BACKGROUND Severe Pediatric Allergic Asthma (SPAA) induces a huge economic burden in terms of direct, indirect and intangible costs. The use of omalizumab for the treatment of these patients has produced a significant improvement in several clinical outcomes, but at the same time, the cost for the management of the disease has also increased. The aim of this report was to evaluate whether the use of omalizumab is cost-effective. METHODS A sample of 426 children with SPAA from the ANCHORS study was used to calculate the Incremental Cost Effectiveness Ratio (ICER) for the avoidance of Moderate to Severe Exacerbations (MSE), and also for the improvement in childhood Asthma Control Test (c-ACT) or the Asthma Control Questionnaire (ACQ5). We retrospectively collected data of health encounters and drug consumption before and up to six years after the beginning of the treatment with omalizumab. RESULTS The ICER per avoided MSE was \euro2,107 after one year, and it consistently decreased to \euro656 in those followed up to six years. Similarly, the ICER for the Minimally Important Difference in control tests showed a decrease from \euro2,059 to \euro380 per each 0.5 points of improvement in ACQ5, and from \euro3,141 to \euro2,322 per each 3 points improvement in c-ACT, at years 1 and 6 respectively. CONCLUSION The use of OMZ is a cost-effective option for most children with uncontrolled SPAA, mainly those who have frequent exacerbations, showing progressively reduced costs in successive years of treatment.

The receptor for advanced glycation end products (RAGE) has been studied in several respiratory diseases described as an important inflammatory mediator. The RAGE-axis is activated by multiple endogenous ligands related to pro-inflammatory states, upregulate the RAGE expression. The function of soluble RAGE (sRAGE) is not completely understood, it has been hypothesized an anti-inflamatory role as RAGE decoy receptor. Few studies have explored the RAGE-axis in Cystic Fibrosis (CF) with contradictory results. Based on previously, we present this pilot study with the aim of describe the plasma sRAGE levels in children with cystic CF (CFp), compare with the sRAGE levels in a healthy cohort and study its possible correlation with CFp clinical features. We conducted a single-center, cross-sectional observational study. We included 35 clinically stable CF patients (aged < 18 years). The median plasma sRAGE level in CFp was 1494,75 pg/ml [interquartile range (IQR) 708,75pg/ml], compared with 714,20 pg/ml (IQR 490,50 pg/ml)) in the historical cohort of healthy controls (p < 0,001). A positive correlation was found between plasma sRAGE level and forced expiratory volume in 1 second/forced vital capacity ratio (FEV1/FVC) (p 0,004) and forced expiratory flow between 25% and 75% (FEF25%-75%) (p 0,032). In this preliminary study, the plasma sRAGE level were higher in CFp than in healthy controls. Also, we described a positive correlation between FEV1/FVC and FEF25%-75% and plasma sRAGE. To our knowledge, our study is the largest to describe plasma sRAGE values ​​in CFp and the only one carried out in pediatric CF population.

Introduction. Exercise intolerance is common in chronic respiratory diseases (CRD), but its mechanisms are still poorly understood. The aim of this study was to evaluate exercise capacity and its association with lung function, ventilatory limitation, and ventilatory efficiency in children and adolescents with cystic fibrosis (CF) and asthma when compared to healthy controls. Methods. Cross‐sectional study including patients with mild‐to‐moderate asthma, CF and healthy children and adolescents. Anthropometric data, lung function (spirometry) and exercise capacity (cardiopulmonary exercise testing) were evaluated. Primary outcomes were peak oxygen consumption (VO 2peak), forced expiratory volume in 1 second (FEV 1), breathing reserve (BR), ventilatory equivalent for oxygen consumption (V E/VO 2) and for carbon dioxide production (V E/VCO 2), both at the ventilatory threshold (VT 1) and peak exercise. Results. Mean age of 147 patients included was 11.8±3.0 years. There were differences between asthmatics and CF children when compared to their healthy peers for anthropometric and lung function measurements. Asthmatics showed lower VO 2peak when compared to both healthy and CF subjects, although no differences were found between healthy and CF patients. A lower BR was found when CF patients were compared to both healthy and asthmatic. Both CF and asthmatic patients presented higher values for V E/VO 2 and V E/VCO 2 at VT 1 when compared to healthy individuals. For both V E/VO 2 and V E/VCO 2 at peak exercise CF patients presented higher values when compared to their healthy peers. Conclusion. Patients with CF achieved good exercise capacity despite low ventilatory efficiency, low BR, and reduced lung function. However, asthmatics reported reduced cardiorespiratory capacity and normal ventilatory efficiency at peak exercise. These results demonstrate differences in the mechanisms of ventilatory limitation to maximum exercise testing in children and adolescents with CRD.

Background: Various studies have assessed omalizumab outcomes in the clinical practice setting but follow-up and/or number of patients included were limited. We aim to describe the long-term outcomes of pediatric patients with severe persistent allergic asthma receiving omalizumab in the largest real-life cohort reported to date. Methods: ANCHORS was a multicenter, observational, retrospective cohort study conducted in 25 Pediatric Allergy and Pulmonology units in Spain. We collected data of patients <18 years and initiating omalizumab between 2006-2018, from the year prior to omalizumab initiation to discontinuation or last available follow-up. The primary outcome was the evolution of the annual number of moderate-to-severe exacerbations compared to the baseline period. Results: Of the 484 patients included, 101 (20.9%) reached six years of treatment. The mean±standard deviation number of exacerbations decreased during the first year of treatment (7.9±6.6 to 1.1±2.0, p<0.001) and remained likewise for up to six years. The other clinical parameters assessed also improved significantly during the first year and stabilized or continued to improve thereafter. The percentage of patients experiencing adverse events was consistently low, and the main reason for discontinuation was good disease evolution. Conclusion: In this large, long-term, observational study, moderate-to-severe exacerbations decreased significantly from the first year of treatment with omalizumab. The beneficial effect was maintained in the long-term, along with a good safety profile. Our results position omalizumab as an effective long-term treatment in pediatric patients with severe persistent allergic asthma.