Cystic Fibrosis

Impact of the vancomycin and amikacin therapeutic monitoring in the optimization of antimicrobial dose in pediatric patients

Fri, 2021-09-03 06:00

Rev Chilena Infectol. 2021 Jun;38(3):317-323. doi: 10.4067/S0716-10182021000300317.

ABSTRACT

BACKGROUND: The monitoring of antimicrobial therapy through plasma levels makes it possible to determine the optimal dosage of antimicrobials, an essential approach in pediatrics.

AIM: To describe the monitoring of plasma antimicrobial levels and dose adjustment in the pediatric population to determine if the doses used reach therapeutic ranges.

METHODS: Retrospective, descriptive study using a database with measurement of plasma levels of amikacin and vancomycin in pediatric patients at San Borja Arriarán Hospital between 2015-2018. The number of patients who reached the therapeutic range with the initial dose, how many required adjustment and their characteristics were determined.

RESULTS: 104 total levels were monitored. For vancomycin 65 plasmatic levels were baseline, being outside the therapeutic range 56.5%; 25% of those requiring adjustment were neonates with a higher probability of being out of range versus others (p = 0.022). For amikacin, Cpeak was in range in 60% of measurements; 15.4% required adjustment, including patients with cystic fibrosis and cancer, without adjustments in patients without comorbidity.

CONCLUSION: Measurement of plasma levels is necessary to individually adjust the dose, especially in pediatric patients with cystic fibrosis, oncology and in neonatology where it is more likely not to reach a therapeutic range with initial doses.

PMID:34479286 | DOI:10.4067/S0716-10182021000300317

Categories: Literature Watch

Tandem mass tag-based quantitative proteomic analysis of the liver reveals potential protein targets of Xiaochaihutang in CUMS model of depression

Fri, 2021-09-03 06:00

J Chromatogr B Analyt Technol Biomed Life Sci. 2021 Aug 28;1181:122898. doi: 10.1016/j.jchromb.2021.122898. Online ahead of print.

ABSTRACT

Depression is a global mental disorder disease and greatly threatened human health. Xiaochaihutang (XCHT) has been used successfully in treatment of depression for many years in China, but the mechanism is unclear. Using the chronic unpredictable mild stress (CUMS) mice model of depression, the present study aimed to reveal possible antidepressant mechanisms of XCHT from the perspective of liver by analyzing hepatic proteomics in mice. Bioinformatics analysis identified 31 differentially expressed proteins (DEPs), including 5 upregulated and 26 downregulated proteins, between the CUMS model and XCHT groups. The bile secretion pathway was found by KEGG pathway analysis of these DEPs. Four of the 31 differentially expressed proteins, including 2 active proteins involved in bile secretion, carbonic anhydrase 2 (CA2) and cystic fibrosis transmembrane conductance regulator (CFTR), were selected to verify their genes. Four genes (Cyp7a1, Fxr, Shp and Ntcp) related to bile acid synthesis and transport were further investigated by quantitative real-time polymerase chain reaction (qRT-PCR). Both biochemical tests and gene studies demonstrated that CUMS affected bile acid synthesis and transport, while XCHT regulated this pathway. The results indicated that there may be a potential relationship between CUMS induced depression and hepatic injury caused by increased bile acid, and also provide a novel insight to understand the underlying anti-depression mechanisms of XCHT.

PMID:34479180 | DOI:10.1016/j.jchromb.2021.122898

Categories: Literature Watch

Simple Measurement of IgA Predicts Immunity and Mortality in Ataxia-Telangiectasia

Fri, 2021-09-03 06:00

J Clin Immunol. 2021 Sep 3. doi: 10.1007/s10875-021-01090-8. Online ahead of print.

