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reduction of 100% with OMZ vs. 66.7% with placebo (p=0.001) BDP completely withdrawn in 55% receiving OMZ vs. 39% receiving placebo (p=0.004) Steroid stable phase: Asthma exacerbations occurred in 15.6% receiving OMZ vs. 22.9% receiving placebo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