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International Journal of Chronic... 2023To evaluate the clinical efficacy and safety of bisoprolol in patients with chronic obstructive pulmonary disease (COPD). (Meta-Analysis)
Meta-Analysis
PURPOSE
To evaluate the clinical efficacy and safety of bisoprolol in patients with chronic obstructive pulmonary disease (COPD).
RESEARCH METHODS
This systematic review and meta-analysis was conducted following the Preferred Reporting Items for Systematic Review and Meta-analyses (PRISMA) statements. The primary outcome measures analyzed included: Pulmonary function(FEV1, FEV1%, FVC), 6-minute walking distance (6MWD), adverse events and inflammatory cytokines(IL-6, IL-8, CRP).
RESULTS
Thirty-five studies were included with a total of 3269 study participants, including 1650 in the bisoprolol group and 1619 in the control group. The effect of bisoprolol on lung function in patients with COPD, FEV, MD (0.46 [95% CI, 0.27 to 0.65], P=0.000), FEV%, MD (-0.64 [95% CI, 0.42 to 0.86], P=0.000), FVC, MD (0.20 [95% CI, 0.05 to 0.34], P=0.008), the results all showed a statistically significant result. The effect of bisoprolol on 6MWD in COPD patients, MD (1.37 [95% CI, 1.08 to 1.66], P=0.000), which showed a statistically significant result. The occurrence of adverse events in COPD patients treated with bisoprolol, RR (0.83 [95% CI, 0.54 to 1.26], P=0.382), resulted in no statistical significance. The effect of bisoprolol on inflammatory cytokines in COPD patients, IL-6, MD (-1.16 [95% CI, -1.67 to -0.65], P=0.000), IL-8, MD (-0.94 [95% CI, -1.32 to -0.56], P=0.000), CRP, MD (-1.74 [95% CI, -2.40 to -1.09], P=0.000), the results were statistically significant. We performed a subgroup analysis of each outcome indicator according to whether the patients had heart failure or not, and the results showed that the therapeutic effect of bisoprolol on COPD did not change with the presence or absence of heart failure.
CONCLUSION
Bisoprolol is safe and effective in the treatment of COPD, improving lung function and exercise performance in patients with COPD, and also reducing inflammatory markers in patients with COPD, and this effect is independent of the presence or absence of heart failure.
Topics: Humans; Bisoprolol; Heart Failure; Interleukin-6; Interleukin-8; Pulmonary Disease, Chronic Obstructive; Quality of Life
PubMed: 38152590
DOI: 10.2147/COPD.S438930 -
World Journal of Emergency Surgery :... 2017The objective of this systematic review was to determine the effectiveness and safety of propranolol compared to placebo or usual care for improving clinical relevant... (Meta-Analysis)
Meta-Analysis Review
BACKGROUND
The objective of this systematic review was to determine the effectiveness and safety of propranolol compared to placebo or usual care for improving clinical relevant outcomes in severely burned patients (TBSA >20%).
METHODS
Relevant articles from randomized controlled trials were identified by a literature search in MEDLINE, EMBASE, and CENTRAL. We included trials involving patients with a severe burn (>20% of total body surface area affected). Trials were eligible if they evaluated propranolol and compared to usual care or placebo. Two investigators independently assessed articles for inclusion and exclusion criteria and selected studies for the final analysis. We conducted a meta-analysis using a random-effects model.
RESULTS
We included ten studies in our systematic review. These studies randomized a total of 1236 participants. There were no significant differences between propranolol and placebo with respect to mortality (RD -0.02 [95% CI -0.06 to 0.02]), sepsis (RD -0.03 [95% CI -0.09 to 0.04]), and the overall hospital stay (MD -0.37 [-4.52 to 3.78]). Propranolol-treated adults had a decrease in requirements of blood transfusions (MD -185.64 [95% CI -331.06 to -40.43]) and a decreased heart rate (MD -26.85 [95% CI -39.95 to -13.75]).