ABSTRACT

Patients with ataxia-telangiectasia (A-T) suffer from progressive cerebellar ataxia, immunodeficiency, respiratory failure, and cancer susceptibility. From a clinical point of view, A-T patients with IgA deficiency show more symptoms and may have a poorer prognosis. In this study, we analyzed mortality and immunity data of 659 A-T patients with regard to IgA deficiency collected from the European Society for Immunodeficiencies (ESID) registry and from 66 patients with classical A-T who attended at the Frankfurt Goethe-University between 2012 and 2018. We studied peripheral B- and T-cell subsets and T-cell repertoire of the Frankfurt cohort and survival rates of all A-T patients in the ESID registry. Patients with A-T have significant alterations in their lymphocyte phenotypes. All subsets (CD3, CD4, CD8, CD19, CD4/CD45RA, and CD8/CD45RA) were significantly diminished compared to standard values. Patients with IgA deficiency (n = 35) had significantly lower lymphocyte counts compared to A-T patients without IgA deficiency (n = 31) due to a further decrease of naïve CD4 T-cells, central memory CD4 cells, and regulatory T-cells. Although both patient groups showed affected TCR-ß repertoires compared to controls, no differences could be detected between patients with and without IgA deficiency. Overall survival of patients with IgA deficiency was significantly diminished. For the first time, our data show that patients with IgA deficiency have significantly lower lymphocyte counts and subsets, which are accompanied with reduced survival, compared to A-T patients without IgA deficiency. IgA, a simple surrogate marker, is indicating the poorest prognosis for classical A-T patients. Both non-interventional clinical trials were registered at clinicaltrials.gov 2012 (Susceptibility to infections in ataxia-telangiectasia; NCT02345135) and 2017 (Susceptibility to Infections, tumor risk and liver disease in patients with ataxia-telangiectasia; NCT03357978).

PMID:34477998 | DOI:10.1007/s10875-021-01090-8

Categories: Literature Watch

Highly efficient photothermal nanoparticles for the rapid eradication of bacterial biofilms

Fri, 2021-09-03 06:00

Nanoscale. 2021 Aug 28;13(32):13610-13616. doi: 10.1039/d1nr03471e. Epub 2021 Aug 3.

ABSTRACT

Biofilm-related infections, such as dental plaque, chronic sinusitis, native valve endocarditis, and chronic airway infections in cystic fibrosis have brought serious suffering to patients and financial burden to society. Materials that can eliminate mature biofilms without developing drug resistance are promising tools to treat biofilm-related infections, and thus they are in urgent demand. Herein, we designed and readily prepared organic nanoparticles (NPs) with highly efficient photothermal conversion by harvesting energy via excited-state intramolecular motions and enlarging molar absorptivity. The photothermal NPs can sufficiently eliminate mature bacterial biofilms upon low-power near-infrared laser irradiation. NPs hold great promise for the rapid eradication of bacterial biofilms by photothermal therapy.

PMID:34477635 | DOI:10.1039/d1nr03471e

Categories: Literature Watch

Serum inflammatory profiles in cystic fibrosis mice with and without Bordetella pseudohinzii infection

Fri, 2021-09-03 06:00

Sci Rep. 2021 Sep 2;11(1):17535. doi: 10.1038/s41598-021-97033-9.

ABSTRACT

Cystic fibrosis (CF) is an autosomal recessive disease caused by dysfunctional cystic fibrosis transmembrane conductance regulator (CFTR) protein, and is marked by an accumulation of mucus in affected airways resulting in persistent infection and chronic inflammation. Quantitative differences in inflammatory markers have been observed in CF patient serum, tracheal cells, and bronchoalveolar lavage fluid, in the absence of detectable infection, implying that absent CFTR function alone may result in dysregulated immune responses. To examine the relationship between absent CFTR and systemic inflammation, 22 analytes were measured in CF mice (F508del/F508del) sera using the MSD multiplex platform. Pro-inflammatory cytokines IL-2, TNF-α, IL-17α, IFN-γ, IL-1β, and MIP-3α are significantly elevated in infection-naïve CF mice (p < 0.050). Anti-inflammatory cytokines IL-10 and IL-4 are also significantly increased (p = 0.00003, p = 0.004). Additionally, six general markers of inflammation are significantly different from non-CF controls (p < 0.050). To elucidate the effects of chronic infection on the CF inflammatory profile, we examined CF mice exposed to spontaneous Bordetella pseudohinzii infections. There are no statistical differences in nearly all inflammatory markers when compared to their infection-naïve CF counterparts, except in the Th2-derived IL-4 and IL-5 which demonstrate significant decreases following exposure (p = 0.046, p = 0.045). Lastly, following acute infection, CF mice demonstrate elevations in nearly all inflammatory markers, but exhibit a shortened return to uninfected levels over time, and suppression of Th1-derived IL-2 and IL-5 (p = 0.043, p = 0.011). These results imply that CF mice have a persistent inflammatory profile often indistinguishable from chronic infection, and a dysregulated humoral response during and following active infection.

