Economic Outcome: Comparison of Number of Missed School Days Due to Asthma
The number of school days missed was available for 307 of the 419 (73%) study participants, of which 152 were in the Omalizumab (Xolair) + Conventional Therapy arm. Source of data: caretaker/participant self-report. Data represent an average of those collected in the time period (weeks 12-60), where at least one value was available in this assessment period and at baseline for a participant.
Economic Outcome: Number of Missed Work Days by Caretaker Due to Asthma
The number of work days missed by the caretaker due to the study participant's asthma was available for 138 of 419 (33%) study participant caretakers. Source of data: caretaker self-report. Data represent an average of those collected in the time period (weeks 12-60).
Child Asthma Control Test (C-ACT) Score
The Childhood Asthma Control Test (C-ACT) is a validated tool to assess overall asthma control (over the last 4 weeks) in patients ages 4 to 11 years. Scores can range from 0 to 27. A score of 19 or less is indicative of asthma that is not well controlled. The minimally important difference in C-ACT scores is not defined. C-ACT scores were available as an outcome measure in 236 of the 419 participants, 118 of whom were in the Omalizumab (Xolair) + Conventional Therapy arm. Data represent an average of those collected in the time period (weeks 12-60), where at least one value was available in this assessment period and at baseline for a participant.
Asthma Control Test (ACT) Score
The Asthma Control Test (ACT) is a validated tool to assess overall asthma control (over the last 4 weeks) in patients >= 12 years of age. It is a questionnaire comprised of 5 questions assessing: asthma symptoms, use of rescue medications, and the impact of asthma on everyday functioning. All questions are scored on a 5-point Likert scale, with a higher score indicating better control. All scores were added together to calculate a total score. Total scores can range from 5 to 25. A score of 19 or less is indicative of asthma that is not well controlled. The minimally important difference for ACT is 3 points. ACT scores as an outcome measure were available in 150 of the 419 participants, 77 of whom were in the Omalizumab (Xolair) + Conventional Therapy arm. Data represent an average of those collected in the time period (weeks 12-60), where at least one value was available in this assessment period and at baseline for a participant.
Forced Expiratory Volume in 1 Second (FEV1) % Predicted
FEV1 is air volume exhaled in 1 second during spirometry. For the trial, mild asthma is defined as pre-bronchodilator FEV1 ≥80% predicted, requiring no/low-moderate dose of inhaled glucocorticoids; moderate asthma and severe asthma, respectively, as pre-bronchodilator FEV1 <80% predicted requiring the same glucocorticoids as mild asthma and FEV1 <80% predicted requiring high-dose inhaled glucocorticoids (with/without continuous oral glucocorticoids) or uncontrolled despite treatment. FEV1 % of predicted is FEV1 converted to a percentage of normal, based on height, weight, and race. FEV1 percent predicted data as an outcome measure were available in 363 of the 419 participants, 190 of whom were in the Omalizumab (Xolair) + Conventional Therapy arm. Data represent an average of those collected in the time period (weeks 12-60), where at least one value was available in this assessment period and at baseline for a participant.
FEV1/FVC Ratio
The FEV1 (forced expiratory volume 1))/ FVC (forced vital capacity) ratio is used to evaluate airways obstructions since pure restrictive ventilatory defects cause an equal reduction in the FEV1 and the FVC. An FEV1/FVC ratio below 80% indicates airflow obstruction. Normal FEV1/FVC: 8 - 19 years of age=85%.
FEV1/FVC ratio data as an outcome measure were available in 363 of the 419 participants, 190 of whom were in the Omalizumab (Xolair) + Conventional Therapy arm. Data represent an average of those collected in the time period (weeks 12-60), where at least one value was available in this assessment period and at baseline for a participant.
Exhaled Nitric Oxide
Exhaled nitric oxide is a biomarker of airway inflammation. Measurement (in parts per billion,ppb) of exhaled nitric oxide (eNO) prior to spirometry, employing a technique modified after Silkoff et al (1997) and following American Thoracic Society guidelines for eNO assessment (American Thoracic Society, 1999). Nitric oxide concentrations were measured using a rapid-response chemiluminescent analyzer (NIOX™ System, Aerocrine, Sweden) which has a response time of < 700 ms for 10-90% full scale. The Food and Drug Administration has approved this device for clinical application in asthma management. Data represent an average of those collected in the time period (weeks 12-60), where at least one value was available in this assessment period and at baseline for a participant.
Percent Adherence to Asthma Medication
Adherence to the study regimen and other asthma treatments, assessed as percent of expected dose taken, by means of study interviews and study physician corroboration every 3 months. Adherence data as an outcome were available in 384 of the 419 participants, 193 of whom were in the Omalizumab (Xolair) + Conventional Therapy arm. Data represent an average of those collected in the time period (weeks 12-60), where at least one value was available in this assessment period and at baseline for a participant.
