery
PubMed: 25854714
DOI: 10.1111/bjh.13447