Omalizumab is the first biological therapy used to treat moderate-to-severe asthma and certainly the one with the highest number of publications.
A systematic review and meta-analysis were performed ...to examine two critical outcomes of omalizumab therapy, asthma exacerbation rate, the reduction of the use of inhaled corticosteroids (ICS), and the improvement of the lung function as a secondary outcome using the following keywords in the MEDLINE database: "anti-IgE, severe asthma, children, and randomized controlled trial." We specifically selected papers that included moderate-to-severe asthma patients and collected data on children and adolescents.
Four RCT studies (total number of patients = 1,239) were included in the analysis. The reported data on exacerbations showed an overall improvement in the exacerbation rate with a decreased use of inhaled steroids and some other minimal clinically important difference (MCID).
Our systematic review confirms the known findings that omalizumab therapy decreases asthma exacerbation rate and reduces background therapy inhaled steroid dose. Therefore, add-on therapy with omalizumab shows a good efficacy and safety profile, thus proving to be a useful additional therapeutic option.
https://www.crd.york.ac.uk/prospero/, identifier: CRD42023396785.
Lung function is an important tool in the diagnosis and monitoring of patients with asthma at all ages. Airway obstruction is a typical feature of asthma and it can be assessed with several lung ...function techniques. Spirometry, respiratory resistance and reactance, and lung volumes are available to measure it at different ages and in children. The assessment of a bronchodilator response is always recommended to show the reversibility of the obstruction. Poor lung function is a predictor of poor asthma outcome and a low Forced Expiratory Volume in the first second of expiration percent predicted measured with spirometry, has been shown to be associated with a higher risk of having an exacerbation during the following year independently of the presence of asthma symptoms. In severe asthma lung function assessment is used to distinguish different phenotypes, children with severe asthma have worse airflow limitation prior to administration of a bronchodilator than children with non severe asthma. Airway resistance and reactance are indirect measurements of airway obstruction and they can be measured with the forced oscillation technique, which is feasible also in non-collaborative children. This technique can be more informative in discriminating patients with asthma from healthy controls and is able to indicate a more peripheral involvement of the airways. The role of this technique in severe asthma is still debated. In conclusion lung function is useful in the clinical management of children with severe asthma.
We assessed lung function and respiratory health in an area-based prospective cohort of preschool children born very preterm.
Lung function was measured by interrupter respiratory resistance (Rint) ...and forced oscillation technique (FOT) (respiratory resistance (Rrs8), reactance (Xrs8), and area under the reactance curve (AX)) at a median age of 5.2 years in a cohort of 194 children born at 22-31 weeks of gestational age (GA) in Tuscany, Italy. Respiratory symptoms and hospitalizations were also assessed.
Mean (SD) lung function Z-scores were impaired in preterm children for Rint (0.72 (1.13)), Xrs8 (-0.28 (1.34)), and AX (0.29 (1.41)). However, only a relatively small proportion of children (14.5-17.4%) had values beyond the 95th centile or below the 5th. Children with bronchopulmonary dysplasia (BPD) (n = 24) had slightly but not significantly impaired lung function indices in comparison with those without BPD (n = 170). In a multivariable analysis, lower GA was associated with worse lung function indices. Fifty-five percent of children had a history of wheezing ever and 21% had been hospitalized in their lifetime because of lower respiratory infections; 31% had wheezing in the last 12 months and this was associated with increased Rrs8 (P = 0.04) and AX (P = 0.08), and with decreased Xrs8 (P = 0.04) Z-scores.
Irrespectively of BPD preschool children born very preterm had impaired lung function indices, as measured by Rint and FOT, and a slightly higher burden of respiratory problems than the general population. GA seems to be crucial for lung development.
Introduction
There are no recent data on primary ciliary dyskinesia (PCD) distribution, diagnosis and treatment in Italy.
Methods
A descriptive study based on a survey questionnaire. It consisted of ...three sections (patients, diagnosis, and treatment), and sent to all the Italian PCD Centers.
Results
Questionnaires obtained from 20/22 centers in 12/20 regions showed that the total number of PCD patients treated at the participating centers was of 416. Out of all centers, 55% follow <20 patients, two centers have >40 patients, and 75% follow both pediatric and adults. Age at diagnosis was between 4 and 8 years in 45% of the centers, <3 years in three centers. Nasal nitric oxide, transmission electron microscopy and ciliary high‐speed video microscopy are performed in 75%, 90%, and 40% of centers, respectively. Immunofluorescence is available in five centers. Genetic analysis is offered in 55% of the centers, and in seven centers >50% of the patients have a known genetic profile. Patients treated at all centers receive inhaled saline solutions, corticosteroids and chest physiotherapy. Prophylactic antibiotics and mucolytics are prescribed in 95% and 50% of the centers, respectively. Pseudomonas infection is treated with oral or inhaled antibiotics.
