Omalizumab has been shown to reduce exacerbation rates in moderate to severe allergic asthma. Our aim was to evaluate omalizumab efficacy and safety in a real-life setting in severe asthmatic ...children. 104 children (aged 6-18 years), followed up in paediatric pulmonary tertiary care centres, were included at the beginning of omalizumab treatment. Asthma control levels, exacerbations, inhaled corticosteroid dose, lung function and adverse events were evaluated over 1 year. Children were characterised by allergic sensitisation to three or more allergens (66%), high IgE levels (mean 1125 kU · L(-1)), high rate of exacerbations (4.4 per year) and healthcare use during the previous year, and high inhaled corticosteroid dose (mean 703 μg equivalent fluticasone per day). Asthma control levels defined as good, partial or poor, improved from 0%, 18% and 82% at entry to 53%, 30% and 17% at week 20, and to 67%, 25% and 8% at week 52, respectively (p<0.0001). Exacerbation and hospitalisation rates dropped by 72% and 88.5%, respectively. At 12 months, forced expiratory volume in 1 s improved by 4.9% (p=0.023), and inhaled corticosteroid dose decreased by 30% (p<0.001). Six patients stopped omalizumab for related significant adverse events. Omalizumab improved asthma control in children with severe allergic asthma and was generally well tolerated. The observed benefit was greater than that reported in clinical trials.
Inorganic antigens may contribute to paediatric sarcoidosis. Thirty-six patients matched with 36 healthy controls as well as a group of 21 sickle-cell disease (SCD) controls answered an environmental ...questionnaire. Patients' indirect exposure to inorganic particles, through coresidents' occupations, was higher than in healthy and SCD controls (median score: 2.5 (0.5-7) vs 0.5 (0-2), p=0.003 and 1 (0-2), p=0.012, respectively), especially for construction, exposures to metal dust, talc, abrasive reagents and scouring products. Wood or fossil energies heating were also linked to paediatric sarcoidosis. This study supports a link between mineral environmental exposure due to adult coresident occupations and paediatric sarcoidosis.
Pulmonary alveolar proteinosis (PAP) is a rare form of chronic interstitial lung disease, characterised by the intra-alveolar accumulation of lipoproteinaceous material. Numerous conditions can lead ...to its development. Whereas the autoimmune type is the main cause in adults, genetic defects account for a large part of cases in infants and children. Even if associated extra-respiratory signs may guide the clinician during diagnostic work-up, next-generation sequencing panels represent an efficient diagnostic tool. Exome sequencing also allowed the discovery of new variants and genes involved in PAP. The aim of this article is to summarise our current knowledge of genetic causes of PAP.
Hyperventilation syndrome in children with asthma Beauvais, Manon; Taam, Rola Abou; Neuraz, Antoine ...
The Journal of asthma,
11/2023, Letnik:
ahead-of-print, Številka:
ahead-of-print
Journal Article
Recenzirano
Odprti dostop
Hyperventilation syndrome (HVS) may be associated with asthma. In the absence of a gold standard diagnosis for children, its impact on asthma has been rarely assessed.
To assess the impact of HVS on ...the symptoms and lung function of children with asthma and determine the diagnostic value of the Nijmegen questionnaire in comparison to a hyperventilation test (HVT).
Data from asthmatic children followed in the department of Pediatric Pulmonology of Necker Hospital and explored for HVS were retrospectively analyzed. HVS was diagnosed by a positive HVT. Asthma exacerbations, control and lung function were assessed in children with or without a positive HVT. The sensitivity and specificity of the Nijmegen questionnaire were determined relative to the positivity of a HVT. The Nijmegen questionnaire threshold was ≥23.
Data from 112 asthmatic children, median age 13.9 years 11.6-16, were analyzed. Twenty-eight children (25%) had mild or moderate asthma and 84 (75%) severe asthma. The HVT was performed on 108 children and was negative for 34 (31.5%) and positive for 74 (68.5%). The number of asthma exacerbations in the past 12 months, Asthma Control Test (ACT) score, and lung function did not differ between children with a positive HVT and a negative HVT. The Nijmegen questionnaire was administered to 103 children. Its sensitivity was 56.3% and specificity 56.3%.
The symptoms and lung function of adolescents with asthma are not affected by the presence of HVS. The sensitivity and specificity of the Nijmegen questionnaire are low.
Severe asthma (SA) affects less than 5% of the pediatric asthma population but is considered to account for approximately half of total pediatric asthma healthcare costs. Allergic comorbidities, ...including food allergies (FA) and allergic rhinitis (AR), are frequent in children with SA (1). The presence of FA and AR increases asthma severity (1-6) and medication use (4-6).
Primary ciliary dyskinesia (PCD) is a rare airway disorder caused by defective motile cilia. Only male patients have been reported with pathogenic mutations in X-linked
, which result in the absence ...of ciliary dynein arms, whereas their heterozygous mothers are supposedly healthy. Our objective was to assess the possible clinical and ciliary consequences of X-chromosome inactivation (XCI) in these mothers.
XCI patterns of six mothers of male patients with
-related PCD were determined by DNA-methylation studies and compared with their clinical phenotype (6/6 mothers), as well as their ciliary phenotype (4/6 mothers), as assessed by immunofluorescence and high-speed videomicroscopy analyses. The mutated X chromosome was tracked to assess the percentage of cells with a normal inactivated
allele.
The mothers' phenotypes ranged from absence of symptoms to mild/moderate or severe airway phenotypes, closely reflecting their XCI pattern. Analyses of the symptomatic mothers' airway ciliated cells revealed the coexistence of normal cells and cells with immotile cilia lacking dynein arms, whose ratio closely mirrored their XCI pattern.
This study highlights the importance of searching for heterozygous pathogenic
mutations in all female relatives of male PCD patients with a
defect, as well as in females consulting for mild chronic respiratory symptoms. Our results also demonstrate that about one-third-ranging from 20% to 50%-normal ciliated airway cells sufficed to avoid severe PCD, a result paving the way for gene therapy.
Hymenoptera venom allergy is the second cause of anaphylaxis in European children. Systemic reactions occur in up to 0.8% of schoolchildren. Venom immunotherapy (VIT) is recommended in sensitized ...children having systemic skin reactions exceeding generalized skin symptoms. VIT carries an 8–20% risk of systemic adverse events. Short protocols achieve the maintenance dose faster than conventional protocols but are more frequently associated with anaphylactic reactions. We report the cases of teenagers who experienced severe anaphylactic reactions (SAR) during the rush phase of VIT (Table 1). After an informed consent, a second rush VIT was performed after a pretreatment with omalizumab and was tolerated. No injection of omalizumab was required during the maintenance phase.
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