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Obesity Reviews : An Official Journal... Mar 2024Social jetlag, the weekly variation in sleep timing, is proposed to contribute to increased obesity risk, potentially because of the misalignment of behavioral cycles... (Meta-Analysis)
Meta-Analysis Review
Social jetlag, the weekly variation in sleep timing, is proposed to contribute to increased obesity risk, potentially because of the misalignment of behavioral cycles relative to the endogenous circadian timing system. This systematic review and meta-analysis aim to determine the association between social jetlag and adiposity-related measures using observational studies. We reviewed 477 references, of which 43 studies met inclusion criteria with a total sample size of 231,648. There was a positive association between social jetlag and body mass index (correlation coefficient [r]: 0.12; 95%CI, 0.07, 0.17; P < 0.001; I = 94.99%), fat mass (r: 0.10; 95%CI, 0.05, 0.15; P < 0.001; I = 0.00%), fat mass index (fat mass divided by height in meter squared, β: 0.14 kg/m ; 95%CI, 0.05, 0.23; P < 0.001; I = 56.50%), percent of body fat (r: 0.37; 95%CI, 0.33, 0.41; P < 0.001; I = 96.17%), waist circumference (r: 0.15; 95%CI, 0.06, 0.24; P = 0.001; I = 90.83%), and the risk of having overweight/obesity (odds ratio: 1.20; 95%CI, 1.02, 1.140; P = 0.039; I = 98.25%). Social jetlag is positively and consistently associated with multiple obesity-related anthropometric measures. Further studies are needed to test causality, underlying mechanisms, and whether obesity interventions based on increasing regularity of the sleep/wake cycle can aid in the battle against the obesity pandemic.
Topics: Humans; Circadian Rhythm; Obesity; Sleep; Jet Lag Syndrome; Body Mass Index; Observational Studies as Topic
PubMed: 38072635
DOI: 10.1111/obr.13664 -
NPJ Primary Care Respiratory Medicine Apr 2017Inhaler device errors are common and may impact the effectiveness of the delivered drug. There is a paucity of up-to-date systematic reviews (SRs) or meta-analyses (MAs)... (Meta-Analysis)
Meta-Analysis Review
UNLABELLED
Inhaler device errors are common and may impact the effectiveness of the delivered drug. There is a paucity of up-to-date systematic reviews (SRs) or meta-analyses (MAs) of device errors in asthma and chronic obstructive pulmonary disease (COPD) patients. This SR and MA provides an estimate of overall error rates (both critical and non-critical) by device type and evaluates factors associated with inhaler misuse. The following databases from inception to July 23, 2014 (Embase®, MEDLINE®, MEDLINE® In-Process and CENTRAL) were searched, using predefined search terms. Studies in adult males and females with asthma or COPD, reporting at least one overall or critical error, using metered dose inhalers and dry powder inhalers were included. Random-effect MAs were performed to estimate device error rates and to compare pairs of devices. Overall and critical error rates were high across all devices, ranging from 50-100% and 14-92%, respectively. However, between-study heterogeneity was also generally >90% (I-squared statistic), indicating large variability between studies. A trend towards higher error rates with assessments comprising a larger number of steps was observed; however no consistent pattern was identified. This SR and MA highlights the relatively limited body of evidence assessing device errors and the lack of standardised checklists. There is currently insufficient evidence to determine differences in error rates between different inhaler devices and their impact on clinical outcomes. A key step in improving our knowledge on this topic would be the development of standardised checklists for each device.
CHRONIC LUNG DISEASES
CALL TO STANDARDISE RESEARCH INTO INHALER DEVICE ERRORS: Researchers should adopt a standardised approach to investigate the incorrect use of inhalers and its associated clinical implications. Henry Chrystyn at Plymouth University, together with scientists across the UK and the Netherlands, conducted a review of research related to inhaled medication errors made by patients with asthma or chronic obstructive pulmonary disease. It is widely acknowledged that many patients with lung conditions don't use their inhaler devices correctly, which affects drug effectiveness and disease control. While Chrystyn's team found high critical error rates reported across all devices, their meta-analysis and systematic review highlighted significant gaps in knowledge regarding different inhalers and associated error rates, and how these affect clinical outcomes. The researchers call for in-depth studies into device use, alongside standardised checklists and definitions for such studies to use to ensure consistency.
