Table 3. Selected Pediatric Omalizumab Clinical Trials6,7
Study Patients and Baseline Treatment Omalizumab Study Treatment Primary Endpoints Results
Milgrom et al.6
RDBPC
n = 334
 
Age: 6-12 years
 
Well controlled with inhaled corticosteroids (doses equivalent to 168 to 420 mcg/day of BDP) and bronchodilators
Inhaled BDP and OMX SC 0.016 mg/kg/IgE (IU/mL) for 28 weeks or placebo
OMZ n=225
 
Placebo n=109
 
Steroid-stable phase: BDP dose kept constant for 16 weeks
 
Steroid-reduction phase: BDP dose reduced over 8 weeks to minimum tolerated dose: Minimum dose maintained for 4 additional weeks
BDP does reduction, symptoms, spirometry, AE Median BDP dose reduction of 100% with OMZ vs. 66.7% with placebo (p=0.001)
 
BDP completely withdrawn in 55% receiving OMZ vs. 39% receiving placebo (p=0.004)
 
Steroid stable phase:
Asthma exacerbations occurred in 15.6% receiving OMZ vs. 22.9% receiving placebo (p=0.095)
 
Steroid reduction phase:
Asthma exacerbations occurred in 18.2% receiving OMZ vs. 38.5% receiving placebo (p<0.001)
 
No statistical difference in symptom scores or spirometry
 
No serious AE
Lemanske et al.7
RDBPC
Same as Milgrom et al.6 Same as Milgrom et al.6 Mean change from baseline in asthma-related QOL scores End of steroid-stable phase:
No statistical difference in PAQLQ scores between groups
 
End of steroid-reduction phase:
-Greater mean change from baseline in activities scores (p<0.05) and symptoms scores (p<0.05) with OMZ vs. placebo
-No statistical difference in emotions scores between groups
-The overall mean score change from baseline was statistically significant with OMZ vs. placebo (p<0.05)
 
End of study:

Greater proportion of OMZ patients achieved a clinical improvement in asthma-related QOL

AE=Adverse event
BDP=beclomethasone dipropionate or equivalent inhaled steroid
DBPC=double-blind, placebo-controlled, n=number of patients in study group
OMZ=omalizumab
PAQLQ=Pediatric Asthma Quality of Life Questionnaire
QOL=Quality of Life
RDBPC=randomized, double-blind, placebo-controlled trial
SC=subcutaneous

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