Thesis in a nutshell – Celeste Ewig
Asthma is one of the most prevalent childhood chronic conditions affecting more than 4.7 million children and adolescents in the United States. While asthma is largely referred to as a singular disease, advances in our understanding of inflammatory pathways have led to the recognition of asthma as a heterogeneous condition with unique immunopathogenic pathways and diverse patient phenotypes. In recent years, several regulatory actions pertaining to emerging safety warnings and approval of new therapies may potentially influence the use of asthma treatments. This study aims to use real world evidence to bridge the gaps in our understanding of treatment approaches to capture the heterogeneity of pediatric asthma patients and support personalized clinical decisions. Specifically, this study aims to:
Study 1: Examine the impact of regulatory actions on trends in utilization of asthma treatments among subgroups of pediatric asthma populations.
Study 2: Evaluate the relationship between relevant asthma comorbidities on adherence and subsequent asthma control.
Study 3: To scan for potential safety outcomes associated with the use of biologic therapies in children with asthma using data mining approaches.
Inspiration
Although clinical guidelines offer several options for children with moderate to severe persistent asthma, treatment selection is largely based on patient’s preference or avoidance of adverse effects. Yet asthma is a heterogenous condition with several clinical phenotypes. Concurrent comorbidities such as atopic dermatitis, obesity and mental health concerns are prevalent. The influence of these conditions on worsening asthma control is well-recognized however limited information exists regarding which treatment regimens may be more beneficial to patients with multiple conditions. Furthermore, while the evidence regarding the benefit of biologic therapies in asthma is promising, utilization of these agents in children with asthma remain low.
Cool methodological features
This study will apply epidemiologic methodologies that will measure treatment effect heterogeneity within a dynamic population that differs by age, asthma presentation, concurrent conditions and environmental factors. This study will also leverage on data mining approaches to conduct safety surveillance of potential adverse outcomes in emerging therapies.
Impact
The current gaps in our understanding of asthma treatments present a challenge for clinicians caring for children, particularly those with uncontrolled disease. The overarching goal of this study is to generate the necessary evidence to guide clinical decisions that will promote personalized treatment selection and apply advanced pharmacovigilance methodologies in pediatric populations.
Next steps
After completing my training, I plan to pursue a career in an academic setting where I intend to continue to examine and address questions that focuses on improving the care of pediatric patients particularly those with complex chronic conditions. These include questions related to the impact of health care policy on patients and prescribers, post-marketing safety surveillance of new medications, and using population-level data to explore evidence of previously undetected patient phenotypes.