PMID:34475490 | DOI:10.1038/s41598-021-97033-9

Categories: Literature Watch

A comparison of clinic and home spirometry as longtudinal outcomes in cystic fibrosis

Fri, 2021-09-03 06:00

J Cyst Fibros. 2021 Aug 30:S1569-1993(21)01354-0. doi: 10.1016/j.jcf.2021.08.013. Online ahead of print.

ABSTRACT

BACKGROUND: The COVID-19 pandemic has accelerated the transition to telehealth, including the use of home spirometry in cystic fibrosis. Evaluating the accuracy and precision of longitudinal home spirometry is a requisite for telehealth-based research. This secondary analysis of a CF study (eICE) evaluates whether there are cross-sectional or longitudinal differences between home and clinic spirometry.

METHODS: Participants age ≥14 years with ppFEV1>25 were recruited from 2011-2015, issued a home spirometer, and asked to complete spirometry efforts twice per week for one year. Clinic spirometry was collected at baseline and every three months. Cross-sectional differences between clinic spirometry and the closest home spirometry measurement were analyzed. Longitudinally, we apply 5 methods to analyze the precision of home spirometry, and differences between clinic vs. home data.

RESULTS: Home spirometry is estimated to be 2.0 (95% CI: 0.3, 3.5) percentage points lower than clinic spirometry cross-sectionally. Longitudinally, the estimates of 12-month change in home spirometry varied by analysis method from -2.6 to -1.0 ppFEV1/ year, with precision markedly different. However, home spirometry change estimates were qualitatively similar to the clinic results: -3.0 ppFEV1/year (95% CI: -4.1, -1.9).

CONCLUSIONS: To leverage the potential cost, feasibility and convenience of home spirometry, the differences with clinic spirometry must be acknowledged. Significantly lower ppFEV1 in home devices shows that direct comparison to clinic spirometers may induce a spurious change from baseline, and additional variability in home devices impacts statistical power. The effect of coaching, setting, and equipment must be understood to use and improve home spirometry in CF.

PMID:34474987 | DOI:10.1016/j.jcf.2021.08.013

Categories: Literature Watch

Cation complexation by mucoid Pseudomonas aeruginosa extracellular polysaccharide

Thu, 2021-09-02 06:00

PLoS One. 2021 Sep 2;16(9):e0257026. doi: 10.1371/journal.pone.0257026. eCollection 2021.

ABSTRACT

Mucoid Pseudomonas aeruginosa is a prevalent cystic fibrosis (CF) lung colonizer, producing an extracellular matrix (ECM) composed predominantly of the extracellular polysaccharide (EPS) alginate. The ECM limits antimicrobial penetration and, consequently, CF sufferers are prone to chronic mucoid P. aeruginosa lung infections. Interactions between cations with elevated concentrations in the CF lung and the anionic EPS, enhance the structural rigidity of the biofilm and exacerbates virulence. In this work, two large mucoid P. aeruginosa EPS models, based on β-D-mannuronate (M) and β-D-mannuronate-α-L-guluronate systems (M-G), and encompassing thermodynamically stable acetylation configurations-a structural motif unique to mucoid P. aeruginosa-were created. Using highly accurate first principles calculations, stable coordination environments adopted by the cations have been identified and thermodynamic stability quantified. These models show the weak cross-linking capability of Na+ and Mg2+ ions relative to Ca2+ ions and indicate a preference for cation binding within M-G blocks due to the smaller torsional rearrangements needed to reveal stable binding sites. The geometry of the chelation site influences the stability of the resulting complexes more than electrostatic interactions, and the results show nuanced chemical insight into previous experimental observations.

