Dupilumab in Children with Uncontrolled Moderate-to-Severe Asthma
Leonard B. Bacharier, Jorge F. Maspero, Constance H. Katelaris, Alessandro G. Fiocchi, Remi Gagnon, Ines de Mir, Neal Jain, Lawrence D. Sher, Xuezhou Mao, Dongfang Liu, Yi Zhang, Asif H. Khan, Upender Kapoor, Faisal A. Khokhar, Paul J. Rowe, Yamo Deniz, Marcella Ruddy, Elizabeth Laws, Naimish Patel, David M. Weinreich, George D. Yancopoulos, Nikhil Amin, Leda P. Mannent, David J. Lederer, Megan Hardin
Abstract
BACKGROUND: Children with moderate-to-severe asthma continue to have disease complications despite the receipt of standard-of-care therapy. The monoclonal antibody dupilumab has been approved for the treatment of adults and adolescents with asthma as well as with other type 2 inflammatory diseases. METHODS: ) at week 12 and in the score on the Asthma Control Questionnaire 7 Interviewer-Administered (ACQ-7-IA) at week 24. End points were evaluated in the two primary efficacy populations who had either a type 2 inflammatory asthma phenotype (≥150 blood eosinophils per cubic millimeter or a fraction of exhaled nitric oxide of ≥20 ppb at baseline) or a blood eosinophil count of at least 300 cells per cubic millimeter at baseline. RESULTS: was 10.5±1.0 percentage points with dupilumab and 5.3±1.4 percentage points with placebo (mean difference, 5.2 percentage points; 95% CI, 2.1 to 8.3; P<0.001). Dupilumab also resulted in significantly better asthma control than placebo (P<0.001). Similar results were observed in the patients with an eosinophil count of at least 300 cells per cubic millimeter at baseline. The incidence of serious adverse events was similar in the two groups. CONCLUSIONS: Among children with uncontrolled moderate-to-severe asthma, those who received add-on dupilumab had fewer asthma exacerbations and better lung function and asthma control than those who received placebo. (Funded by Sanofi and Regeneron Pharmaceuticals; Liberty Asthma VOYAGE ClinicalTrials.gov number, NCT02948959.).