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Rheumatology (Oxford, England) Nov 2023To evaluate the effectiveness and safety of current treatment strategies for the vacuoles, E1 enzyme, X-linked, autoinflammatory, somatic (VEXAS) syndrome.
OBJECTIVES
To evaluate the effectiveness and safety of current treatment strategies for the vacuoles, E1 enzyme, X-linked, autoinflammatory, somatic (VEXAS) syndrome.
METHODS
A protocolized systematic review according to the Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) guidelines was performed. Three databases were searched for reports on treatment strategies for VEXAS. Data from the included publications was extracted and a narrative synthesis was performed. Treatment response was recorded as complete (CR), partial (PR) or none (NR) depending on changes in clinical symptoms and laboratory parameters. Patient characteristics, safety data and previous treatments were analysed.
RESULTS
We identified 36 publications with a total of 116 patients; 113 (98.3%) were male. The identified reports included azacytidine (CR 9/36, 25%; PR 14/36, 38.9%), Janus kinase inhibitors (JAKi) (CR 11/33, 33%; PR 9/33, 27.3%), tocilizumab (CR 3/15, 20%; PR 6/15, 40%), allogeneic stem cell transplantation (CR 6/7, 85.7%; one patient died), anakinra (CR 4/5, 80%; NR 1/5, 20%), canakinumab (CR 1/2, 50%; PR 1/2, 50%) and glucocorticoid monotherapy (CR 1/6, 16.7%; PR 4/6, 66.7%). Individual reports were available for TNF inhibitors, rituximab and MTX. Data on adverse events were available for 67 patients (67/116, 57.8%) and included: pneumonia (12/67, 17.9%), other infections (9/67, 13.4%), venous thromboembolisms (6/67, 8.9%), cytopenias (4/67, 5.9%), and acute (4/67, 5.9%) and chronic graft-vs-host-disease (2/67, 2.9%).
CONCLUSION
Current data on VEXAS treatment are limited and inhomogeneous. Treatment decisions should be individualized. For the devolvement of treatment algorithms clinical trials are needed. Adverse events remain a challenge, especially an elevated risk for venous thromboembolism associated to JAKi treatment should be carefully considered.
Topics: Humans; Male; Female; Algorithms; Azacitidine; Bronchiolitis Obliterans Syndrome; Databases, Factual; Janus Kinase Inhibitors; Mutation
PubMed: 37233149
DOI: 10.1093/rheumatology/kead240 -
International Journal of Medical... Aug 2023Acute respiratory diseases are a leading cause of morbidity and mortality in children. Cough is a common symptom of acute respiratory diseases and the sound of cough can... (Review)
Review
BACKGROUND
Acute respiratory diseases are a leading cause of morbidity and mortality in children. Cough is a common symptom of acute respiratory diseases and the sound of cough can be indicative of the respiratory disease. However, cough sound assessment in routine clinical practice is limited to human perception and the skills of the clinician. Objective cough sound evaluation has the potential to aid clinicians in acute respiratory disease diagnosis. In this systematic review, we assess and summarize the predictive ability of machine learning algorithms in analyzing cough sounds of acute respiratory diseases in the pediatric population.
METHOD
Our systematic search of the Scopus, Medline, and Embase databases on 25 January 2023 identified six articles meeting the inclusion criteria. Quality assessment of the included studies was performed using the checklist for the assessment of medical artificial intelligence.
RESULTS
Our analysis shows variability in the input to the machine learning algorithms, such as the use of various cough sound features and combining cough sound features with clinical features. The use of the machine learning algorithms also varies from conventional algorithms, such as logistic regression and support vector machine, to deep learning techniques, such as convolutional neural networks. The classification accuracy for the detection of bronchiolitis, croup, pertussis, and pneumonia across five articles is in the range of 82-96%. However, a significant drop is observed in the detection accuracy for bronchiolitis and pneumonia in the remaining article.
CONCLUSION
The number of articles is limited but, in general, the predictive ability of cough sound classification algorithms in childhood acute respiratory diseases shows promise.
