Omalizumab is approved for inadequately controlled allergic (IgE-mediated) asthma in adults and adolescents, and the goal of the research was to test its safety and efficacy in children between the ages of 6 and 11.
Dr. Bob Lanier, from the University of North Texas, Fort Worth, and colleagues randomized 627 children to add either omalizumab or placebo to their current asthma regimen. All of the children were having asthma exacerbations despite taking at least medium-dose inhaled corticosteroids.
As the authors report in the Journal of Allergy and Clinical Immunology for December, the dose of omalizumab ranged from 75 to 375 mg subcutaneously, depending on weight, and was given once or twice a month for 52 weeks.
During the first 24 weeks of the study, the children remained on a constant steroid inhaler dose. In the last 28 weeks, the dose could be reduced if clinical improvements were seen.
Efficacy was analyzed in 576 children. During the fixed-dose steroid phase, omalizumab cut the occurrence of significant asthma exacerbations by 31% relative to placebo (p = 0.007). (Exacerbations were defined as requiring a doubling of the inhaled steroid dose and/or systemic steroids.)
Over the full 52-week period, omalizumab reduced the overall exacerbation rate by 43% (p < 0.001) and the severe exacerbation rate by 50% (p = 0.004).
Omalizumab was generally well tolerated, and the overall rate of side effects was comparable to that of placebo, the report shows.
“This clear positive benefit-to-risk ratio suggests that the addition of omalizumab may provide an important new management option for improving asthma control in children underserved by current therapeutic strategies,” the authors conclude.
The study was supported by Novartis Pharma AG, which markets omalizumab as Xolair.
J Allergy Clin Immunol 2009;124:1210-1216.