Topics: Anti-Asthmatic Agents; Asthma; Bronchodilator Agents; Drug Delivery Systems; Dry Powder Inhalers; Humans; Metered Dose Inhalers; Nebulizers and Vaporizers; Patient Education as Topic; Pulmonary Disease, Chronic Obstructive
PubMed: 28373682
DOI: 10.1038/s41533-017-0016-z -
Journal of the American Academy of... Sep 2023Globally, rates of youth suicide vary considerably. Suicidal thoughts and behaviors (STB) are consistently associated with risk of death by suicide. However,... (Meta-Analysis)
Meta-Analysis
OBJECTIVE
Globally, rates of youth suicide vary considerably. Suicidal thoughts and behaviors (STB) are consistently associated with risk of death by suicide. However, international trends in STB have not yet been compared. To address this gap, an international meta-analysis of epidemiological and school-based studies that report on STB in youth was conducted.
METHOD
Systematic searches were conducted in PubMed and PsycINFO through April 2022. Eligible studies included prevalence of active suicidal ideation (SI) or suicide attempts (SA) in community youth younger than age 22. All studies were coded by 2 authors. Mixed models accounting for shared methods and including hypothesized moderators were conducted using the metafor package in R.
RESULTS
There were 371 effect sizes for SI, 94 for SI with a plan, and 316 for SA, representing 149 regions. Year of data collection ranged from 1981 to 2021. Participants were 6 to 21 years old. The prevalence of SI ranged across regions from 14.3% to 22.6%; the prevalence of SA ranged from 4.6% to 15.8%. Year was not associated with increasing STB prevalence except for studies from the United States, which showed increasing rates of SI and SA since 2007.
CONCLUSION
This is the most comprehensive meta-analysis of STB in youth, providing valuable data about how risk factors most commonly associated with suicide vary internationally and over time. International rates of STB among youth are not improving and may be getting worse in the United States, despite efforts to reduce suicide risk. Most studies did not report rates of SI or SA separately for LGBTQIA+ (lesbian, gay, bisexual, transgender, queer, intersex, asexual, and others) youth and youth of color. A better understanding of proximal risk at the individual level will be important to informing future prevention efforts, especially for high-risk groups.
Topics: Female; Humans; Adolescent; United States; Young Adult; Adult; Child; Suicidal Ideation; Prevalence; Suicide, Attempted; Sexual Behavior; Sexual and Gender Minorities; Risk Factors
PubMed: 36563876
DOI: 10.1016/j.jaac.2022.07.867 -
The Cochrane Database of Systematic... Dec 2021Peripheral arterial disease (PAD) is a manifestation of systemic atherosclerosis. Intermittent claudication is a symptomatic form of PAD that is characterized by pain in... (Meta-Analysis)
Meta-Analysis Review
BACKGROUND
Peripheral arterial disease (PAD) is a manifestation of systemic atherosclerosis. Intermittent claudication is a symptomatic form of PAD that is characterized by pain in the lower limbs caused by chronic occlusive arterial disease. This pain develops in a limb during exercise and is relieved with rest. Propionyl-L-carnitine (PLC) is a drug that may alleviate the symptoms of PAD through a metabolic pathway, thereby improving exercise performance.
OBJECTIVES
The objective of this review is to determine whether propionyl-L-carnitine is efficacious compared with placebo, other drugs, or other interventions used for treatment of intermittent claudication (e.g. exercise, endovascular intervention, surgery) in increasing pain-free and maximum walking distance for people with stable intermittent claudication, Fontaine stage II.
SEARCH METHODS
The Cochrane Vascular Information Specialist searched the Cochrane Vascular Specialised Register, CENTRAL, MEDLINE, Embase, and CINAHL databases and the World Health Organization International Clinical Trials Registry Platform and the ClinicalTrials.gov trials register to July 7, 2021. We undertook reference checking and contact with study authors and pharmaceutical companies to identify additional unpublished and ongoing studies.
SELECTION CRITERIA
Double-blind randomized controlled trials (RCTs) in people with intermittent claudication (Fontaine stage II) receiving PLC compared with placebo or another intervention. Outcomes included pain-free walking performance (initial claudication distance - ICD) and maximal walking performance (absolute claudication distance - ACD), analyzed by standardized treadmill exercise test, as well as ankle brachial index (ABI), quality of life, progression of disease, and adverse events.