CONCLUSIONS
Our analysis indicates that there were no differences in mortality or sepsis in severely burned patients treated with propranolol compared with those who had usual care or placebo. However, the use of propranolol in these patients resulted in lower requirements of blood transfusion and lower values of heart rate. The evidence synthesized in this systematic review is limited to conclude that propranolol reduces the length of hospital stay among severely burned patients. Future trials should assess the impact of propranolol on clinically relevant outcomes such as mortality and adverse events.
Topics: Adrenergic beta-Antagonists; Burns; Humans; Patient Safety; Propranolol
PubMed: 28265298
DOI: 10.1186/s13017-017-0124-7 -
Revista Latino-americana de Enfermagem Dec 2016evaluate the effectiveness of epinephrine used during cardiac arrest and its effect on the survival rates and neurological condition. (Meta-Analysis)
Meta-Analysis Review
OBJECTIVE
evaluate the effectiveness of epinephrine used during cardiac arrest and its effect on the survival rates and neurological condition.
METHOD
systematic review of scientific literature with meta-analysis, using a random effects model. The following databases were used to research clinical trials and observational studies: Medline, Embase and Cochrane, from 2005 to 2015.
RESULTS
when the Return of Spontaneous Circulation (ROSC) with administration of epinephrine was compared with ROSC without administration, increased rates were found with administration (OR 2.02. 95% CI 1.49 to 2.75; I2 = 95%). Meta-analysis showed an increase in survival to discharge or 30 days after administration of epinephrine (OR 1.23; 95% IC 1.05-1.44; I2=83%). Stratification by shockable and non-shockable rhythms showed an increase in survival for non-shockable rhythm (OR 1.52; 95% IC 1.29-1.78; I2=42%). When compared with delayed administration, the administration of epinephrine within 10 minutes showed an increased survival rate (OR 2.03; 95% IC 1.77-2.32; I2=0%).
CONCLUSION
administration of epinephrine appears to increase the rate of ROSC, but when compared with other therapies, no positive effect was found on survival rates of patients with favorable neurological status.
Topics: Epinephrine; Heart Arrest; Humans; Treatment Outcome; Vasopressins
PubMed: 27982306
DOI: 10.1590/1518-8345.1317.2821 -
The Quarterly Journal of Nuclear... Jun 2023Primary hyperparathyroidism (pHPT) is a common endocrine disorder caused by an autonomous overproduction of parathyroid hormone (PTH) by a parathyroid gland. Over the... (Meta-Analysis)
Meta-Analysis
INTRODUCTION
Primary hyperparathyroidism (pHPT) is a common endocrine disorder caused by an autonomous overproduction of parathyroid hormone (PTH) by a parathyroid gland. Over the last decade, F-choline (FCH) PET has emerged as a highly performant imaging technique for guiding parathyroidectomy. As cure is the goal of surgery, the main aims of this study were to summarize patient-based sensitivity, positive predictive value (PPV), and cure rate of FCH PET guided surgery in the surgical management of pHPT.
EVIDENCE ACQUISITION
We conducted a systematic review and metaanalysis according to the PRISMA Guidelines. A literature search was performed in the PubMed, Web of Science and Cochrane databases, last updated November 2022. Original articles on choline PET in patients with pHPT mentioning patient-based sensitivity, PPV and cure rate were retained. Quality of included studies was assessed using the QUADAS-2 Tool. Patient-based sensitivity, PPV and cure rate were pooled by using a random-effects model.
EVIDENCE SYNTHESIS
Twenty-three studies including 1716 patients were included for quantitative assessment. FCH PET showed a pooled patient-based sensitivity of 93.8% (95% CI: 89.8-96.3) and PPV of 97% (95% CI: 92.8-98.8) in patients with pHPT. Parathyroid surgery was performed in 1129 patients. The pooled cure rate of PET-guided surgery was 92.8% (95% CI: 87.4-96.0). Heterogeneity was shown to be moderate for all effect sizes.
