Table 1.
Demographics of the study population*
| Overall | Guidelines Based Therapy | Guidelines Based Therapy + Omalizumab | P | |
|---|---|---|---|---|
|
| ||||
| N=478 | N=89 | N=259 | ||
| Study Cohort - no. (%): | ||||
| 2012 | 229 (47.9%) | 43 (48.3%) | 127 (49.0%) | 0.99 |
| 2013 | 249 (52.1%) | 46 (51.7%) | 132 (51.0%) | |
| Injection schedule - no. (%): | ||||
| Once per 2 weeks | 183 (38.3%) | 36 (40.4%) | 100 (38.6%) | 0.86 |
| Once per 4 weeks ¶ | 295 (61.7%) | 53 (59.6%) | 159 (61.4%) | |
| Race or ethnic group - no. (%): | ||||
| African American | 279 (58.4%) | 54 (60.7%) | 145 (56.0%) | 0.43 |
| Hispanic | 161 (33.7%) | 30 (33.7%) | 88 (34.0%) | |
| White, mixed or other | 38 (7.95%) | 5 (5.62%) | 26 (10.0%) | |
| Caretaker completed high school - no. (%) | 340 (71.3%) | 55 (61.8%) | 188 (72.9%) | 0.07 |
| 1+ household member employed - no. (%) | 330 (69.0%) | 59 (66.3%) | 175 (67.6%) | 0.93 |
| Annual household income <$15,000 - no. (%) | 262 (55.4%) | 51 (58.6%) | 145 (56.6%) | 0.84 |
| Age – yr. | 10.0 [8.0–12.0] | 9.00 [8.0–12.0] | 10.0 [8.0–12.0] | 0.48 |
| Male sex - no. (%) | 303 (63.4%) | 59 (66.3%) | 174 (67.2%) | 0.98 |
| Duration of asthma – yr. | 7.25 [4.85–9.90] | 6.75 [4.92–9.50] | 7.67 [4.96–10.1] | 0.15 |
| C-ACT score in the previous month, age 4 to11 yr. (n=358) † | 21.6 (3.63) | 21.3 (3.52) | 21.3 (3.70) | 0.91 |
| ACT score in the previous month, age 12 yr. or older (n=119) † | 21.5 (3.18) | 21.2 (3.87) | 21.4 (3.05) | 0.84 |
| Asthma-related symptoms - days in prior 2 weeks ‡ | 2.34 (3.13) | 2.56 (2.95) | 2.51 (3.25) | 0.89 |
| Wheezing | 1.79 (2.65) | 1.98 (2.37) | 1.89 (2.70) | 0.77 |
| Interference with activity | 1.39 (2.61) | 1.72 (2.82) | 1.56 (2.84) | 0.65 |
| Nighttime sleep disruption | 0.77 (1.80) | 0.90 (1.98) | 0.88 (1.84) | 0.93 |
| FEV1 - % of predicted value | 90.8 [79.7–101] | 89.6 [77.4–102] | 88.7 [78.9–98.9] | 0.81 |
| FEV1:FVC ×100 | 78.3 [72.0–84.6] | 78.2 [70.0–84.8] | 77.3 [71.1–84.4] | 0.97 |
| Medication - no. (%) § | 0.89 | |||
| Step level 2 to 4 | 294 (61.5%) | 43 (48.3%) | 121 (46.7%) | |
| Step level 5 | 184 (38.5%) | 46 (51.7%) | 138 (53.3%) | |
| 1+ Asthma Exacerbation** | 168 (35.1%) | 35 (39.3%) | 106 (40.9%) | 0.89 |
Values are counts (percentages), means (SDs) or medians [IQR]. Calculation of the p-values for the independence test between groups was calculated using the Mann-Whitney U test, and the chi-square test for continuous and categorical variables respectively.
Scores on the Childhood Asthma Control Test (C-ACT) and the Asthma Control Test (ACT) were measured on scales of 0 to 27 and 5 to 25, respectively. A score of 19 or less on either test indicates that asthma is not well controlled. The minimally important difference for ACT equals 3 points; for the C-ACT, a 3-point increase suggests a clinically relevant improvement in asthma control, whereas a 2-point decrease suggests a clinically relevant worsening.
The number of days with symptoms was calculated as the largest of the following variables during the previous 2 weeks: number of days with wheezing, chest tightness, or cough; number of nights of sleep disturbance; and number of days when activities were affected. This symptom scale ranges from 0 to 14 days per 2-week period.
Six treatment steps were established, which is consistent with report 3 of the National Asthma Education and Prevention Program guidelines to standardize prescribing patterns according to levels of asthma severity summarized here. Steps 1 and 2 apply to mild asthma, step 3 to moderate asthma, and steps 4 through 5 to severe asthma. At step 0, the recommendation is for no asthma control medication or albuterol as needed; at step 1, the recommendation is for 50 μg of fluticasone twice a day; at step 2, the recommendation is for 100 μg of fluticasone twice a day; at step 3, the recommendation is for 250 μg of fluticasone twice a day; at step 4, the recommendation is for 250 μg of fluticasone and 50 μg of salmeterol twice a day (Advair, GlaxoSmithKline); and at step 5, the recommendation is for 500 μg of fluticasone and 50 μg of salmeterol twice a day (Advair, GlaxoSmithKline).
Injections once every 2 or 4 weeks based on bodyweight and IgE , see APPENDIX 1: XOLAIR® (OMALIZUMAB) DOSING AND INJECTIONS, Table A1a Xolair® (omalizumab) Dosing Table in original protocol.
One or more asthma-related exacerbations, requiring treatment with a systemic corticosteroid course, during the double blind phase of the study (90 days period).