Asthma, a chronic respiratory condition affecting millions worldwide, particularly in children, has been a focus of medical research for decades. The quest for effective treatments to manage this condition, especially in its more severe forms, has led to the development and testing of various medications. One such medication, dupilumab (DUPIXENT), has recently been the subject of an in-depth study, the findings of which are crucial for the medical community and those affected by asthma.
About the Study
The study, titled “Assessment of long-term safety and efficacy of dupilumab in children with asthma (LIBERTY ASTHMA EXCURSION),” was published in The Lancet Respiratory Medicine on November 10, 2023. It is an extension of the VOYAGE study—a 52-week, multinational, multicentre, phase 3 randomized, double-blind, placebo-controlled trial. The focus of this study was to evaluate the long-term safety and efficacy of dupilumab in children aged 6–11 years with uncontrolled, moderate-to-severe asthma.
In this open-label extension study, named EXCURSION, 365 children from the original VOYAGE study participated. Conducted across 70 centers in 17 countries, the study involved 240 children continuing with add-on dupilumab and 125 children who were on placebo during VOYAGE initiating dupilumab treatment. The primary endpoint was to assess the number and proportion of patients experiencing any treatment-emergent adverse event (TEAE) during the 52-week study period.
The findings revealed that 63.6% of the participants experienced at least one TEAE, with the most frequently reported being nasopharyngitis, pharyngitis, and upper respiratory tract infections. However, the study concluded that long-term treatment with dupilumab was well tolerated and had an acceptable safety profile.
The EXCURSION study’s findings on the long-term use of dupilumab in children with moderate-to-severe asthma are a beacon of hope in pediatric respiratory medicine. Dr. Isaac Goldstein, a prominent figure in this field, provides valuable insights into the significance of these results.
Dr. Isaac Goldstein emphasizes the importance of this study in understanding chronic asthma management in children. “The EXCURSION study sheds light on a crucial aspect of pediatric asthma treatment – long-term safety and efficacy. For years, we’ve grappled with the challenge of not just controlling severe asthma symptoms but doing so in a way that’s safe for children in the long run. Dupilumab’s performance in this study is a promising development.”
He further elaborates on the broader implications of these findings. “This study is not just about dupilumab. It’s a testament to the potential of biological therapies in treating chronic conditions like asthma. We’re entering an era where personalized medicine could become a reality for these young patients. Instead of a one-size-fits-all approach, we’re looking at treatments tailored to individual needs and conditions, which is a significant advancement in pediatric care.”
Dr. Isaac Goldstein also addresses the concerns regarding the treatment-emergent adverse events (TEAEs) observed in the study. “While the occurrence of TEAEs is something we cannot overlook, it’s important to contextualize these findings. The majority of these events were mild to moderate and similar to those observed in the parent VOYAGE study. This consistency is reassuring and indicates that while there are side effects, they are manageable and do not outweigh the benefits of the treatment for most patients.”
He concludes with a note on the future of asthma treatment. “The journey doesn’t end here. This study opens numerous avenues for further research. We need to continue exploring not just dupilumab but other potential treatments, their long-term impacts, and how they can be integrated into comprehensive care plans for children with asthma. It’s about building a future where asthma doesn’t limit our children’s potential to live full, active lives.”
The EXCURSION study, through its detailed exploration of dupilumab’s long-term use in pediatric asthma, marks a significant milestone in respiratory medicine. It not only confirms the safety and efficacy of this treatment but also paves the way for more nuanced, personalized approaches to managing chronic conditions like asthma in children. This advancement is a step towards a future where such conditions are no longer a barrier to the health and well-being of the younger population.
Bacharier, L. B., Maspero, J. F., Katelaris, C. H., Fiocchi, A. G., Gagnon, R., de Mir, I., et al. (2023). Assessment of long-term safety and efficacy of dupilumab in children with asthma (LIBERTY ASTHMA EXCURSION): an open-label extension study. The Lancet Respiratory Medicine. https://doi.org/10.1016/S2213-2600(23)00303-X