CONCLUSIONS
FCH PET showed a high patient-based sensitivity, PPV and cure rate of PET guided surgery in patients with pHPT.
Topics: Humans; Hyperparathyroidism, Primary; Parathyroid Glands; Choline; Positron-Emission Tomography; Positron Emission Tomography Computed Tomography
PubMed: 36756935
DOI: 10.23736/S1824-4785.23.03512-4 -
JAMA Pediatrics Apr 2020Migraine is one of the most common neurologic disorders in children and adolescents. However, a quantitative comparison of multiple preventive pharmacologic treatments... (Meta-Analysis)
Meta-Analysis
IMPORTANCE
Migraine is one of the most common neurologic disorders in children and adolescents. However, a quantitative comparison of multiple preventive pharmacologic treatments in the pediatric population is lacking.
OBJECTIVE
To examine whether prophylactic pharmacologic treatments are more effective than placebo and whether there are differences between drugs regarding efficacy, safety, and acceptability.
DATA SOURCES
Systematic review and network meta-analysis of studies in MEDLINE, Cochrane, Embase, and PsycINFO published through July 2, 2018.
STUDY SELECTION
Randomized clinical trials of prophylactic pharmacologic treatments in children and adolescents diagnosed as having episodic migraine were included. Abstract, title, and full-text screening were conducted independently by 4 reviewers.
DATA EXTRACTION AND SYNTHESIS
Data extraction was conducted according to Preferred Reporting Items for Systematic Reviews and Meta-Analysis network meta-analysis guidelines. Quality was assessed with the Cochrane Risk of Bias tool. Effect sizes, calculated as standardized mean differences for primary outcomes and risk ratios for discontinuation rates, were assessed in a random-effects model.
MAIN OUTCOMES AND MEASURES
Primary outcomes were efficacy (ie, migraine frequency, number of migraine days, number of headache days, headache frequency, or headache index), safety (ie, treatment discontinuation owing to adverse events), and acceptability (ie, treatment discontinuation for any reason).
RESULTS
Twenty-three studies (2217 patients) were eligible for inclusion. Prophylactic pharmacologic treatments included antiepileptics, antidepressants, calcium channel blockers, antihypertensive agents, and food supplements. In the short term (<5 months), propranolol (standard mean difference, 0.60; 95% CI, 0.03-1.17) and topiramate (standard mean difference, 0.59; 95% CI, 0.03-1.15) were significantly more effective than placebo. However, the 95% prediction intervals for these medications contained the null effect. No significant long-term effects for migraine prophylaxis relative to placebo were found for any intervention.
CONCLUSIONS AND RELEVANCE
Prophylactic pharmacologic treatments have little evidence supporting efficacy in pediatric migraine. Future research could (1) identify factors associated with individual responses to pharmacologic prophylaxis, (2) analyze fluctuations of migraine attack frequency over time and determine the most clinically relevant length of probable prophylactic treatment, and (3) identify nonpharmacologic targets for migraine prophylaxis.
Topics: Adolescent; Anticonvulsants; Antidepressive Agents; Antihypertensive Agents; Calcium Channel Blockers; Child; Dietary Supplements; Humans; Migraine Disorders; Propranolol; Topiramate; Vasodilator Agents
PubMed: 32040139
DOI: 10.1001/jamapediatrics.2019.5856 -
The Journal of Trauma and Acute Care... Jan 2016The hypermetabolic state after severe burns is a major problem that can lead to several pathophysiologic changes and produce multiple sequelae. Adrenergic blockade has... (Meta-Analysis)
Meta-Analysis Review
BACKGROUND
The hypermetabolic state after severe burns is a major problem that can lead to several pathophysiologic changes and produce multiple sequelae. Adrenergic blockade has been widely used to reverse these changes and improve outcomes in burned patients but has not been rigorously evaluated. The aim of this systematic review was to investigate the efficacy and safety of the use of adrenergic blockade after burn injury.
