Omalizumab in children with inadequately controlled severe allergic (IgE-mediated) asthma

Abstract

Background:

Many children with severe persistent allergic (IgE-mediated) asthma remain inadequately controlled despite treatment with high-dose inhaled corticosteroids (ICS) plus a long-acting β₂-agonist (LABA).

Research and design methods:

This pre-specified analysis of a randomized, double-blind, placebo-controlled trial evaluated the efficacy and safety of omalizumab in children (6–<12 years) with perennial allergen sensitivity, and history of asthma exacerbations and symptoms despite treatment with ICS (fluticasone ≥500 µg · day⁻¹ or equivalent) plus a LABA. Patients received omalizumab (75–375 mg once or twice a month by subcutaneous injection, as determined from dosing tables) or placebo over 52 weeks (24-week fixed-steroid then 28-week adjustable-steroid phases).

Results:

Out of 246 randomized patients (omalizumab, n = 166; placebo, n = 80), efficacy was analysed in 235 (omalizumab, n = 159; placebo, n = 76). Over the 24-week fixed-steroid phase, omalizumab reduced the rate of clinically significant asthma exacerbations (worsening symptoms requiring doubling of baseline ICS dose and/or systemic steroids) by 34% versus placebo (0.42 vs 0.63, rate ratio 0.662; P = 0.047). Over 52 weeks, the exacerbation rate was reduced by 50% (P < 0.001). Omalizumab had an acceptable safety profile, with no statistically significant (P < 0.05) differences in adverse events observed between omalizumab and placebo.

Conclusion:

Add-on omalizumab is well-tolerated and reduces exacerbations in children (6–<12 years) with severe persistent allergic asthma, inadequately controlled despite high-dose ICS plus a LABA. It should be noted that the sample size was not based on providing statistical power in the severe subgroup, and no corrections were made for multiple comparisons; however, outcomes consistently favoured omalizumab.

Trial registration: ClinicalTrials.gov identifier: NCT00079937.

Key words:

• Asthma
• Efficacy
• Omalizumab
• Paediatrics
• Safety

Transparency

Declaration of funding

This study was sponsored by Novartis Pharma AG, who also funded the preparation of this manuscript.

Declaration of financial/other relationships

M.K. has no relevant financial interests to disclose. J.H. has disclosed that he has received research support from Merck, GSK, Novartis, Shire, Boehringer Ingelheim, Medimmune, Circassa and Stallergene. He has acted as a consultant for GSK, Novartis, Merck, CSL Behring, Allergilab and Nycomed, and has been part of Speaker Bureau for Merck, Novartis, King, CSL Behring and GSK. E.G. has disclosed that she has served on the speakers’ bureau and has received research support from Novartis; A.F.T., C.F.V. and M.B. have disclosed that they are all employees and shareholders of Novartis.

Some peer reviewers receive honoraria from CMRO for their review work. The peer reviewers of this paper have disclosed that they have no relevant financial relationships.

Acknowledgements

The authors disclose that they were assisted in the preparation of the manuscript by professional medical writer Tom McMurray, ACUMED, funded by Novartis.