PMID:34473773 | DOI:10.1371/journal.pone.0257026

Categories: Literature Watch

Recent Advances in the Early Treatment of Cystic Fibrosis: Bridging the Gap to Highly Effective Modulator Therapy

Thu, 2021-09-02 06:00

Pediatr Pulmonol. 2021 Sep 2. doi: 10.1002/ppul.25660. Online ahead of print.

ABSTRACT

Highly effective modulator therapy (HEMT) for cystic fibrosis (CF) has been touted as one of the greatest advances to date in CF care. As these therapies are now available for many older children and adults with CF, marked improvement of their nutritional status, pulmonary and gastrointestinal symptoms have been observed. However, most infants and younger children are not current candidates for HEMT due to age and/or cystic fibrosis transmembrane conductance regulator (CFTR) mutation. For these young children, it is essential to provide rigorous monitoring and care to avoid potential disease sequelae while awaiting HEMT availability. The following article highlights recent advances in the care of infants and young children with CF with regard to surveillance and treatment of nutritional, pulmonary, and gastrointestinal disorders. Recent clinical trials in this population are also reviewed. This article is protected by copyright. All rights reserved.

PMID:34473419 | DOI:10.1002/ppul.25660

Categories: Literature Watch

The chloride channel CFTR is not required for cyst growth in an ADPKD mouse model

Thu, 2021-09-02 06:00

FASEB J. 2021 Oct;35(10):e21897. doi: 10.1096/fj.202100843R.

ABSTRACT

Autosomal dominant polycystic kidney disease (ADPKD) is characterized by the development of bilateral renal cysts which enlarge continuously, leading to compression of adjacent intact nephrons. The growing cysts lead to a progressive decline in renal function. Cyst growth is driven by enhanced cell proliferation and chloride secretion into the cyst lumen. Chloride secretion is believed to occur mainly by the cAMP-activated cystic fibrosis transmembrane conductance regulator (CFTR), with some contribution by the calcium-activated chloride channel TMEM16A. However, our previous work suggested TMEM16A as a major factor for renal cyst formation. The contribution of CFTR to cyst formation has never been demonstrated in an adult ADPKD mouse model. We used mice with an inducible tubule-specific Pkd1 knockout, which consistently develop polycystic kidneys upon deletion of Pkd1. Cellular properties, ion currents, and cyst development in these mice were compared with that of mice carrying a co-deletion of Pkd1 and Cftr. Knockout of Cftr did not reveal any significant impact on cyst formation in the ADPKD mouse model. Furthermore, knockout of Cftr did not attenuate the largely augmented cell proliferation observed in Pkd1 knockout kidneys. Patch clamp analysis on primary renal epithelial cells lacking expression of Pkd1 indicated an only marginal contribution of CFTR to whole cell Cl- currents, which were clearly dominated by calcium-activated TMEM16A currents. In conclusion, CFTR does not essentially contribute to renal cyst formation in mice caused by deletion of Pkd1. Enhanced cell proliferation and chloride secretion is caused primarily by upregulation of the calcium-activated chloride channel TMEM16A.

PMID:34473378 | DOI:10.1096/fj.202100843R

Categories: Literature Watch

Efficacy of Problem-Solving Intervention to Improve Adherence in Adolescents and Adults with Cystic Fibrosis

Thu, 2021-09-02 06:00

Isr Med Assoc J. 2021 Sep;23(9):584-589.

ABSTRACT

BACKGROUND: Adherence to treatment by adolescents and adults with cystic fibrosis (CF) is often poor.

