Real-World Experience with Dupilumab in Severe Asthma: One-Year Data from an Italian Named Patient Program.

Introduction: Dupilumab is a monoclonal antibody targeting IL-4Rα recently licensed for severe asthma (SA). A Named Patients Program (NPP) was created in Italy before its commercial availability for SA patients with no other available therapeutic options. We aimed to assess the real-world effectiveness of dupilumab in patients with SA and unmet needs.

Methods: We performed a multicentre retrospective study, including SA patients admitted to the NPP treated with dupilumab for 12 months. Data on the number of exacerbations, Asthma Control Test (ACT), pre-bronchodilator FEV%, oral corticosteroids (OCSs) use, FeNO and eosinophils count in peripheral blood were recorded at baseline and after 3, 6, and 12 months.

Results: We included 18 SA patients (mean age 53.3±12.4 years, 66.7% female). Eleven (61.1%) were OCSs dependent. Five patients (27.8%) received previous anti-IgE and/or anti-IL-5 agents. A significant improvement in ACT score (from 15.7±5.1 to 18.8±4.8, p=0.023), OCSs intake [10 (5-25) mg/day to 0 (0-5) mg/day, p=0.0333] and FeNO [from 25 (20-80) ppb to 21 (10.9-55.3) ppb, p=0.0190] was already detected after 3 months of treatment. After 12 months, a statistically significant decrease in the number of exacerbations from 2 (0-3) to 0 (0-1) (p<0.0068) and increase in FEV% from 73.5±19.5% to 87.1±19.2% (p=0.0407) and ACT to a mean value of 22.4±1.7 (p<0.0001) and the interruption of OCSs in all the patients (p<0.0001) was observed. A transient increase in the eosinophil count was observed in five patients (above 1000 cells/μL in 2 cases) after 3 months, without any clinical effect.

Conclusion: Dupilumab improved all the explored clinical outcomes after 12 months, and the transient hypereosinophilia did not modify treatment response. These real-world data confirm the results reported in randomized controlled trials and provide an important opportunity to characterize the clinical impact of the treatment in a non-trial setting. Further real-world studies with a larger cohort of patients are needed to confirm these findings.