Topics: Child; Humans; Cough; Artificial Intelligence; Algorithms; Pneumonia; Bronchiolitis; Machine Learning
PubMed: 37224643
DOI: 10.1016/j.ijmedinf.2023.105093 -
Pediatric Research May 2024The aim of this systematic review and meta-analysis was to analyse the efficacy of azithromycin in acute bronchiolitis and wheezing. (Meta-Analysis)
Meta-Analysis
BACKGROUND
The aim of this systematic review and meta-analysis was to analyse the efficacy of azithromycin in acute bronchiolitis and wheezing.
METHODS
PubMed, Scopus, and Web of Science databases were searched for randomized controlled trials comparing azithromycin to placebo in children <2 years of age. Main outcomes were progress of acute wheezing episode and recurrence of wheezing. We used random-effects model to calculate mean difference (MD) with 95% confidence interval (CI) or risk ratios (RR) with CI.
RESULTS
We screened 1604 abstracts and included 7 studies. Risk of bias was low in three and had some concerns in four studies. Need for intensive care unit treatment was assessed in four studies (446 children) and the risk difference was 0.0% (CI -2.0 to 2.0; low quality evidence). Hospitalization duration was -0.27 days shorter in the azithromycin group (MD-0.27, CI -0.47 to -0.07; three studies; moderate quality evidence). Azithromycin did not prevent recurrence of wheezing (RR 0.84, CI 0.45-1.56; three studies), hospital readmissions (RR 1.14, CI 0.82-1.60; four studies).
CONCLUSIONS
We found moderate quality evidence that azithromycin may reduce hospitalization duration. Low certainty evidence suggests that azithromycin does not reduce the need for intensive care unit treatment. Furthermore, azithromycin did not prevent wheezing recurrence.
IMPACT
Azithromycin may reduce hospitalization time in acute bronchiolitis and wheezing episodes among children aged less than two. Azithromycin administrated during the acute wheezing period, does not have preventive effect on wheezing recurrence. Azithromycin seemed to have similar adverse event profile than placebo. Future studies with clinically relevant outcomes, and sufficient sample sizes are needed, before implementing azithromycin into clinical use.
Topics: Humans; Azithromycin; Bronchiolitis; Respiratory Sounds; Anti-Bacterial Agents; Infant; Acute Disease; Treatment Outcome; Hospitalization; Randomized Controlled Trials as Topic; Recurrence; Length of Stay
PubMed: 38066246
DOI: 10.1038/s41390-023-02953-z -
Journal of Ethnopharmacology Mar 2024Tanreqing injection (TRQI) is an intravenous herbal preparation derived from 5 types of traditional Chinese medicines including Scutellariae Radix, Lonicerae Japonicae... (Meta-Analysis)
Meta-Analysis
ETHNOPHARMACOLOGICAL RELEVANCE
Tanreqing injection (TRQI) is an intravenous herbal preparation derived from 5 types of traditional Chinese medicines including Scutellariae Radix, Lonicerae Japonicae Flos, Forsythiae Fructus, bear bile powder and goral horn, incorporating baicalin, chlorogenic acid, ursodeoxycholic acid, and goose deoxycholic acid and other compounds known for anti-inflammatory properties, is widely used in China to treat cough caused by acute trachea-bronchitis disease (ATB).
AIM OF THE STUDY
To investigate the clinical efficacy and safety of Tanreqing injection (TRQI) with and without Western medicine (WM) for cough caused by acute trachea-bronchitis (ATB).
MATERIALS AND METHODS
We systematically searched eight databases, including CENTRAL, Embase, PubMed, Science Direct, Wiley, China National Knowledge Infrastructure, Chinese Biomedical Literature Database and WanFang, from inception to August 2023 for randomized clinical trials (RCTs) on TRQI for cough caused by ATB. The critical outcomes of interest were time to symptom disappearance, including time for cough symptom to disappear and time to improve cough and sputum production. Important outcomes included symptom disappearance rate, adverse events (AEs) and lung function. We carried out random-effects meta-analysis using Review Manager 5.4 and assessed the certainty of evidence utilizing the Grading of Recommendations Assessment, Development and Evaluation (GRADE) approach.