OBJECTIVES: To assess the impact of a focused clinical intervention on adherence in individual patients, including help in problem-solving key barriers to adherence. To implement a patient-centered problem-solving intervention using CF My Way tools. To identify and overcome a selected barrier to adherence.

METHODS: Medication possession ratios (MPRs), number of airway clearance sessions, forced expiratory volume (FEV1), body mass index (BMI), and health-related quality of life (HRQoL) were measured before and after the intervention.

RESULTS: Sixteen patients with CF, aged 23.4 ± 6.7 years, participated. MPR increased for colistimethate sodium and tobramycin inhalations from a median of 21 (range 0-100) to 56 (range 0-100), P = 0.04 and 20 (range 0-100) to 33.3 (range 25-100), P = 0.03, respectively. BMI standard deviation score rose from -0.37 to -0.21, P = 0.05. No significant improvements were found in FEV1, airway clearance, or HRQoL scores.

CONCLUSIONS: The CF My Way problem-solving intervention increased adherence to medical treatments by removing barriers directly related to the needs and goals of young adults with CF.

PMID:34472235

Categories: Literature Watch

Current and emerging artificial intelligence applications in chest imaging: a pediatric perspective

Thu, 2021-09-02 06:00

Pediatr Radiol. 2021 Sep 1. doi: 10.1007/s00247-021-05146-0. Online ahead of print.

ABSTRACT

Artificial intelligence (AI) applications for chest radiography and chest CT are among the most developed applications in radiology. More than 40 certified AI products are available for chest radiography or chest CT. These AI products cover a wide range of abnormalities, including pneumonia, pneumothorax and lung cancer. Most applications are aimed at detecting disease, complemented by products that characterize or quantify tissue. At present, none of the thoracic AI products is specifically designed for the pediatric population. However, some products developed to detect tuberculosis in adults are also applicable to children. Software is under development to detect early changes of cystic fibrosis on chest CT, which could be an interesting application for pediatric radiology. In this review, we give an overview of current AI products in thoracic radiology and cover recent literature about AI in chest radiography, with a focus on pediatric radiology. We also discuss possible pediatric applications.

PMID:34471961 | DOI:10.1007/s00247-021-05146-0

Categories: Literature Watch

The impact of cystic fibrosis on the working life of patients: A systematic review

Thu, 2021-09-02 06:00

J Cyst Fibros. 2021 Aug 29:S1569-1993(21)01352-7. doi: 10.1016/j.jcf.2021.08.011. Online ahead of print.

ABSTRACT

Advances in the treatment and management of cystic fibrosis (CF) have led to a substantial increase in patient life expectancy, thus facilitating healthier lives and labour force participation. This review aimed to address the impact of CF on the occupational functioning of patients. A significant proportion of patients were reported to retain a job on a full- or part-time schedule. Less physically demanding occupations were most frequently performed, perhaps due to CF-related inability to sustain a heavy workload. Disease severity parameters (e.g., lung function measurements, or personal, psycho-social, or economic conditions) have been reported as determinant or co-determinant factors for the development of work-related disability. Although further research is necessary, our results may be useful to inform interdisciplinary CF healthcare management, including the assessment of work function, and to define career counselling plans and workplace risk assessment and management strategies to support the personal, social and professional lives of patients.

PMID:34470710 | DOI:10.1016/j.jcf.2021.08.011

Categories: Literature Watch

"He's not fat, he just has asthma": A qualitative study exploring weight management in families living with pediatric asthma

Thu, 2021-09-02 06:00

J Asthma. 2021 Sep 1:1-12. doi: 10.1080/02770903.2021.1975739. Online ahead of print.

