Biological treatments in childhood asthma.

PURPOSE OF REVIEW: The aim is to update the information currently available for the use of biologics in severe asthma in children, in order to facilitate their prescription as far as possible. RECENT FINDINGS: The appearance of biologics for the treatment of severe asthma has meant a revolutionary change in the therapeutic approach to this disease. Currently, five biologics have been approved for severe asthma in children and/or adolescents by the regulatory agencies: omalizumab, mepolizumab, benralizumab, dupilumab and tezepelumab. But despite their positive results in terms of efficacy, there are still relevant points of debate that should induce caution when selecting the most appropriate biologic in a child with severe asthma. Indeed, safety is essential and, for several of the existing treatments, the availability of medium-term to long-term data in this regard is scarce. SUMMARY: The use of biologics can facilitate the therapeutic paradigm shift from pleiotropic treatments to personalized medicine. However, the choice of the most appropriate biologics remains a pending issue. On the other hand, to the extent that several of the biologics have been available for a relatively short time, the most robust evidence in terms of efficacy and safety in children is that of omalizumab.

Cost-effectiveness of omalizumab for the treatment of severe pediatric allergic asthma-Results of a real-life study in Spain.

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 (Asthma iN CHildren: Omalizumab in Real-life in Spain) 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 on health encounters and drug consumption before and up to 6 years after the beginning of the treatment with omalizumab. RESULTS: The ICER per avoided MSE was €2107 after 1 year, and it consistently decreased to €656 in those followed up to 6 years. Similarly, the ICER for the minimally important difference in control tests showed a decrease from €2059 to €380 per each 0.5 points of improvement in ACQ5 and from €3141 to €2322 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, especially those who have frequent exacerbations; the costs are progressively reduced in successive years of treatment.

Grado de información percibida por los tutores legales en el Consentimiento Informado en el servicio de Neumología y Alergia Infantil / Grau d'informació percebuda pels tutors legals en el Consentiment Informat de proves de provocació a Al·lèrgia Pediàtrica / Degree of information perceived by legal guardians in the Informed Consent of challenge tests in Pediatric Allergy

Introduction: Knowledge about the degree of information perceived by the legal guardians from the Informed Consent (IC) in pediatric setting is scarce. Objectives: To evaluate the degree of information perceived by legal guardians in the IC for challenge tests in Pediatric Allergy. Methods and Materials: It is a descriptive, observational and cross-sectional study of information assessment through a questionnaire, in the period April-May 2021. The questionnaire was developed by the author and his team on the base of a publicly available one. A descriptive and analytical statistical study was carried out using the SPSS 15.0 software. Results: Ninety-nine tutors with a mean age of 41 years were recruited, with 52% university, 27% professional, 17% secondary and 4% primary education. More than 70% of the sample positively rated the degree of information provided in relation to the procedure, its objective, its risk-benefit, and the dedication and decision time. 50% of the tutors negatively rated the information provided regarding alternative treatment, the possibility of denying and revoking the IC. The internal consistency of the questionnaire was very good, with a Cronbach's alpha value of 0.88. No correlation was found between the degree of perception and several demographic or clinical variables. Conclusions: The degree of information perceived by the tutors is very good in general. The information in relation to the alternative procedures, the possibility of rejection and revocation of the IC can be improved.

Introducción: Existe escasa información sobre el grado de información percibida por los tutores legales en el Consentimiento informado (CI) en el ámbito pediátrico. Objetivos: Evaluar el grado de información percibida por los tutores legales en el CI de pruebas de provocación en Alergia Pediátrica. Métodos y Materiales: Estudio descriptivo, observacional y transversal de valoración de información mediante cuestionario, en el período de abril-mayo de 2021. El cuestionario fue desarrollado por el autor y su equipo, tomando como base un cuestionario disponible en la literatura. Se realizó estudio estadístico descriptivo y analítico mediante el software SPSS 15.0. Resultados: Se reclutaron 99 tutores con una edad media de 41 años, con formación universitaria 52%, profesional 27%, secundaria 17% y primaria 4%. Más del 70% de la muestra calificó positivamente el grado de información proporcionada en relación al procedimiento, su objetivo, su riesgo-beneficio y el tiempo de dedicación y de decisión. Un 50% de los tutores calificó negativamente la información proporcionada en cuanto al tratamiento alternativo, posibilidad de negar y revocar el CI. La consistencia interna del cuestionario fue muy buena, con un valor de la alfa de Cronbach de 0.88. No se encontró correlación entre el grado de percepción y diversas variables demográficas o clínicas. Conclusiones: El grado de información percibida por los tutores es en general muy bueno. Las informaciones en relación a los procedimientos alternativos, la posibilidad de rechazo y revocación del CI fueron los puntos mejorables.(AU)

Introducció: Hi ha escassa informació sobre el grau d'informació percebuda pels tutors legalsal Consentiment informat (CI) a l'àmbit pediàtric. Objectius: Avaluar el grau d'informació percebuda pels tutors legals al CI de proves de provocació a Al·lèrgia Pediàtrica. Mètodes i Materials: Estudi descriptiu, observacional i transversal de valoració d'informació mitjançant un qüestionari, en el període d'abril-maig de 2021. El qüestionari va ser desenvolupat per l'autor i el seu equip, prenent com a base un qüestionari disponible a la literatura. Es va realitzar estudi estadístic descriptiu i analíticmitjançant el programari SPSS 15.0. Resultats: Es van reclutar 99 tutors amb una edat mitjana de 41 anys, amb formació universitària 52%, professional 27%, secundària 17% i primària 4%. Més del 70% de la mostra va qualificar positivament el grau d'informació proporcionada en relació amb el procediment, el seu objectiu, el risc-benefici i el temps de dedicació i de decisió. Un 50% dels tutors va qualificar negativament la informació proporcionada pel que fa al tractament alternatiu, possibilitat de negar i revocar el CI. La consistència interna del qüestionari va ser molt bona, amb un valor de l'alfa de Cronbach de 0,88. No es va trobar correlació entre el grau de percepció i diverses variables demogràfiques o clíniques. Conclusions: En general, el grau d'informació percebuda pels tutors és molt bo. Les informacions en relació amb els procediments alternatius, la possibilitat de rebuig i revocació del CI van ser els punts millorables.(AU)

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

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.