RESULTS
A total of 2872 citations were identified by our search, of which 26 eligible RCTs enrolled 2731 participants. Low to moderate certainty evidence showed that when compared with WM, TRQI plus WM treatment was associated with a favorable effect on the time for cough symptom to disappear (MD -2.21 d, 95% CI -2.64 to -1.78), time to improve cough and sputum production (MD -0.68 d, 95% CI -0.83 to -0.53), symptom disappearance rate (RR 1.37, 95% CI 1.20 to 1.55), forced vital capacity, and forced expiratory volume in 1 s (MD 0.38 L, 95% CI 0.26 to 0.50; MD 2.92%, 95% CI 1.29 to 4.56, respectively). In terms of AEs, there was no association between TRQI plus WM and WM (RR 0.55, 95% CI 0.14 to 2.21; low-certainty evidence). Very low certainty evidence showed that TRQI alone was associated with reduced time to improve cough and sputum (MD -0.14 d, 95% CI -0.26 to -0.02) and increased symptom disappearance rate (RR 1.89, 95% CI 1.24 to 2.88; low certainty evidence) compared to WM.
CONCLUSIONS
The overall efficacy of TRQI or WM for ATB cough is better than that of WM, and TRQI also effectively improve symptoms in patients with similar adverse events. However, due to the lack of methodological rigor of included studies, the present findings should be interpreted with caution. We advocate better high-quality and convincing clinical studies to be performed to prove the effectiveness and safety of TRQIs.
Topics: Humans; Acute Disease; Bronchitis; Cough; Randomized Controlled Trials as Topic; Trachea
PubMed: 38007165
DOI: 10.1016/j.jep.2023.117429 -
Pediatric Pulmonology Feb 2024Until date there is lack of effective therapies in acute bronchiolitis in infants. The aim was to analyze inhaled nitric oxide efficacy in acute bronchiolitis. (Meta-Analysis)
Meta-Analysis
OBJECTIVE
Until date there is lack of effective therapies in acute bronchiolitis in infants. The aim was to analyze inhaled nitric oxide efficacy in acute bronchiolitis.
DESIGN
Systematic review and meta-analysis of randomized controlled trials.
SETTING
Pediatric specialized healthcare.
PATIENTS
All infants (age less than 2 years) having acute bronchiolitis, which requires emergency room visit or hospitalization.
INTERVENTION
Inhaled nitric oxide.
MAIN OUTCOME MEASURES
Need for intensive care unit admission. Secondary outcomes were length of hospital stay and adverse events. Risk ratios (RR) and mean differences with 95% confidence intervals (CI) calculated by random-effects DerSimonian and Laird inverse variance method. Peto Odds ratios were used for rare outcomes. Evidence certainty assessed according to GRADE.
RESULTS
186 studies were screened and three included for analysis. Two had low risk of bias and one had some concerns. Three studies (166 infants) analyzed length of hospital stay and the duration was -11.3 h (CI: -26.8 to +4.2 h) shorter in the nitric oxide group. Evidence certainty was ranked as low. Overall adverse event rates were similar (3 studies, 166 infants, RR: 0.94, CI: 0.70-1.26), but treatment related harms were more common in nitric oxide group (2 studies, 98 infants, OR: 3.86, CI: 1.04-14.40). Evidence certainty in both was rated as low.
CONCLUSIONS
Low certainty evidence suggests that inhaled nitric oxide does not reduce length of hospital stay but may have higher rate of treatment associated harms. Future studies with larger sample sizes are needed to better estimate both the efficacy and adverse events.
Topics: Child; Child, Preschool; Humans; Infant; Bronchiolitis; Hospitalization; Length of Stay; Nitric Oxide; Respiratory Therapy
PubMed: 37988259
DOI: 10.1002/ppul.26767 -
The Lancet. Respiratory Medicine Apr 2024Exposure to household air pollution from polluting domestic fuel (solid fuel and kerosene) represents a substantial global public health burden and there is an urgent... (Meta-Analysis)
Meta-Analysis
Estimated health effects from domestic use of gaseous fuels for cooking and heating in high-income, middle-income, and low-income countries: a systematic review and meta-analyses.