DATA COLLECTION AND ANALYSIS
Two review authors independently selected trials, extracted data, and evaluated trials for risk of bias. We contacted study authors for additional information. We resolved any disagreements by consensus. We performed fixed-effect model meta-analyses with mean differences (MDs) and 95% confidence intervals (CIs). We graded the certainty of evidence according to GRADE.
MAIN RESULTS
We included 12 studies in this review with a total number of 1423 randomized participants. A majority of the included studies assessed PLC versus placebo (11 studies, 1395 participants), and one study assessed PLC versus L-carnitine (1 study, 26 participants). We identified no RCTs that assessed PLC versus any other medication, exercise, endovascular intervention, or surgery. Participants received PLC 1 grams to 2 grams orally (9 studies) or intravenously (3 studies) per day or placebo. For the comparison PLC versus placebo, there was a high level of both clinical and statistical heterogeneity due to study size, participants coming from different countries and centres, the combination of participants with and without diabetes, and use of different treadmill protocols. We found a high proportion of drug company-backed studies. The overall certainty of the evidence was moderate. For PLC compared with placebo, improvement in maximal walking performance (ACD) was greater for PLC than for placebo, with a mean difference in absolute improvement of 50.86 meters (95% CI 50.34 to 51.38; 9 studies, 1121 participants), or a 26% relative improvement (95% CI 23% to 28%). Improvement in pain-free walking distance (ICD) was also greater for PLC than for placebo, with a mean difference in absolute improvement of 32.98 meters (95% CI 32.60 to 33.37; 9 studies, 1151 participants), or a 31% relative improvement (95% CI 28% to 34%). Improvement in ABI was greater for PLC than for placebo, with a mean difference in improvement of 0.09 (95% CI 0.08 to 0.09; 4 studies, 369 participants). Quality of life improvement was greater with PLC (MD 0.06, 95% CI 0.05 to 0.07; 1 study, 126 participants). Progression of disease and adverse events including nausea, gastric intolerance, and flu-like symptoms did not differ greatly between PLC and placebo. For the comparison of PLC with L-carnitine, the certainty of evidence was low because this included a single, very small, cross-over study. Mean improvement in ACD was slightly greater for PLC compared to L-carnitine, with a mean difference in absolute improvement of 20.00 meters (95% CI 0.47 to 39.53; 1 study, 14 participants) or a 16% relative improvement (95% CI 0.4% to 31.6%). We found no evidence of a clear difference in the ICD (absolute improvement 4.00 meters, 95% CI -9.86 to 17.86; 1 study, 14 participants); or a 3% relative improvement (95% CI -7.4% to 13.4%). None of the other outcomes of this review were reported in this study.
AUTHORS' CONCLUSIONS
When PLC was compared with placebo, improvement in walking distance was mild to moderate and safety profiles were similar, with moderate overall certainty of evidence. Although In clinical practice, PLC might be considered as an alternative or an adjuvant to standard treatment when such therapies are found to be contraindicated or ineffective, we found no RCT evidence comparing PLC with standard treatment to directly support such use.
Topics: Ankle Brachial Index; Carnitine; Humans; Intermittent Claudication; Peripheral Arterial Disease; Randomized Controlled Trials as Topic; Walking
PubMed: 34954832
DOI: 10.1002/14651858.CD010117.pub2 -
The Cochrane Database of Systematic... Jan 2017Individuals with pulmonary hypertension (PH) have reduced exercise capacity and quality of life. Despite initial concerns that exercise training may worsen symptoms in... (Meta-Analysis)
Meta-Analysis Review
BACKGROUND
Individuals with pulmonary hypertension (PH) have reduced exercise capacity and quality of life. Despite initial concerns that exercise training may worsen symptoms in this group, several studies have reported improvements in functional capacity and well-being following exercise-based rehabilitation in PH.
OBJECTIVES
To assess the efficacy and safety of exercise-based rehabilitation for people with PH. Primary outcomes were exercise capacity, adverse events during the intervention period and health-related quality of life (HRQoL). Secondary outcomes included cardiopulmonary haemodynamics, functional class, clinical worsening during follow-up, mortality and changes in B-type natriuretic peptide.
