Asthma is one of the most common diseases affecting children. It is a main cause of morbidity and mortality in pediatric patients, and it represents a severe disease burden for patients and families. Asthma is an inflammatory disease of the airways, and it can be caused by many factors. Patients can have edema, bronchospasms, and increased mucus production as a result of this inflammatory reaction. All of this impairs the ability of the patients to breathe. Lack of oxygen throughout the body can quickly turn fatal for some patients. Patients and their caregivers always worry about what would trigger a patients’ asthma, what they can do to avoid this trigger, if they have a rescue inhaler on their person, and what the nearest emergency room is just in case of a flare up. There are many patients who do not respond well to the standard treatment, which has been recently updated to include inhaled corticosteroids replacing or working in addition to short-acting beta agonists. With routine use, short-acting beta agonists have been associated with increased morbidity and mortality. They have been shown to increase the number and severity of asthma exacerbations. With all that is changing in terms of routine asthma care, patients with moderate to severe asthma are often left with limited treatment options. Oral corticosteroids are used to treat exacerbations, but the long-term effects of oral corticosteroids in children are well-documented.(1)
Dupilumab is a monoclonal antibody marketed by Regeneron and Sanofi Genzyme. It is a revolutionary medication taking the type 2 inflammatory disease treatment to new heights. It is approved for many indications, and it is being investigated for other type 2 inflammatory diseases. Dupilumab is currently approved for moderate to severe asthma in adults, atopic dermatitis, and rhinosinusitis with nasal polyps. Dupilumab was recently approved for the treatment of moderate to severe asthma in patients 6-11 years old. The trial that granted this approval was a 52-week, phase 3, randomized, double-blind, placebo-controlled clinical trial. The primary endpoint of the trial was the annualized rate of severe asthma exacerbations. Patients who were given subcutaneous dupilumab had an annualized rate of 0.31 as compared to 0.75 in the placebo arm. When discussing life-threatening clinical asthma exacerbations, this difference is clinically and statistically significant. In terms of overall asthma control, patients in the dupilumab group had better asthma control when compared to placebo. Dupilumab is also quite helpful for patients with an eosinophilic asthma type. Interestingly enough, the amount of adverse events was similar between the two groups. This indicates a significant breakthrough and a new option for patients with moderate to severe asthma which cannot be controlled using other measures.(2)
In patients who fail conventional therapy, the options can be limited. This challenge for patients, families, and caregivers can be quite daunting. With a new agent on the market for these children, the amount of asthma exacerbations and associated healthcare costs is less. Although a new monoclonal antibody is a pricy therapy, the amount that could be saved due to less hospitalizations can be a significant help for the healthcare system overall.
Lizzo JM, Cortes S. Pediatric Asthma. [Updated 2021 Aug 11]. In: StatPearls [Internet]. Treasure Island (FL): StatPearls Publishing; 2021 Jan-. Available from: https://www.ncbi.nlm.nih.gov/books/NBK551631/
Bacharier LB, Maspero JF, Katelaris CH et al. Dupilumab in Children with Uncontrolled Moderate-to-Severe Asthma. N Engl J Med. 2021 Dec 9;385(24):2230-2240.