BACKGROUND
Exposure to household air pollution from polluting domestic fuel (solid fuel and kerosene) represents a substantial global public health burden and there is an urgent need for rapid transition to clean domestic fuels. Gas for cooking and heating might possibly affect child asthma, wheezing, and respiratory health. The aim of this review was to synthesise the evidence on the health effects of gaseous fuels to inform policies for scalable clean household energy.
METHODS
In this systematic review and meta-analysis, we summarised the health effects from cooking or heating with gas compared with polluting fuels (eg, wood or charcoal) and clean energy (eg, electricity and solar energy). We searched PubMed, Scopus, Web of Science, MEDLINE, Cochrane Library (CENTRAL), Environment Complete, GreenFile, Google Scholar, Wanfang DATA, and CNKI for articles published between Dec 16, 2020, and Feb 6, 2021. Studies eligible for inclusion had to compare gas for cooking or heating with polluting fuels (eg, wood or charcoal) or clean energy (eg, electricity or solar energy) and present data for health outcomes in general populations. Studies that reported health outcomes that were exacerbations of existing underlying conditions were excluded. Several of our reviewers were involved in screening studies, data extraction, and quality assessment (including risk of bias) of included studies; 20% of studies were independently screened, extracted and quality assessed by another reviewer. Disagreements were reconciled through discussion with the wider review team. Included studies were appraised for quality using the Liverpool Quality Assessment Tools. Key health outcomes were grouped for meta-analysis and analysed using Cochrane's RevMan software. Primary outcomes were health effects (eg, acute lower respiratory infections) and secondary outcomes were health symptoms (eg, respiratory symptoms such as wheeze, cough, or breathlessness). This study is registered with PROSPERO, CRD42021227092.
FINDINGS
116 studies were included in the meta-analysis (two [2%] randomised controlled trials, 13 [11%] case-control studies, 23 [20%] cohort studies, and 78 [67%] cross-sectional studies), contributing 215 effect estimates for five grouped health outcomes. Compared with polluting fuels, use of gas significantly lowered the risk of pneumonia (OR 0·54, 95% CI 0·38-0·77; p=0·00080), wheeze (OR 0·42, 0·30-0·59; p<0·0001), cough (OR 0·44, 0·32-0·62; p<0·0001), breathlessness (OR 0·40, 0·21-0·76; p=0·0052), chronic obstructive pulmonary disease (OR 0·37, 0·23-0·60; p<0·0001), bronchitis (OR 0·60, 0·43-0·82; p=0·0015), pulmonary function deficit (OR 0·27, 0·17-0·44; p<0·0001), severe respiratory illness or death (OR 0·27, 0·11-0·63; p=0·0024), preterm birth (OR 0·66, 0·45-0·97; p=0·033), and low birth weight (OR 0·70, 0·53-0·93; p=0·015). Non-statistically significant effects were observed for asthma in children (OR 1·04, 0·70-1·55; p=0·84), asthma in adults (OR 0·65, 0·43-1·00; p=0·052), and small for gestational age (OR 1·04, 0·89-1·21; p=0·62). Compared with electricity, use of gas significantly increased risk of pneumonia (OR 1·26, 1·03-1·53; p=0·025) and chronic obstructive pulmonary disease (OR 1·15, 1·06-1·25; p=0·0011), although smaller non-significant effects were observed for higher-quality studies. In addition, a small increased risk of asthma in children was not significant (OR 1·09, 0·99-1·19; p=0·071) and no significant associations were found for adult asthma, wheeze, cough, and breathlessness (p>0·05). A significant decreased risk of bronchitis was observed (OR 0·87, 0·81-0·93; p<0·0001).
INTERPRETATION
Switching from polluting fuels to gaseous household fuels could lower health risk and associated morbidity and mortality in resource-poor countries where reliance on polluting fuels is greatest. Although gas fuel use was associated with a slightly higher risk for some health outcomes compared with electricity, gas is an important transitional option for health in countries where access to reliable electricity supply for cooking or heating is not feasible in the near term.
FUNDING
WHO.