SEARCH METHODS
We searched the Cochrane Airways Specialised Register of Trials up to August 2016, which is based on regular searches of CINAHL, AMED, Embase, PubMed, MEDLINE, PsycINFO and registries of clinical trials. In addition we searched CENTRAL and the PEDro database up to August 2016 and handsearched relevant journals.
SELECTION CRITERIA
All randomised controlled trials (RCTs) focusing on exercise-based rehabilitation programmes for PH.
DATA COLLECTION AND ANALYSIS
Two reviewers extracted data independently. For binary outcomes, we calculated odds ratios and their 95% confidence interval (CI), on an intention-to-treat basis. For continuous data, we estimated the mean difference (MD) between groups and its 95% CI. We employed a random-effects model for analyses. We assessed risk of bias for included studies and created 'Summary of findings' tables using GRADE.
MAIN RESULTS
We included six RCTs and were able to extract data from five studies. The majority of participants were Group I pulmonary artery hypertension (PAH). Study duration ranged from three to 15 weeks. Exercise programmes included both inpatient- and outpatient-based rehabilitation that incorporated both upper and lower limb exercise. The mean six-minute walk distance following exercise training was 60.12 metres higher than control (30.17 to 90.07 metres, n = 165, 5 RCTs, low-quality evidence; minimal important difference was 30 metres), the mean peak oxygen uptake was 2.4 ml/kg/minute higher (1.4 to 3.4 ml/kg/min, n = 145, 4 RCTs, low-quality evidence) and the mean peak power in the intervention groups was 16.4 W higher (10.9 to 22.0 higher, n = 145, 4 RCTs, low-quality evidence). The mean change in HRQoL for the SF-36 physical component score was 4.63 points higher (0.80 to 8.47 points, n = 33, 2 RCTs, low-quality evidence) and for the SF-36 mental component score was 4.17 points higher (0.01 to 8.34 points; n = 33; 2 RCTs, low-quality evidence). One study reported a single adverse event, where a participant stopped exercise training due to lightheadedness.
AUTHORS' CONCLUSIONS
In people with PH, exercise-based rehabilitation results in clinically relevant improvements in exercise capacity. Exercise training was not associated with any serious adverse events. Whilst most studies reported improvements in HRQoL, these may not be clinically important. Overall, we assessed the quality of the evidence to be low. The small number of studies and lack of information on participant selection makes it difficult to generalise these results across the spectrum of people with PH.
Topics: Exercise Therapy; Exercise Tolerance; Hemodynamics; Humans; Hypertension, Pulmonary; Middle Aged; Oxygen Consumption; Quality of Life; Randomized Controlled Trials as Topic; Selection Bias; Walk Test
PubMed: 28099988
DOI: 10.1002/14651858.CD011285.pub2 -
PloS One 2022To evaluate the efficacy and safety of cilostazol, pentoxifylline, beraprost for intermittent claudication due to lower extremity arterial occlusive disease. (Meta-Analysis)
Meta-Analysis
OBJECTIVE
To evaluate the efficacy and safety of cilostazol, pentoxifylline, beraprost for intermittent claudication due to lower extremity arterial occlusive disease.
METHODS
Randomized controlled clinical trials were identified from PubMed, Scopus, EMbase, Cochrane Library, Web of Science, China National Knowledge Infrastructure, SinoMed, Wanfang and Chongqing VIP databases, from the database inception to 31/12/2021. The outcome measures were walking distance measured by treadmill (maximum and pain-free walking distance), ankle-brachial index and adverse events. The quality of included studies was assessed by the Cochrane bias risk assessment tool. A network meta-analysis was carried out with Stata 16.0 software.
RESULTS
There were 29 RCTs included in the study, covering total 5352 patients. Cilostazol was ranked first for both maximum and pain-free walking distance, followed by beraprost and pentoxifylline. For cilostazol, pentoxifylline and beraprost, maximum walking distance increased by 62.93 95%CI(44.06, 81.79), 32.72 95%CI(13.51, 55.79) and 43.90 95%CI(2.10, 85.71) meters, respectively relative to placebo, and pain-free walking distance increased by 23.92 95%CI(11.24, 36.61), 15.16 95%CI(2.33, 27.99) and 19.78 95%CI(-3.07, 42.62) meters. For cilostazol, pentoxifylline, beraprost and cilostazol combined with beraprost, ankle-brachial index increased by 0.06 95%CI(0.04, 0.07), -0.01 95%CI(-0.08, 0.05), 0.18 95%CI(0.12, 0.23) and 0.23 95%CI(0.18, 0.27), respectively relative to placebo. The pentoxifylline and cilostazol was associated with a lower ratio of adverse events than beraprost and cilostazol combined with beraprost.
