Omalizumab outcomes for up to 6 years in pediatric patients with severe persistent allergic asthma

Abstract

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 and 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 with the baseline period. Results Of the 484 patients included, 101 (20.9%) reached 6 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 < .001) and remained likewise for up to 6 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.

Clinical Experience with Anti-IgE Monoclonal Antibody (Omalizumab) In Paediatric Severe Allergic Asthma – A Romanian Perspective

“…In our study group, we observed a slight decrease in the mean counts of absolute eosinophil cells after one year of treatment. The effect seems to be correlated with the duration of treatment, the lowest value compared to baseline was recently reported in a study after 6 years of treatment [36]. The pulmonary function evaluated by both large (FEV1) and small (MEF50) airway functional parameters, improved over one year of treatment with Omalizumab.…”

“…Three pivotal Phase III clinical trials in patients with moderate-to-severe asthma were performed in the USA and Europe on a population of 1071 ICS-dependent symptomatic adolescents and adults, and 334 pediatric patients (aged 6-12 years), followed by other 25 trials, enrolling a total of 6382 patients with uncontrolled allergic asthma analysed a Cochrane meta-analysis, resulted all in effective reduction of asthma exacerbations by omalizumab treatment [32][33]. Recent systematic reviews of the results of multiple randomized, blinded, placebo-controlled phase III studies involving children 6 to 11 years of age [34] or adults and children over 6 years old age [35,36] resulted also in effective improvement in asthma control by reducing asthma exacerbations, hospital admissions, acute asthma attacks and the related need of oral corticosteroid (OCS) in severe asthmatic children. In our study, we observed a statistically significant reduction of the rate of exacerbations after the first year of treatment (mean rate of severe exacerbations decreased from 4.1 (2.8 SD) per patient during the previous year to 1.15 (0.78 SD) during the first year of treatment (p<0.0001)) and decrease of number of patients who developed asthma exacerbation, with less asthma acute symptoms induced by exercise (2 from 12 patients) and no exacerbations induced by viral respiratory infections during the first year of treatment with Omalizumab.…”

“…It is important to note that the degree of improvement of exacerbations rate has been correlated in clinical trials with the number of exacerbations before starting the treatment with Omalizumab, baseline pulmonary function or eosinophil count, with different results in different studies, improvements being greater in children with more severe subtypes [41]. An elevated eosinophil count is associated with higher risk of asthma exacerbations [1,36]. It is important to note that 91.7% of the patients in our study were polysensitized, a trait that, together with the increase in the number of circulating eosinophils, high levels of total IgE, are parameters related to the severity of asthma and it was suggested that these characteristics may be relevant for a subpopulation of serious, true, highly allergic asthma, with a good response to Omalizumab (37).…”

Clinical Experience with Anti-IgE Monoclonal Antibody (Omalizumab) in Pediatric Severe Allergic Asthma—A Romanian Perspective

“…Three pivotal Phase III clinical trials in patients with moderate to severe asthma were performed in the USA and Europe on a population of 1071 ICS-dependent symptomatic adolescents and adults, and 334 children (aged 6–12 years), followed by 25 trials enrolling a total of 6382 patients with uncontrolled allergic asthma, were analyzed in a Cochrane meta-analysis, which demonstrated effective reduction of asthma exacerbations by Omalizumab treatment [ 31 , 32 ]. Recent systematic reviews of the results of multiple randomized, blinded, placebo-controlled phase III studies involving children aged 6 to 11 years [ 33 ] or adults and children aged over 6 years [ 33 , 34 ] also confirmed effective improvement in asthma control by Omalizumab, with reduction in asthma exacerbations, hospital admissions, acute asthma attacks and the related need for oral corticosteroid (OCS) in children with severe asthma. In our study, a statistically significant reduction in the rate of exacerbations during the first year of treatment was observed; the mean rate of severe exacerbations decreased from 4.1 ± 2.8 per patient during the previous year to 1.15 ± 0.78 during the first year of treatment ( p < 0.0001), and there was a decrease in the number of patients who developed asthma exacerbation, with less acute asthma symptoms induced by exercise (2 out 12 patients) and no exacerbations induced by viral respiratory infections during the first year of treatment with Omalizumab.…”

“…It is also important to note that the degree of decrease in the exacerbation rate has been correlated in clinical trials with the number of exacerbations before starting the treatment with Omalizumab, baseline PFTs, and eosinophil count, with different results in different studies, but with improvements being greater in children with more severe subtypes [ 38 ]. A raised absolute eosinophil count in peripheral blood is associated with a higher risk of asthma exacerbations [ 1 , 33 ]. In our study, 91.7% of the patients were polysensitized, a trait that, together with the high eosinophil counts and the increased and high levels of total IgE, are parameters related to the severity of asthma.…”