Topics: Infant, Newborn; Adult; Child; Female; Humans; Air Pollution, Indoor; Heating; Cross-Sectional Studies; Charcoal; Premature Birth; Asthma; Pulmonary Disease, Chronic Obstructive; Cooking; Dyspnea; Cough; Bronchitis; Pneumonia
PubMed: 38310914
DOI: 10.1016/S2213-2600(23)00427-7 -
Bone Marrow Transplantation Jun 2024Steroid-refractory graft-versus-host disease (SR-GvHD) represents a major complication of pediatric allogenic hematopoietic stem cell transplantation. Ruxolitinib, a... (Meta-Analysis)
Meta-Analysis
Steroid-refractory graft-versus-host disease (SR-GvHD) represents a major complication of pediatric allogenic hematopoietic stem cell transplantation. Ruxolitinib, a selective JAK 1-2 inhibitor, showed promising results in the treatment of SR-GvHD in adult trial, including patients >12 years old. This systematic review aims to evaluate ruxolitinib use for SR-GvHD in the pediatric population. Among the 12 studies included, ruxolitinib administration presented slight differences. Overall response rate (ORR) ranged from 45% to 100% in both acute and chronic GvHD. Complete response rates (CR) varied from 9% to 67% and from 0% to 28% in aGvHD and cGvHD, respectively. Individual-patient meta-analysis from 108 children under 12 years showed an ORR and CR for aGvHD of 74% and 56%, respectively, while in cGvHD ORR was 78% but with only 11% achieving CR. Treatment-related toxicities were observed in 20% of patients, including cytopenia, liver toxicity, and infections. Age, weight, graft source, previous lines of therapy, and dose did not significantly predict response, while a higher rate of toxicities was observed in aGvHD patients. In conclusion, ruxolitinib shows promising results in the treatment of SR-GvHD in children, including those under 12 years. Specific pediatric perspective trials are currently ongoing to definitely assess its efficacy and safety.
Topics: Humans; Graft vs Host Disease; Nitriles; Pyrazoles; Pyrimidines; Child; Chronic Disease; Acute Disease; Child, Preschool; Hematopoietic Stem Cell Transplantation; Male; Female; Adolescent; Infant; Bronchiolitis Obliterans Syndrome
PubMed: 38402346
DOI: 10.1038/s41409-024-02252-z -
European Journal of Pediatrics Feb 2024The objective of the study is to summarize current literature on high-flow nasal cannula (HFNC) use for different indications in pediatric patient excluding acute...
The objective of the study is to summarize current literature on high-flow nasal cannula (HFNC) use for different indications in pediatric patient excluding acute bronchiolitis and neonatal care. The study design is a systematic scoping review. Pubmed, Scopus, and Web of Science databases were searched in February, 2023. All abstracts and full texts were screened by two independent reviewers. Randomized controlled trials focusing on HFNC use in pediatric patients (age < 18 years) were included. Studies focusing on acute bronchiolitis and neonatal respiratory conditions were excluded. Study quality was assessed by Cochrane risk of bias 2.0 tool. The main outcomes are patient groups and indications, key outcomes, and risk of bias. After screening 1276 abstracts, we included 22 full reports. Risk of bias was low in 11 and high in 5 studies. We identified three patient groups where HFNC has been studied: first, children requiring primary respiratory support for acute respiratory failure; second, perioperative use for either intraprocedural oxygenation or postoperative respiratory support; and third, post-extubation care in pediatric intensive care for other than postoperative patients. Clinical and laboratory parameters were assessed as key outcomes. None of the studies analyzed cost-effectiveness.Conclusion: This systematic scoping review provides an overview of current evidence for HFNC use in pediatric patients. Future studies should aim for better quality and include economic evaluation with cost-effectiveness analysis.Protocol registration: Protocol has been published https://osf.io/a3y46/ .
Topics: Adolescent; Child; Humans; Bronchiolitis; Cannula; Continuous Positive Airway Pressure; Oxygen Inhalation Therapy; Randomized Controlled Trials as Topic; Respiratory Distress Syndrome; Infant; Child, Preschool
PubMed: 37962672
DOI: 10.1007/s00431-023-05234-3 -
The Journal of Antimicrobial... Mar 2024Antimicrobial resistance is a worldwide threat, exacerbated by inappropriate prescribing. Most antibiotic prescribing occurs in primary care. Early-career GPs are...