CONCLUSION
Cilostazol, pentoxifylline and beraprost were all effective treatments for intermittent claudication; cilostazol with good tolerance was likely to be the most effective in walking distance, while beraprost and cilostazol combined with beraprost were more prominent in the ankle-brachial index.
Topics: Humans; Cilostazol; Intermittent Claudication; Network Meta-Analysis; Pentoxifylline; Vasodilator Agents; Randomized Controlled Trials as Topic
PubMed: 36318524
DOI: 10.1371/journal.pone.0275392 -
Journal of Medical Systems Sep 2016A systematic review allows us to identify, assess, and interpret all possible relevant work associated with a question in particular or the subject of an area. Different... (Review)
Review
A systematic review allows us to identify, assess, and interpret all possible relevant work associated with a question in particular or the subject of an area. Different authors can use several methodologies to learn about research related to their own research in different fields. The main objective of this review is to identify work, research and publications made in the field of the mobile monitoring of patients through some application or commercial or non-commercial solutions in m-Health. Next, we compare the different solutions with our solution, MoMo (Mobile Monitoring) Framework. MoMo is a solution that allows for patient mobile monitoring through mobile phones and biometric devices (blood pressure meter, glucometer and others). Our systematic review is based on the methodology of B. Kitchenham. She proposed specific guidelines for carrying out a systematic review in software engineering. We prepare our systematic review base in the selection of primary and secondary research related to mobile monitoring solutions following criteria with a specific weight to compare with each part of our research.
Topics: Cell Phone; Computer Systems; Humans; Monitoring, Physiologic; Telemedicine
PubMed: 27464519
DOI: 10.1007/s10916-016-0559-5 -
Child Abuse & Neglect Aug 2023Children with maltreatment histories demonstrate weaker reading abilities compared to their peers. However, the differential processes driving this effect remain...
BACKGROUND
Children with maltreatment histories demonstrate weaker reading abilities compared to their peers. However, the differential processes driving this effect remain unclear. Prior studies focused on social and behavioral factors explaining this effect, yet reading research has shown that one's ability to comprehend written text is driven by a set of underlying dynamic and interactive cognitive abilities.
OBJECTIVE
This systematic review sought to understand what theoretical or conceptual frameworks researchers cited as guiding their studies, what reading processes and abilities were studied as outcomes, how reading processes or abilities were measured, and what constructs were included to help understand the relationship between maltreatment and reading.
METHOD
Three databases were searched for empirical peer-reviewed journal articles. Articles retained using inclusion and exclusion criteria were coded based on their sample characteristics, reference to theoretical or conceptual frameworks, reading processes and abilities measured, and included predictors of reading. Procedures were documented using the reporting items for systematic reviews and meta-analyses (PRISMA) statement (Moher et al., 2009).
RESULTS
Twenty-seven studies were included in the final systematic review. Those that discussed theoretical or conceptual frameworks focused on the social and behavioral predictors of reading. Many studies (51.9 %) examined effects of maltreatment on reading achievement, rather than specific reading processes or abilities. Most studies (92.6 %) used at least one standardized reading measure. However, only four studies included cognitive abilities as potential predictor variables.
CONCLUSIONS
Future research could benefit from investigating specific cognitive and reading-related processes, using measures to examine specific reading processes leading to breakdowns in reading achievement, and incorporation of reading theories to drive research questions and methods.
Topics: Child; Humans; Reading; Cognition; Achievement; Child Abuse
PubMed: 36089407
DOI: 10.1016/j.chiabu.2022.105857 -
Deutsches Arzteblatt International May 2017Infections of the genital tract are considered common causes of male fertility disorders, with a prevalence of 6-10%. Most of the affected men are asymptomatic. The... (Review)
Review
BACKGROUND
Infections of the genital tract are considered common causes of male fertility disorders, with a prevalence of 6-10%. Most of the affected men are asymptomatic. The diagnostic evaluation is based mainly on laboratory testing. Inconsistent diagnostic criteria have been applied to date, and this may explain the controversial debate about the role of infection and inflammation in the genital tract as a cause of infertility. The risk of an irreversible fertility disorder should not be underestimated.