ABSTRACT

Objective: Children and young people living with asthma have an increased risk of overweight/obesity, leading to increased severity of asthma symptoms. Weight management has been recommended to improve asthma symptoms, however, there is limited understanding of how this is experienced or how children and young people with asthma and their families wish to be supported. The aim of this study was to explore parents and children/young people's views and experiences of managing weight while living with asthma, and to identify acceptable strategies for support. Methods: A qualitative methodological approach was taken to facilitate rich understanding of families' insights into weight management while living with asthma. In-depth interviews were conducted with nine families living with pediatric asthma (n = 9 parents, 9 young people). Data were analyzed using a Framework approach. Results: Findings indicated that family engagement with weight management behaviors was primarily influenced by perceptions of risk regarding asthma outcomes and beliefs about asthma control. Families also reported weight management engagement to be influenced by perceptions of the food environment, perceptions of the exercise environment (e.g. weather, anticipated social outcomes) and the availability of weight management support. Participants sought tailored support which gave consideration to the asthma-obesity interaction. It was suggested that this would help reduce perceptions of weight stigma in consultations, thereby supporting behavioral changes. Conclusions: Individualized weight management plans that consider families concerns about asthma-related risk are needed to manage weight in children and young people living with asthma.

PMID:34470559 | DOI:10.1080/02770903.2021.1975739

Categories: Literature Watch

Relationship between peripheral muscle strength, exercise capacity and body composition in children and adolescents with cystic fibrosis

Thu, 2021-09-02 06:00

Physiother Theory Pract. 2021 Sep 1:1-8. doi: 10.1080/09593985.2021.1973165. Online ahead of print.

ABSTRACT

STUDY DESIGN: This was a transversal analytical study.

BACKGROUND: Exercise capacity is usually decreased in cystic fibrosis, impacting the disease prognosis. As well, peripheral muscle strength and nutritional status seem to be related to exercise capacity (EC).

OBJECTIVE: To verify the relationship between peripheral muscle strength, pulmonary function and body composition with EC in children and adolescents with CF.

METHODS: The study included CF children/adolescents that were clinically stable. The disease's severity was classified according to the Schwachman-Doerschuk score. Initially the subjects underwent bioimpedance and spirometry tests. Quadriceps muscle strength (QMS) and handgrip strength (HG) were evaluated by dynamometry. The Modified Shuttle Walk Test (MSWT) was conducted along with gas analysis in order to measure EC.

RESULTS: Twenty-five children/adolescents (10.30 ± 2.33 years old) participated in the survey. 72% were eutrophic, with a mean FEV1 of 68.55%, predicted percentage of the MSWT walked distance (%WD) was 70.91%, and QMS 65.80%. QMS presented significant correlations with absolute WD (r = 0.54), oxygen peak consumption (r = 0.72), lean body mass (LM) (r = 0.83), and body mass index (BMI) (r = 0.69). HG was related with BMI (r = 0.45) and LM (r = 0.65). There was a difference in the %WD between the groups with higher/lower strength (p = .02).

CONCLUSION: There was no correlation between HG and EC in this studied sample. Early involvement of QMS was observed even in individuals with low disease severity. This finding reinforces the importance of including this QMS assessment in CF reference centers to monitor, prevent and prescribe adequate exercise training for these individuals.

PMID:34470539 | DOI:10.1080/09593985.2021.1973165

Categories: Literature Watch

Understanding the Intersection between Gender Transition and Health Outcomes in Cystic Fibrosis

Wed, 2021-09-01 06:00

Ann Am Thorac Soc. 2021 Sep 1. doi: 10.1513/AnnalsATS.202105-535RL. Online ahead of print.

NO ABSTRACT

PMID:34469707 | DOI:10.1513/AnnalsATS.202105-535RL

Categories: Literature Watch

A Randomized Trial of Antimicrobial Duration for Cystic Fibrosis Pulmonary Exacerbation Treatment

Wed, 2021-09-01 06:00

Am J Respir Crit Care Med. 2021 Sep 1. doi: 10.1164/rccm.202102-0461OC. Online ahead of print.

ABSTRACT

RATIONALE: People with cystic fibrosis (CF) experience acute worsening of respiratory symptoms and lung function known as pulmonary exacerbations. Treatment with intravenous antimicrobials is common; however, there is scant evidence to support a standard treatment duration.

