Composite Scores of Asthma Control

1. Composite Scores of Asthma Control Michael Schatz, MD Michelle M. Cloutier, MD Co-Chairs

2. Committee Members • Mario Castro, M.D. • Noreen Clark, Ph.D. • H.W. Kelly, Ph.D. • Rita Mangione-Smith, M.D. • James Sheller, M.D. • Christine Sorkness, Ph.D. • Stuart Stoloff, M.D. • James Tonascia, Ph.D. • Peter Gergen, M.D. (NIH Liaison)

3. ATS-ERS Report • Composite measures divided into 2 types • Expressed as categorical variables (e.g. asthma control days/weeks, control categories) • Expressed as numeric variables (e.g. ACQ, ACT) • Categorical measure limitations include • Empiric derivation • Lack of standardization • Limited information on control in individual patients, especially regarding responsiveness

4. ATS-ERS Report • Reviewed 4 adult numeric variable tools • ACQ • ACT • ATAQ • ACSS • Concluded at least one composite measure expressed as a numeric variable was “essential” for characterization of study populations and as an outcome measure

5. Methods • Systematic literature search to identify • All relevant tools • Validation and use studies • For each identified instrument, repeatability, responsiveness, and validity (criterion, construct, predictive) were assessed • For each identified instrument, its use in clinical trials was assessed • Also evaluated practicality, associations, and demographic considerations

6. Working Definition: Composite Measures of Asthma Control • Single questionnaires (with or without physiologic measures) • Designed to measure the multi-dimensional construct of asthma control • Assess more than symptom and rescue therapy frequency • Could be targeted for adults, children, or both

7. Results • Systematic literature search identified 17 instruments • 7 adults • 8 children • 2 both • Summary of recommendations • Adults • Children • Rationale for recommendations

8. Recommendations for Patients Aged 12 Years and Older

9. Recommendations for Patients Aged 11 Years and Younger

10. General Rationale for Recommendations • Importance of asthma control as a goal of therapy • Extensive validation data and demonstration of responsiveness to therapy for two tools in adults (ACQ and the ACT) • Reasonable validation data for one tool in children (cACT) • Low patient burden and risk

11. Specific Tool Rationale for Patients Aged 12 Years and Over • ACQ used in most clinical trials • ACT has most published validation data • Both tools validated for use in person, at home, or by phone • MID defined for both tools • ACQ and ACT correlate strongly and perform similarly • FEV1 • ACT does not require FEV1 • Most well-validated version of ACQ includes FEV1

12. Specific Tool Rationale for Patients Aged 11 Years and Under • cACT has more validation data for children aged 5-11 than other tools, but needs more responsiveness data and definition of MID • cACT meets minimum standard as a core measure for characterization and observational studies • cACT supplemental for clinical trials pending additional responsiveness data/definition of an MID • Only one validated tool for children under age 5; more validation data are needed before it can be recommended as a core or supplemental measure (potential categorical measures not addressed)

13. Future Research Directions I • Further validation and responsiveness data • Incorporation of risk domain into control tools • Core and supplemental composite outcome measures for children under age 5 • Determine the value of including physiologic measures in the instruments

14. Future Research Directions II • Define predictive properties of the composite measure instruments for other asthma outcomes, especially future outcomes • Validation in specific subgroups defined by race/ethnicity, socioeconomic status, asthma severity, asthma treatment, asthma phenotypes • Public domain composite measure of asthma control if needed