Percent Prevalence: Treatment Step Level 1 or 2 (Mild Asthma)
Treatment steps were established, per the National Asthma Education and Prevention Program Expert Panel Report 3 guidelines. Steps 1-2 apply to mild asthma, 3 to moderate asthma, and 4-6 to severe asthma. At Step 0, the recommendation is for no asthma-control medication or albuterol as needed; at 1, budesonide 180 mcg once a day; at 2, budesonide 180 mcg twice a day; at 3, budesonide 360 mcg twice a day; at 4, fluticasone-salmeterol (Advair, GlaxoSmithKline) 250 mcg fluticasone and 50 mcg salmeterol twice a day; at 5, Advair 250 mcg and 50 mcg twice a day plus montelukast once a day; and at 6, Advair 500 mcg and 50 mcg twice a day plus montelukast once a day. (The doses for montelukast are 5 mg per day for those <=14 years old and 10 mg per day for those >=15 years.) Data represent an average of those in the time period, where at >/= 1 value was available in this period and at baseline for a participant; results are model predicted numbers (e.g.,odds ratios converted to percentages).
Percent Prevalence: Treatment Step Level 4 Through 6 (Severe Asthma)
Steps were established, per the National Asthma Education and Prevention Program Expert Panel Report 3 guidelines. Steps 1-2 apply to mild asthma, 3 to moderate asthma, and 4-6 to severe asthma. At Step 0, the recommendation is for no asthma-control medication or albuterol as needed; at 1, budesonide 180 mcg once a day; at 2, budesonide 180 mcg twice a day; at 3, budesonide 360 mcg twice a day; at 4, fluticasone-salmeterol (Advair, GlaxoSmithKline) 250 mcg fluticasone and 50 mcg salmeterol twice a day; at 5, Advair 250 mcg and 50 mcg twice a day plus montelukast once a day; and at 6, Advair 500 mcg and 50 mcg twice a day plus montelukast once a day. (The doses for montelukast are 5 mg per day for those <=14 years old and 10 mg per day for those >=15 years.) Data represent an average of those in the time period, where at least one value was available in this period and at baseline for a participant. Results values are model predicted numbers,(e.g, odds ratios converted to percentages).
Dose Inhaled Corticosteroids (Glucocorticoids)
Prescribed dose (mcg/day) of inhaled glucocorticoids to maintain asthma control. The dose of inhaled glucocorticoids was converted to the budesonide-equivalent dose. Data represent an average of those collected in the time period (weeks 12-60), where at least one value was available in this assessment period and at baseline for a participant.
Percent Prevalence: Prescribed Rescue Beta 2 Agonists
Percent of participants prescribed long-acting beta 2 agonists to maintain asthma control. Data represent an average of those collected in the time period (weeks 12-60), where at least one value was available in this assessment period and at baseline for a participant. Results values are model predicted numbers, (e.g.,odds ratios converted to percentages).
Percent Prevalence: Asthma-Related Medical Care Resource Utilization - Hospitalizations
Percent participants with >=1 hospitalizations. A hospitalization is defined as an asthma-related, overnight hospitalization. . Results values are model predicted numbers,(e.g., odds ratios converted to percentages).
Percent Prevalence: Asthma Exacerbations
Percent participants with >=1 exacerbations. An exacerbation was defined as a prednisone burst (a minimum of 20 mg per day of prednisone, or the equivalent, taken for any 3 of 5 consecutive days) or hospitalization. Results values are model predicted numbers, (e.g.,odds ratios converted to percentages).
Asthma Caregiver's Quality of Life Questionnaire (PACQLQ) Overall Score
Asthma-Specific Quality of Life (QOL) Measure . The PACQLQ is a validated tool that measures limitations and anxieties faced by primary caregivers of children with asthma. Scores are calculated as the mean score within two domains of questions (re: activity limitation and emotional function) and overall scores represent the mean across all questions. The use of the PACQLQ is valid for use in the caretakers of children ages 7 to 17 years of age. Higher scores indicate better quality of life. Minimum possible score is 1 (maximum impairment); maximum possible score is 7 (no impairment). The range of actual scores were a minimum of 2.4 and a maximum of 7.
Method: Caretaker self-report. PACQLQ scores were available for 320 of 419 (76%) of study participant caretakers (159 in the Omalizumab (Xolair) + Conventional Therapy arm).
Paediatric Asthma Quality of Life Questionnaire (PAQLQ) Overall Score
Asthma-specific quality of life (QOL) validated tool designed for children 7 to 17 years of age. PAQLQ measures functional problems that are most troublesome to children with asthma. PAQLQ has 23 questions in 3 domains (activity limitation=5, emotional function=8, symptoms=10). Patients responded to each question on a 7-point Likert scale. Overall PAQLQ score is mean of 23 questions; each domain score is mean of questions in that domain. Minimum possible score is 1 (maximum impairment); maximum possible score is 7 (no impairment). Actual scores ranged from 2.1 to 7.
PAQLQ scores were available for 338 of 419 (81%) of study participants, 170 of whom were in the Omalizumab (Xolair) + Conventional Therapy arm.