TABLE V.
Recommendations for supplemental asthma outcomes for NIH-initiated clinical research for children*
| Characterization of study population for prospective clinical trials (ie, baseline information) | Prospective clinical trial efficacy/effectiveness outcomes | Observational study outcomes** | |
|---|---|---|---|
| Biomarkers | 1. FeNO 2. Sputum eosinophils 3. CBC (total eosinophils) 4. Total IgE 5. Allergen-specific IgE 6. Urinary LTE4 |
1. FeNO 2. Sputum eosinophils 3. CBC (total eosinophils) 4. Total IgE 5. Allergen-specific IgE 6. Urinary LTE4 |
1. FeNO 2. Sputum eosinophils 3. CBC (total eosinophils) 4. Total IgE 5. Allergen-specific IgE 6. Urinary LTE4 |
| Composite Scores | None | cACT | None |
| Exacerbations | 1. For trials in the acute management of exacerbations (ED setting): a. Validated assessment tools, such as PASS, PS, PRAM, CAS, PI, ASS b. FEV1 (ages 5-11 years, as feasible) 2. Any prior exacerbation 3. Any prior ICU admission/intubation 4. SES of the study population |
For trials in the acute management of exacerbations (ED setting): a. Validated assessment tools such as PASS, PS, PRAM, CAS, PI, ASS b. FEV1 (ages 5-11 years, as feasible) |
None |
| Healthcare Utilization and Costs | 1. Categorization of asthma-specific outpatient visits: a. Primary care i. Scheduled ii. Unscheduled b. Specialty care i. Scheduled ii. Unscheduled 2. Respiratory healthcare use (pneumonia, bronchitis, etc) 3. Asthma school absences 4. Asthma work absences |
1. Categorization of asthma-specific outpatient visits: a. Primary care i. Scheduled ii. Unscheduled b. Specialty care i. Scheduled ii. Unscheduled 2. Respiratory healthcare use 3. Asthma school absences 4. Asthma work presenteeism and absenteeism (WPAI instrument) 5. Cost analysis and cost-effectiveness analysis |
1. Categorization of asthma-specific outpatient visits: a. Primary care i. Scheduled ii. Unscheduled b. Specialty care i. Scheduled ii. Unscheduled 2. Respiratory healthcare use 3. Asthma school absences 4. Asthma work presenteeism and absenteeism (WPAI instrument) 5. Cost analysis and cost-effectiveness analysis |
| Pulmonary Physiology | 1. PEF monitoring 2. Airway responsiveness† 3. Lung volumes 4. Gas exchange‡ |
1. PEF monitoring 2. Airway responsiveness† 3. Lung volumes 4. Spirometry (pre- and post-bronchodilator) 5. Gas exchange‡ |
1. PEF monitoring 2. Airway responsiveness† 3. Lung volumes 4. Gas exchange‡ |
| Quality of Life | 1. CHSA 2. PAQLQ 3. Pediatric Caregiver AQLQ 4. PedsQL 3.0, Asthma Module |
1. CHSA 2. PAQLQ 3. Pediatric Caregiver AQLQ 4. PedsQL 3.0, Asthma Module |
1. CHSA 2. PAQLQ 3. Pediatric Caregiver AQLQ 4. PedsQL 3.0, Asthma Module |
| Symptoms | PACD (daily diary) | PACD (daily diary) | PACD (daily diary) |
ASS, Asthma Severity Score; cACT, childhood Asthma Control Test; CAS, Clinical Asthma Score; CBC, complete blood count; CHSA, Child Health Survey for Asthma; ED, emergency department; FeNO, fractional exhaled nitric oxide; FEV1, forced expiratory volume in 1 second; ICU, intensive care unit; LTE4, leukotriene E4; NIH, National Institutes of Health; PACD, Pediatric Asthma Caregiver Diary; PAQLQ, Pediatric Asthma Quality of Life Questionnaire; PASS, Pediatric Asthma Severity Score; Pediatric Caregiver AQLQ, Pediatric Caregiver Asthma Quality of Life Questionnaire; PEF, peak expiratory flow; PedsQL, Pediatric Quality of Life Inventory; PI, Pulmonary Index; PRAM, Preschool Respiratory Assessment Measure; PS, Pulmonary Score; SES, socioeconomic status; WPAI, Work Productivity and Activity Impairment Questionnaire.
Only some of these outcomes are also suitable for children 0-4 years of age.
Observational study designs include cohort, case control, cross sectional, retrospective reviews, and genome-wide association studies, and secondary analysis of existing data. Some measures may not be available in studies using previously collected data.
Methacholine inhalation and exercise challenge (children aged 5 to 7 years are less likely to perform well on these tests)
Pulmonary diffusing capacity (breath holding is difficult in children aged 5 to 7 years); arterial blood gases and pulse oximetry
Note: For a detailed presentation and discussion of each of these outcomes, the methodology for measurement, and supporting bibliographic references, see respective article in this supplement.