OBJECTIVE: Test differing durations of intravenous antimicrobials for CF exacerbations.

METHODS: STOP2 was a multi-center, randomized, controlled, clinical trial in exacerbation among adults with CF. After 7-10-days of treatment, participants exhibiting pre-defined lung function and symptom improvements were randomized to 10- or 14-days total antimicrobial duration; all others were randomized to 14- or 21-days.

MEASUREMENTS: The primary outcome was percent predicted forced expiratory volume in 1 second (ppFEV1) change from treatment initiation to two weeks after cessation. Among early responders non-inferiority of 10-days to 14-days was tested; superiority of 21-days compared to 14-days was compared for the others. Symptoms, weight, and adverse events were secondary.

RESULTS: Among 982 randomized, 277 met improvement criteria and were randomized to 10- or 14-days treatment; the remaining 705 received 21- or 14-days. Mean ppFEV1 change was 12.8 and 13.4 for 10- and 14-days, respectively, a ‒0.65 difference [95%CI ‒3.3, 2.0], excluding the pre-defined noninferiority margin. The 21- and 14-day arms experienced 3.3 and 3.4 mean ppFEV1 changes, a difference of ‒0.10 [‒1.3, 1.1]. Secondary endpoints and sensitivity analyses were supportive.

CONCLUSIONS: Among CF adults with early treatment improvement during exacerbation, ppFEV1 after 10-days of intravenous antimicrobials is not inferior to 14-days. For those with less improvement after one week, 21-days is not superior to 14-days. Clinical trial registration available at www.clinicaltrials.gov, ID: NCT02781610.

PMID:34469706 | DOI:10.1164/rccm.202102-0461OC

Categories: Literature Watch

Cystic Fibrosis-related Diabetes is Associated with Worse Lung Function Trajectory Despite Ivacaftor Use

Wed, 2021-09-01 06:00

Am J Respir Crit Care Med. 2021 Sep 1. doi: 10.1164/rccm.202104-1060LE. Online ahead of print.

NO ABSTRACT

PMID:34469275 | DOI:10.1164/rccm.202104-1060LE

Categories: Literature Watch

Mild/Moderate Asthma Network in Italy (MANI): a long-term observational study

Wed, 2021-09-01 06:00

J Asthma. 2021 Sep 1:1-6. doi: 10.1080/02770903.2021.1968895. Online ahead of print.

ABSTRACT

OBJECTIVE: The prevalence of asthma in Italy is estimated to be around 4%; it affects approximately 2,000,000 citizens, and up to 80-90% of patients have mild-to-moderate asthma. Despite the clinical relevance of mild-to-moderate asthma, longitudinal observational data are very limited, including data on disease progression (worsening vs. improvement), the response to treatment, and prognosis. Studies are needed to develop long-term, observational, real-life research in large cohorts. The primary outcomes of this study will be based on prospective observation and the epidemiological evolution of mild and moderate asthma. Secondary outcomes will include patient-reported outcomes, treatments over time, disease-related functional and inflammatory patterns, and environmental and life-style influences.

METHODS: This study, called the Mild/Moderate Asthma Network of Italy (MANI), is a research initiative launched by the Italian Respiratory Society and the Italian Society of Allergology, Asthma and Clinical Immunology. MANI is a cluster-based, real world, cross-sectional, prospective, observational cohort study that includes 20,000 patients with mild-to-moderate asthma. (ClinicalTrials.gov Identifier: NCT04796844).

RESULTS AND CONCLUSION: Despite advances in asthma care, several research gaps remain to be addressed through clinical research. This study will add important new knowledge about long-term disease history, the transferability of clinical research results to daily practice, the efficacy of currently recommended strategies, and their impact on the burden and evolution of the disease.

ABBREVIATIONS: MANI:Mild/Moderate Asthma Network of ItalySANI:Severe Asthma Network ItalyGINA:Global Initiative for AsthmaSABA:short acting β2-agonistsICS:inhaled corticosteroidsCRF:Case Report Form.