METHODS
This review is based on pertinent publications retrieved by a selective literature search in PubMed, including guidelines from Germany and abroad and systematic review articles.
RESULTS
The main causes of inflammatory disease of the male genital tract are ascending sexually transmitted infections (STIs) and uropathogens. Chronic prostatitis has no more than a limited influence on ejaculate variables. By contrast, approximately 10% of men who have had acute epididymitis develop persistent azoospermia thereafter, and 30% have oligozoospermia. Obstruction of the excurrent ducts can ensue, as can post-infectious disturbances of spermatogenesis. The differential diagnostic evaluation includes the determination of testicular volumes, hormone concentrations, and ejaculate variables. Epidemiological data are lacking with regard to infertility after primary orchitis of infectious origin; however, up to 25% of testicular biopsies obtained from infertile men reveal focal inflammatory reactions. Multiple studies have suggested a deleterious effect of leukocytes and inflammatory mediators on sperm para - meters. On the other hand, the clinical significance of bacteriospermia remains unclear.
CONCLUSION
Any suspicion of an infectious or inflammatory disease in the male genital tract should prompt a systematic diagnostic evaluation and appropriate treatment. For patients with obstructive azoospermia, the etiology and site of the obstruction determine the surgical approach to be taken. In the near future, the elucidation of underlying pathophysiological mechanisms and the identification of suitable biomarkers may enable new strategies for conservative treatment.
Topics: Germany; Humans; Infertility, Male; Inflammation; Male; Risk Factors; Urinary Tract Infections
PubMed: 28597829
DOI: 10.3238/arztebl.2017.0339 -
The Cochrane Database of Systematic... Dec 2023Pompe disease is caused by a deficiency of the enzyme acid alpha-glucosidase (GAA). People with infantile-onset disease have either a complete or a near-complete enzyme... (Review)
Review
BACKGROUND
Pompe disease is caused by a deficiency of the enzyme acid alpha-glucosidase (GAA). People with infantile-onset disease have either a complete or a near-complete enzyme deficiency; people with late-onset Pompe disease (LOPD) retain some residual enzyme activity. GAA deficiency is treated with an intravenous infusion of recombinant human acid alglucosidase alfa, an enzyme replacement therapy (ERT). Alglucosidase alfa and avalglucosidase alfa are approved treatments, but cipaglucosidase alfa with miglustat is not yet approved.
OBJECTIVES
To assess the effects of enzyme replacement therapies in people with late-onset Pompe disease.
SEARCH METHODS
We searched the Cochrane Inborn Errors of Metabolism Trials Register, compiled from electronic database searches and handsearching of journals and conference abstract books. We also searched MEDLINE OvidSP, clinical trial registries, and the reference lists of relevant articles and reviews. Date of last search: 21 April 2022.
SELECTION CRITERIA
We included randomised controlled trials (RCTs) of ERT in people with LOPD of any age.
DATA COLLECTION AND ANALYSIS
Two review authors independently assessed trial eligibility, extracted data, assessed the risk of bias and the certainty of the evidence (using GRADE). We resolved disagreements through discussion and by consulting a third author.
