Asthma outcomes: Symptoms

doi:10.1016/j.jaci.2011.12.981

Journal of Allergy and Clinical Immunology

Volume 129, Issue 3, Supplement, March 2012, Pages S124-S135

https://doi.org/10.1016/j.jaci.2011.12.981 Get rights and content

Background

Respiratory symptoms are commonly used to assess the impact of patient-centered interventions.

Objective

At the request of National Institutes of Health (NIH) institutes and other federal agencies, an expert group was convened to propose which measurements of asthma symptoms should be used as a standardized measure in future clinical research studies.

Methods

Asthma symptom instruments were classified as daily diaries (prospectively recording symptoms between research visits) or retrospective questionnaires (completed at research visits). We conducted a systematic search in PubMed and a search for articles that cited key studies describing development of instruments. We classified outcome instruments as either core (required in future studies), supplemental (used according to study aims and standardized), or emerging (requiring validation and standardization). This work was discussed at an NIH-organized workshop in March 2010 and finalized in September 2011.

Results

Four instruments (3 daily diaries, 1 for adults and 2 for children; and 1 retrospective questionnaire for adults) were identified. Minimal clinically important differences have not been established for these instruments, and validation studies were only conducted in a limited number of patient populations. Validity of existing instruments may not be generalizable across racial-ethnic or other subgroups.

Conclusions

An evaluation of symptoms should be a core asthma outcome measure in clinical research. However, available instruments have limitations that preclude selection of a core instrument. The working group participants propose validation studies in diverse populations, comparisons of diaries versus retrospective questionnaires, and evaluations of symptom assessment alone versus composite scores of asthma control.

Section snippets

Definitions

Asthma symptoms are measured by patient or caregiver report, via self-administered paper or electronic diaries or questionnaires, or interviewer-administered questionnaires. A variety of considerations influence the measurement of asthma symptoms in research studies. For example, should each commonly recognized symptom of asthma (eg, cough, wheeze, shortness of breath, and chest tightness) be assessed individually, or should a composite overall score of daily symptom burden be recorded? What

Review of specific asthma symptom questionnaires

This section summarizes each of the 4 asthma symptom questionnaires (3 daily diaries, 1 retrospective questionnaire) that were supported by validation studies. For each questionnaire, we present the methods used to develop the questionnaire; the range of scores; the internal consistency and test-retest reliability; validity; responsiveness; and practicality and risk.

Electronic diaries

Patient-reported daily diaries provide the opportunity to collect real-time information about respiratory symptoms, SABA use, and other information. Compared with interval questionnaires at study visits (which are based on retrospective assessments over the previous 1 to 4 weeks or more), daily diaries are theoretically less susceptible to problems related to incomplete recall and/or recall bias.⁴⁰ However, diary data are often incomplete, and results of studies indicate that diaries are often

SFDs

Derivatives of daily symptom diary data, such as SFDs, can be used to compare relative symptom frequency in study participants by reporting the number or proportion of days without asthma symptoms for each study group. However, various investigators have measured SFDs using different instruments in studies of adults (eg, Daily Symptom Diary Scale and Nocturnal Diary Scale,⁴⁵ the Symptom-Free Day Questionnaire,³⁸ and Daily Diary⁴⁶) and children (eg, PACD³). Moreover, as discussed in the ATS/ERS

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: The Asthma Outcomes workshop was funded by contributions from the National Institute of Allergy and Infectious Diseases; the National Heart, Lung, and Blood Institute; the Eunice Kennedy Shriver National Institute of Child Health and Human Development; the National Institute of Environmental Health Sciences; the Agency for Healthcare Research and Quality; and the Merck Childhood Asthma Network, as well as by a grant from the Robert Wood Johnson Foundation. Contributions from the National Heart, Lung, and Blood Institute; the National Institute of Allergy and Infectious Diseases; the Eunice Kennedy Shriver National Institute of Child Health and Human Development; the National Institute of Environmental Health Sciences, and the US Environmental Protection Agency funded the publication of this article and all other articles in this supplement.

: Disclosure of potential conflict of interest: R. F. Lemanske, Jr, is a speaker for Merck and AstraZeneca and has consulted for AstraZeneca, Map Pharmaceuticals, Gray Consulting, Smith Research Inc, Merck Childhood Asthma Network, Novartis, Quintiles/Innovax, RC Horowitz & Co Inc. Scienomics, Scientific Therapeutics, Cognimed Inc, SA Boney and Associates, GlaxoSmithKline, and Double Helix Development Inc. K. S. Elward is a speaker on guidelines implementation for Merck; has received research support from the National Asthma Control Initiative; is a member of the Virginia Asthma Coalition; and is a member of the National Asthma Education and Prevention Program Coordination Committee. M. Kattan has received research support from the NIH-NIAID. The rest of the authors declare that they have no relevant conflicts of interest.

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Asthma outcomes: Symptoms

Background

Objective

Methods

Results

Conclusions

Section snippets

Definitions

Review of specific asthma symptom questionnaires

Electronic diaries

SFDs

Chest

J Allergy Clin Immunol

Ann Allergy Asthma Immunol

Chest

J Allergy Clin Immunol

J Allergy Clin Immunol

Ann Allergy Asthma Immunol

J Allergy Clin Immunol

J Allergy Clin Immunol

J Allergy Clin Immunol

Health Policy

Chest

J Adolesc Health

Chest

J Allergy Clin Immunol

J Allergy Clin Immunol

Measurement characteristics of two asthma symptom diary scales for use in clinical trials

Eur Respir J

Validation of an asthma symptom diary for interventional studies

Arch Dis Child

Quality of care for childhood asthma: estimating impact and implications

Pediatrics

An official American Thoracic Society/European Respiratory Society statement: asthma control and exacerbations: standardizing endpoints for clinical asthma trials and clinical practice

Am J Respir Crit Care Med

Outcomes validity and reliability of the modified Rankin scale: implications for stroke clinical trials: a literature review and synthesis

Stroke

Relation of the perception of airway obstruction to the severity of asthma

Thorax

The Childhood Asthma Management Program Research Group

N Engl J Med

Measuring quality of life in children with asthma

Qual Life Res

Montelukast dose selection in children ages 2 to 5 years: comparison of population pharmacokinetics between children and adults

J Clin Pharmacol

Randomized trial of the effect of drug presentation on asthma outcomes: the American Lung Association Asthma Clinical Research Centers

J Allergy Clin Immunol