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Expert meeting report : towards a joint European roadmap to address the unmet needs and priorities of paediatric asthma patients on biologic therapy
Kornel Golebski, Uroš Potočnik, 2021, review article

Abstract: Biologics use in severe paediatric asthma The global prevalence of severe asthma among adolescents ranges from 4% to 11%; and up to 7% of children with asthma display an uncontrolled and severe form that is often associated with a substantial burden on the quality of life of patients and their families, and increasing costs of healthcare [1, 2]. “Childhood asthma” is an umbrella term describing a heterogeneous disease comprising different phenotypes and a wide range of symptoms [3–5]. Despite decades of basic and clinical research, tailored strategies to modify the natural course of asthma, prevent severe exacerbations and inhibit lung function decline are still lacking. In addition, clinical phenotypes are only moderately reliable in the prediction of treatment responses and our current understanding of asthma endotypes is limited. Most asthma endotypes involve concomitant inflammatory pathways and distorted immune parameters. Advances in understanding severe paediatric asthma pathophysiological mechanisms and immunological pathways mediating the airway inflammation would allow better characterisation of these patients as well as optimised intervention, guided by treatable traits and biomarkers [6, 7]. Recent studies have demonstrated the effectiveness of monoclonal antibodies (mAbs), also known as biologics, targeting type 2 inflammation in controlling the symptoms of severe asthma. Currently, four human mAbs are approved for use in children: mAbs that target interleukin (IL)-5 or IL-5 receptor (R) (mepolizumab and benralizumab), mAbs that target IL-4R (dupilumab), and mAbs that target immunoglobulin E (omalizumab). Omalizumab was the first biologic approved to treat moderate-to-severe allergic asthma (≥6 years of age). Mepolizumab and dupilumab have been approved for severe eosinophilic asthma (≥6 and ≥12 years of age, respectively), while benralizumab has been approved in the USA to treat children (≥12 years of age) with severe eosinophilic asthma [8–13]. The introduction of mAb agents in asthma treatment is a milestone in the application of personalised medicine. However, comparative studies and standardised algorithms for the management of paediatric severe asthma to guide the best therapeutic option for paediatric patients with severe asthma are lacking [14]. More personalised medicine approaches may benefit the patient by better matching patients with the most appropriate therapy. Risk stratification, remote monitoring and the integration of multiple data sources could help tailor management for the individual child with severe asthma. A digital multidisciplinary European expert meeting took place on 9 July 2020. In this workshop, we brought together European respiratory/allergy paediatricians, immunologists, epidemiologists and basic scientists to identify the unmet needs of paediatric severe asthma patients, and set the priorities for clinical and research activities ahead. The participants discussed ongoing initiatives and knowledge gaps, and formulated proposals on how to address these challenges. In this report, we describe the main findings of this expert meeting.
Keywords: asthma, paediatric asthma, severe asthma, children, biologics, monoclonal antibodies, biologic therapy, therapy
Published in DKUM: 14.08.2024; Views: 84; Downloads: 4
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Abstract: Background: The prevalence of paediatric hypertension is increasing worldwide, especially due to the childhood obesity epidemic, and is an important public-health concern. While the Health-Related Quality of Life (HRQoL) was already shown to be impaired in the adult hypertensive population, a scarcity of data still exists on HRQoL in paediatric hypertensive patients. Our purpose was thus to assess the HRQoL of children and adolescents with arterial hypertension, using self- and proxy-reports, and to determine the correlations between child and parent questionnaire scores. Methods: The Paediatric Quality of Life Inventory™ 4.0 Generic Core Scales were administered via post to children and adolescents, aged 5-18 years, with primary or secondary arterial hypertension and parents as proxy-reports. Patients were recruited from a paediatric nephrology unit in a tertiary hospital, using an out-patient clinic visit registry. Healthy school children and adolescents from a local primary school, aged 6 to 15 years, and their parents formed the control group. HRQoL group comparisons were calculated with independent samples t-test and child-parent correlations with the Pearson’s r correlation coefficient. Results: In total we recruited 139 patient and 199 control group participants as self- and proxy-reports. Scores from self- as well as proxy-reports indicated a significantly lower overall HRQoL in the paediatric hypertensive population (95% CI for mean score difference: − 11.02, − 2.86 for self- and − 10.28, − 2.67 for proxy-reports; p = .001). In self-reports, lower physical (95% CI: -13.95, − 4.89; p = <.001), emotional (95% CI: -12.96, − 2.38; p = .005), school (95% CI: -11.30, − 0.42; p = .035), and psychosocial functioning scores were observed (95% CI: -10.34, − 1.89; p = .005). Parent proxy-reports were lower in physical (95% CI: -14.31, − 5.39; p = <.001), emotional (95% CI: -12.39, − 2.60; p = .003) and psychosocial scores (95% CI: -9.36, − 1.34; p = .009). Pearson’s r values ranged between 0.62 to 0.79 in patient and 0.56 to 0.80 in control sample (p < .001). Interestingly, hypertensive children reported lower social functioning scores than hypertensive adolescents (p < .001). Conclusions: This cross-sectional study gives insight into the detrimental impact of hypertension on children’s and adolescents HRQoL, which may inform public health experts. Furthermore, it shows that clinicians should aim to improve patients’ physical and psychosocial well-being throughout their development.
Keywords: arterial hypertension, paediatric, health-related quality of life, PedsQL
Published in DKUM: 26.10.2018; Views: 1783; Downloads: 165
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