MAIN RESULTS
We included six trials (358 randomised participants) lasting from 12 to 78 weeks. A single trial reported on each comparison listed below. None of the included trials assessed two of our secondary outcomes: need for respiratory support and use of a walking aid or wheelchair. Certainty of evidence was most commonly downgraded for selective reporting bias. Alglucosidase alfa versus placebo (90 participants) After 78 weeks, alglucosidase alfa probably improves the six-minute walk test (6MWT) distance compared to placebo (mean difference (MD) 30.95 metres, 95% confidence interval (CI) 7.98 to 53.92; moderate-certainty evidence) and probably improves respiratory function, measured as the change in per cent (%) predicted forced vital capacity (FVC) (MD 3.55, 95% CI 1.46 to 5.64; moderate-certainty evidence). There may be little or no difference between the groups in occurrence of infusion reactions (risk ratio (RR) 1.21, 95% CI 0.57 to 2.61; low-certainty evidence), quality of life physical component score (MD -1.36 points, 95% CI -5.59 to 2.87; low-certainty evidence), or adverse events (RR 0.94, 95% CI 0.64 to 1.39; low-certainty evidence). Alglucosidase alfa plus clenbuterol versus alglucosidase alfa plus placebo (13 participants) The evidence is very uncertain about the effect of alglucosidase alfa plus clenbuterol compared to alglucosidase alfa plus placebo on: change in 6MWT distance after 52 weeks (MD 34.55 metres, 95% CI-10.11 to 79.21; very low-certainty evidence) and change in % predicted FVC (MD -13.51%, 95% CI -32.44 to 5.41; very low-certainty evidence). This study did not measure infusion reactions, quality of life, and adverse events. Alglucosidase alfa plus albuterol versus alglucosidase alfa plus placebo (13 participants) The evidence is very uncertain about the effect of alglucosidase alfa plus albuterol compared to alglucosidase alfa plus placebo on: change in 6MWT distance after 52 weeks (MD 30.00 metres, 95% CI 0.55 to 59.45; very low-certainty evidence), change in % predicted FVC (MD -4.30%, 95% CI -14.87 to 6.27; very low-certainty evidence), and risk of adverse events (RR 0.67, 95% CI 0.38 to 1.18; very low-certainty evidence). This study did not measure infusion reactions and quality of life. VAL-1221 versus alglucosidase alfa (12 participants) Insufficient information was available about this trial to generate effect estimates measured at one year or later. Compared to alglucosidase alfa, VAL-1221 may increase or reduce infusion-associated reactions at three months, but the evidence is very uncertain (RR 2.80, 95% CI 0.18 to 42.80). This study did not measure quality of life and adverse events. Cipaglucosidase alfa plus miglustat versus alglucosidase alfa plus placebo (125 participants) Compared to alglucosidase alfa plus placebo, cipaglucosidase alfa plus miglustat may make little or no difference to: 6MWT distance at 52 weeks (MD 13.60 metres, 95% CI -2.26 to 29.46); infusion reactions (RR 0.94, 95% CI 0.49 to 1.80); quality of life scores for physical function (MD 1.70, 95% CI -2.13 to 5.53) and fatigue (MD -0.30, 95% CI -2.76 to 2.16); and adverse effects potentially related to treatment (RR 0.83, 95% CI 0.49 to 1.40) (all low-certainty evidence). Cipaglucosidase alfa plus miglustat probably improves % predicted FVC compared to alglucosidase alfa plus placebo (MD 3.10%, 95% CI 1.04 to 5.16; moderate-certainty evidence); however, it may make little or no change in % predicted sniff nasal inspiratory pressure (MD -0.06%, 95% CI -8.91 to 7.71; low-certainty evidence). Avalglucosidase alfa versus alglucosidase alfa (100 participants) After 49 weeks, avalglucosidase alfa probably improves 6MWT compared to alglucosidase alfa (MD 30.02 metres, 95% CI 1.84 to 58.20; moderate-certainty evidence). Avalglucosidase alfa probably makes little or no difference to % predicted FVC compared to alglucosidase alfa (MD 2.43%, 95% CI -0.08 to 4.94; moderate-certainty evidence). Avalglucosidase alfa may make little or no difference to infusion reactions (RR 0.78, 95% CI 0.42 to 1.45), quality of life (MD 0.77, 95% CI -2.09 to 3.63), or treatment-related adverse events (RR 0.92, 95% CI 0.61 to 1.40), all low-certainty evidence.
AUTHORS' CONCLUSIONS
One trial compared the effect of ERT to placebo in LOPD, showing that alglucosidase alfa probably improves 6MWT and respiratory function (both moderate-certainty evidence). Avalglucosidase alfa probably improves 6MWT compared with alglucosidase alfa (moderate-certainty evidence). Cipaglucosidase plus miglustat probably improves FVC compared to alglucosidase alfa plus placebo (moderate-certainty evidence). Other trials studied the adjunct effect of clenbuterol and albuterol along with alglucosidase alfa, with little to no evidence of benefit. No significant rise in adverse events was noted with all ERTs. The impact of ERT on some outcomes remains unclear, and longer RCTs are needed to generate relevant information due to the progressive nature of LOPD. Alternative resources, such as post-marketing registries, could capture some of this information.
Topics: Humans; Glycogen Storage Disease Type II; Enzyme Replacement Therapy; Clenbuterol; Albuterol
PubMed: 38084761
DOI: 10.1002/14651858.CD012993.pub2