Conclusions
Many Italian centers care for a small number of pediatric and adult patients, and diagnosis is often delayed. We found a great variability in the available diagnostic procedures, as well in the prescribed therapies. Our study will help to uniform diagnostic algorithm and share treatments protocols for PCD in Italy and allowed to set specific national goals.
Tubulin, one of the most abundant cytoskeletal building blocks, has numerous isotypes in metazoans encoded by different conserved genes. Whether these distinct isotypes form cell type- and ...context-specific microtubule structures is poorly understood. Based on a cohort of 12 patients with primary ciliary dyskinesia as well as mouse mutants, we identified and characterized variants in the
isotype that specifically perturbed centriole and cilium biogenesis. Distinct
variants differentially affected microtubule dynamics and cilia formation in a dominant-negative manner. Structure-function studies revealed that different TUBB4B variants disrupted distinct tubulin interfaces, thereby enabling stratification of patients into three classes of ciliopathic diseases. These findings show that specific tubulin isotypes have distinct and nonredundant subcellular functions and establish a link between tubulinopathies and ciliopathies.
Although pulmonary function testing plays a key role in the diagnosis and management of chronic pulmonary conditions in children under 6 years of age, objective physiologic assessment is limited in ...the clinical care of infants and children less than 6 years old, due to the challenges of measuring lung function in this age range. Ongoing research in lung function testing in infants, toddlers, and preschoolers has resulted in techniques that show promise as safe, feasible, and potentially clinically useful tests. Official American Thoracic Society workshops were convened in 2009 and 2010 to review six lung function tests based on a comprehensive review of the literature (infant raised-volume rapid thoracic compression and plethysmography, preschool spirometry, specific airway resistance, forced oscillation, the interrupter technique, and multiple-breath washout). In these proceedings, the current state of the art for each of these tests is reviewed as it applies to the clinical management of infants and children under 6 years of age with cystic fibrosis, bronchopulmonary dysplasia, and recurrent wheeze, using a standardized format that allows easy comparison between the measures. Although insufficient evidence exists to recommend incorporation of these tests into the routine diagnostic evaluation and clinical monitoring of infants and young children with cystic fibrosis, bronchopulmonary dysplasia, or recurrent wheeze, they may be valuable tools with which to address specific concerns, such as ongoing symptoms or monitoring response to treatment, and as outcome measures in clinical research studies.
Aims: The study aimed to determine how childhood asthma is managed in Western Australia by general practitioners (GPs) and specialist paediatricians.
Methods: A questionnaire survey was sent to 992 ...GPs and specialist paediatricians, asking about practice and preferences regarding maintenance management of childhood asthma and treatment of acute asthma. Questions about asthma in infants, pre‐school and school‐aged children were asked separately.
Results: The overall response rate was 24.7%, with 188/878 (21.4%) of GPs and 44/62 (71.0%) of paediatricians returning the questionnaire. The decision to start maintenance therapy was generally based on symptom frequency and severity. The first choice for maintenance treatment in all age groups was inhaled corticosteroids (ICS). The second most common treatment varied according to age group, with short‐acting β2‐agonist (SBA) preferred for infants, montelukast or short‐acting β2‐agonist for pre‐schoolers and combination therapy (ICS + long action β2‐agonist) for school‐aged children. Objective monitoring of lung function with peak flow or spirometry, was used by 40% of GPs and 59% of paediatricians. Acute asthma was primarily managed with inhaled salbutamol and oral corticosteroids. There were few differences in treatment choice between GPs and paediatricians. Many GPs indicated that they did not treat asthma in infants without specialist consultation.
Conclusions: These data show good compliance by the minority of GPs responding to the survey and by paediatricians practising in Western Australia with current Australian asthma management guidelines. Major differences in treatment preferences between the groups were not detected.
Lung Function Tests in Preschool Children Fenu, Grazia; Calogero, Claudia; Lombardi, Enrico
Turkish Thoracic Journal,
10/2015, Letnik:
16, Številka:
4
Journal Article
Recenzirano
Odprti dostop
The measurement of lung function by spirometry is routinely used to monitor and adequately treat children with asthma. The assessment and evaluation of lung function in children aged 3-5 years has ...been neglected for a long time because of the difficulty to perform forced expiratory maneuvers. However, the use of techniques such as the interrupter technique and the forced oscillation technique, which only require passive collaboration and where the only request to the child is to breathe at tidal volume, has overcome this limitation. Other techniques such as the measurement of specific airway resistance by plethysmography or the measurement of the lung clearance index using the multiple-breath washout might be helpful in this regard, although these techniques are less standardized in preschool children.
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