BACKGROUND
Antimicrobial resistance is a worldwide threat, exacerbated by inappropriate prescribing. Most antibiotic prescribing occurs in primary care. Early-career GPs are important for the future of antibiotic prescribing and curbing antimicrobial resistance.
OBJECTIVES
To determine antibiotic prescribing patterns by early-career GPs for common acute infections.
METHODS
A systematic literature search was conducted using PubMed, Embase and Scopus. Two authors independently screened abstracts and full texts for inclusion. Primary outcomes were antibiotic prescribing rates for common acute infections by GPs with experience of 10 years or less. Secondary outcomes were any associations between working experience and antibiotic prescribing.
RESULTS
Of 1483 records retrieved, we identified 41 relevant studies. Early-career GPs were less likely to prescribe antibiotics compared with their more experienced colleagues (OR range 0.23-0.67). Their antibiotic prescribing rates for 'any respiratory condition' ranged from 14.6% to 52%, and for upper respiratory tract infections from 13.5% to 33%. Prescribing for acute bronchitis varied by country, from 15.9% in Sweden to 26% in the USA and 63%-73% in Australia. Condition-specific data for all other included acute infections, such as sinusitis and acute otitis media, were limited to the Australian context.
CONCLUSIONS
Early-career GPs prescribe fewer antibiotics than later-career GPs. However, there are still significant improvements to be made for common acute conditions, as their prescribing is higher than recommended benchmarks. Addressing antimicrobial resistance requires an ongoing worldwide effort and early-career GPs should be the target for long-term change.
Topics: Humans; Acute Disease; Anti-Bacterial Agents; Australia; General Practitioners; Respiratory Tract Infections; Sinusitis; Practice Patterns, Physicians'
PubMed: 38252922
DOI: 10.1093/jac/dkae002 -
The Journal of Infectious Diseases Mar 2024Respiratory syncytial virus (RSV) is a widespread respiratory pathogen, and RSV-related acute lower respiratory tract infections are the most common cause of respiratory...
BACKGROUND
Respiratory syncytial virus (RSV) is a widespread respiratory pathogen, and RSV-related acute lower respiratory tract infections are the most common cause of respiratory hospitalization in children <2 years of age. Over the last 2 decades, a number of severity scores have been proposed to quantify disease severity for RSV in children, yet there remains no overall consensus on the most clinically useful score.
METHODS
We conducted a systematic review of English-language publications in peer-reviewed journals published since January 2000 assessing the validity of severity scores for children (≤24 months of age) with RSV and/or bronchiolitis, and identified the most promising scores. For included articles, (1) validity data were extracted, (2) quality of reporting was assessed using the Transparent Reporting of a multivariable prediction model for Individual Prognosis Or Diagnosis checklist (TRIPOD), and (3) quality was assessed using the Prediction Model Risk Of Bias Assessment Tool (PROBAST). To guide the assessment of the validity data, standardized cutoffs were employed, and an explicit definition of what we required to determine a score was sufficiently validated.
RESULTS
Our searches identified 8541 results, of which 1779 were excluded as duplicates. After title and abstract screening, 6670 references were excluded. Following full-text screening and snowballing, 32 articles, including 31 scores, were included. The most frequently assessed scores were the modified Tal score and the Wang Bronchiolitis Severity Score; none of the scores were found to be sufficiently validated according to our definition. The reporting and/or design of all the included studies was poor. The best validated score was the Bronchiolitis Score of Sant Joan de Déu, and a number of other promising scores were identified.
CONCLUSIONS
No scores were found to be sufficiently validated. Further work is warranted to validate the existing scores, ideally in much larger datasets.
Topics: Child; Humans; Bronchiolitis; Consensus; Hospitalization; Respiratory Syncytial Virus, Human; Respiratory Tract Infections; Respiratory Syncytial Virus Infections
PubMed: 37797314
DOI: 10.1093/infdis/jiad436