PMID:34469268 | DOI:10.1080/02770903.2021.1968895

Categories: Literature Watch

Outcomes of infants born during the first 9 years of CF newborn screening in the United States: a retrospective Cystic Fibrosis Foundation Patient Registry cohort study

Wed, 2021-09-01 06:00

Pediatr Pulmonol. 2021 Sep 1. doi: 10.1002/ppul.25658. Online ahead of print.

ABSTRACT

INTRODUCTION: Newborn screening (NBS) for cystic fibrosis (CF) was implemented in all US states and DC by 2010. This hypothesis generating study was designed to form the basis of additional analyses and to plan quality improvement initiatives. The aims were to describe the outcomes of infants with CF born during the first 9 years of universal NBS.

METHODS: We included participants in the CF Foundation Patient Registry born 2010-2018 with age of recorded CF diagnosis 0-365 days old. We compared age of center-reported diagnosis, age at first CF event (defined as earliest sweat test, clinic visit or hospitalization), demographics, and outcomes between 3 cohorts born between 2010-2012, 2013-2015, and 2016-2018.

RESULTS: In 6354 infants, the median age at first CF event decreased from the 1st to the 3rd cohort. Weight-for-age (WFA) was < 10th percentile in about 40% of infants at the first CF Center visit. Median WFA z-score at 1-2 years was > 0 but height-for-age (HFA) z-score was < 0 through age 5-6 years. The second cohort had a higher HFA z-score than the first cohort at age 5-6 years. Pseudomonas aeruginosa infection was less common in later cohorts. About 1/3 of infants were hospitalized in the first year of life with no changes over time.

CONCLUSION: Over 9 years of CF NBS, median age at first CF event decreased. CF NBS had positive health impacts, but early life nutritional deficits and a high rate of infant hospitalizations persist. This article is protected by copyright. All rights reserved.

PMID:34469079 | DOI:10.1002/ppul.25658

Categories: Literature Watch

Clinical behavior of infection and disease caused by non-tuberculous mycobacteria in Latin America: Scoping review

Wed, 2021-09-01 06:00

Rev Peru Med Exp Salud Publica. 2021 Apr-Jun;38(2):318-325. doi: 10.17843/rpmesp.2021.382.6108. Epub 2021 Aug 30.

ABSTRACT

Reports of infection and/or disease caused by non-tuberculous mycobacteria (NTM) are becoming increasingly frequent. This scope review describes the epidemiological and clinical trend of infection/disease caused by NTM in Latin America. OVID MEDLINE, Embase and LILACS databases were explored for relevant articles. After filtering, we included 44 articles, representing an overall population of 2,826 subjects diagnosed with NTM infection and disease; the majority of the publications included subjects from Brazil and Colombia (75%), cross-sectional studies were the most common (36.6%), most subjects were male (61.3%) and the median age of subjects was 40.1 years. Disease by NTM was reported in 37 publications, extrapulmonary presentation was the most frequent (54%), main comorbidities were other pulmonary diseases, HIV, cystic fibrosis, diabetes and malnutrition, as reported in 13 studies; tuberculosis diagnosis previous to NTM disease was reported in 15 articles. Aesthetic procedures were reported in 12 articles while clinical procedures were reported in 3 articles. Several NTM species were reported, being Mycobacterium avium (52%), M. abscessus (34%), M. chelonae (18%), M. fortuitum (16%) and M. kansasii (9.1%) the most frequent. Culture and molecular testing were the main methods for diagnosis and identification. Scientific literature on NTM from Latin American countries is scarce. There is an urgent need to conduct studies on the frequency and clinical impact of NTM infections, in order to accurately identify the current morbidity and mortality associated with NTM in Latin American. It is also important to strengthen the local diagnostic capacity and the existing networks focused on studying NTM.

PMID:34468583 | DOI:10.17843/rpmesp.2021.382.6108

Categories: Literature Watch

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