METHODS
The databases MEDLINE via OVID, PubMed, EMBASE, CINAHL, Cochrane Library, and Web of Science were searched from inception to December 2014 with search terms including burns and beta-blockers with appropriate synonyms. Articles were restricted to those published in English, French, or Spanish. Randomized controlled trials, nonrandomized controlled trials, and systematic reviews were screened. After an independent screening and full-text review, 10 articles were selected, and an appraisal of risk of bias was performed.
RESULTS
From 182 articles screened, 9 randomized controlled trials and 1 nonrandomized controlled trial met the inclusion criteria. Pooled analyses were performed to calculate effect sizes and 95% confidence intervals (CIs). There was a positive effect favoring propranolol use that significantly decreased resting energy expenditure (g = -0.64; 95% CI, -0.8 to -0.5; p < 0.001) and trunk fat (g = -0.3; 95% CI, -0.4 to -0.1; p < 0.001) as well as improved peripheral lean mass (g = 0.45; 95% CI, 0.3-0.6; p < 0.001) and insulin resistance (g = -1.35; 95% CI, -2.0 to -0.6; p < 0.001). Occurrence of adverse events was not significantly different between the treated patients the and controls.
CONCLUSION
Limited evidence suggests beneficial effects of propranolol after burn injury, and its use seems safe. However, further trials on adult population with a broader range of outcome measures are warranted.
LEVEL OF EVIDENCE
Systematic review and meta-analysis, level III.
Topics: Adrenergic beta-Antagonists; Burns; Energy Metabolism; Humans; Patient Safety; Propranolol
PubMed: 26517779
DOI: 10.1097/TA.0000000000000887 -
Journal of Oral Rehabilitation May 2021To synthesise and critically review the association between sleep bruxism (SB) and stress symptoms in adults. A systematic review was performed. The search was completed... (Meta-Analysis)
Meta-Analysis Review
To synthesise and critically review the association between sleep bruxism (SB) and stress symptoms in adults. A systematic review was performed. The search was completed using seven primary electronic databases in addition to a grey literature search. Two reviewers blindly selected studies based on pre-defined eligibility criteria. Risk of bias of the included articles was performed using the Joanna Briggs Institute Critical Appraisal Checklist for Analytical Cross-Sectional Studies. RevMan 5.4 was used to perform the meta-analysis. The quality of evidence was evaluated according to the Grading of Recommendations Assessment, Development and Evaluation (GRADE). Ten studies were included for qualitative analysis, of which three were included for quantitative analysis. Three studies were evaluated to have low risk of bias, and seven were assessed with moderate risk of bias. Quality of evidence was classified as very low for all outcomes. Individuals with SB were found to have higher levels of some self-reported stress symptoms as assessed through questionnaires with a mean difference of 4.59 (95% CI 0.26-8.92). Biomarkers like epinephrine, norepinephrine, cortisol, adrenaline, dopamine, noradrenaline and prolidase enzyme levels also showed a positive association with SB. Although some associations were identified between probable SB and self-reported stress symptoms and biomarkers of stress in adults, given that the quality of evidence was found to be very low, caution should be exercised in interpreting these results. These findings suggest that additional and better designed studies are warranted in order to clarify the link between SB and stress.
Topics: Adult; Cross-Sectional Studies; Epinephrine; Humans; Self Report; Sleep Bruxism; Surveys and Questionnaires
PubMed: 33377534
DOI: 10.1111/joor.13142 -
The American Journal of Emergency... Apr 2023Cardiogenic shock (CS) is associated with high morbidity and mortality. In recent times, there is increasing interest in the role of angiotensin II in CS. We sought to... (Review)
Review
BACKGROUND
Cardiogenic shock (CS) is associated with high morbidity and mortality. In recent times, there is increasing interest in the role of angiotensin II in CS. We sought to systematically review the current literature on the use of angiotensin II in CS.
