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 CharacterizationClinical Trial Outcomes Observational Studies Core OutcomeEither ACQ (especially if FEV 1 available) or ACT (especially if FEV 1 not available) Supplemental Outcome ATAQ in studies of health care utilization NoneATAQ in studies of health care utilization EmergingOther adult tools

9. Recommendations for Patients Aged 11 Years and Younger CharacterizationClinical Trial Outcomes Observational Studies Core OutcomecACT (ages 5-11)NonecACT (ages 5-11) Supplemental Outcome NonecACT (ages 5-11)None EmergingOther childhood tools

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 FEV 1 – ACT does not require FEV 1 – Most well-validated version of ACQ includes FEV 1

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