METHODS
PubMed, EMBASE, Medline, Web of Science, PubMed Central, and CINAHL databases were systematically searched for studies that evaluated the efficacy of angiotensin II in patients with CS during 01/01/2010-07/07/2022. Outcomes of interest included change in mean arterial pressure (MAP), vasoactive medication requirements (percent change in norepinephrine equivalent [NEE] dose), all-cause mortality, and adverse events.
RESULTS
Of the total 2,402 search results, 15 studies comprising 195 patients were included of which 156 (80%) received angiotensin II. Eleven patients (84.6%) in case reports and case series with reported MAP data at hour 12 noted an increase in MAP. Two studies noted a positive hemodynamic response (defined a priori) in eight (88.9%) and five (35.7%) patients. Eight studies reported a reduction in NEE dose at hour 12 after angiotensin II administration and one study noted a 100% reduction in NEE dose. Out of 47 patients with documented information, 13 patients had adverse outcomes which included hepatic injury (2), digital ischemia (1), ischemic optic neuropathy (1), ischemic colitis (2), agitated delirium (1), and thrombotic events (2).
CONCLUSIONS
In this first systematic review of angiotensin II in CS, we note the early clinical experience. Angiotensin II was associated with improvements in MAP, decrease in vasopressor requirements, and minimal reported adverse events.
Topics: Humans; Shock, Cardiogenic; Angiotensin II; Vasoconstrictor Agents; Norepinephrine; Arterial Pressure; Peptide Hormones; Shock
PubMed: 36753927
DOI: 10.1016/j.ajem.2023.01.050 -
Respiratory Medicine Nov 2022This network meta-analysis (NMA) compared fixed-dose, twice daily fluticasone propionate/salmeterol (FP/Sal) vs. inhaled corticosteroid (ICS) and other ICS/long-acting... (Meta-Analysis)
Meta-Analysis Review
OBJECTIVE
This network meta-analysis (NMA) compared fixed-dose, twice daily fluticasone propionate/salmeterol (FP/Sal) vs. inhaled corticosteroid (ICS) and other ICS/long-acting beta-agonists (LABA) treatments, including when administered using maintenance and reliever therapy (MART) regimens, in terms of improvements in health-related quality of life (HRQoL). The relationship between changes in asthma control and HRQoL was assessed.
METHODS
Articles published between 2001 and 2021, reporting change from baseline (CFB) in Asthma Quality of Life Questionnaire (AQLQ) in patients with moderate-to-severe asthma, were identified by a systematic review. Random effects Bayesian NMAs derived estimates of the mean difference in CFB in AQLQ vs. other interventions connected to the network (included 15 studies). Sensitivity analyses explored the impacts of differences in follow-up duration, baseline asthma control, the inclusion of observational studies, adjusting for baseline FEV, and low-medium ICS dose arms only. Linear regression analysis compared CFBs in AQLQ and Asthma Control Questionnaire (ACQ) score.
RESULTS
Mean CFB in AQLQ with FP/Sal vs. comparators demonstrated expected ranked effects: mean difference 0.65 [95% credible interval: 0.54, 0.78] versus placebo, 0.58 [ 0.33, 0.84] versus LABA, 0.21 [ 0.13, 0.31] versus ICS alone, 0.06 [-0.04, 0.19] versus other ICS/LABA, and 0.00 [-0.13, 0.14] versus ICS/formoterol MART. Sensitivity analyses largely showed consistent results. Improvements in AQLQ and ACQ were strongly correlated (R = 0.94).
CONCLUSIONS
This NMA demonstrates that HRQoL is responsive to treatment, is strongly related to asthma control and that it can be well-managed in patients with moderate-to-severe asthma using regular treatment with inhaled FP/Sal.
Topics: Humans; Fluticasone-Salmeterol Drug Combination; Quality of Life; Bronchodilator Agents; Network Meta-Analysis; Bayes Theorem; Administration, Inhalation; Asthma; Formoterol Fumarate; Adrenal Cortex Hormones; Fluticasone; Drug Combinations
PubMed: 36257125
DOI: 10.1016/j.rmed.2022.106993 -
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