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Proxy-reported questionnaires for young children with asthma: a structured review
Amy Barrett1, Marci Clark, Carla Demuro
1RTI Health Solutions, Manchester, UK. abarrett@rti.org
Insights
This study identified and evaluated proxy-reported outcome measures for young children with asthma. The Pediatric Asthma Caregiver Diary showed the strongest measurement properties for clinical trials.
Area of Science:
- Pediatric Pulmonology
- Clinical Trial Methodology
- Health Outcomes Research
Background:
- Accurate assessment of asthma in children under 6 is challenging.
- Validated proxy-reported outcome measures are crucial for clinical trials.
- Existing measures require evaluation for suitability as trial endpoints.
Purpose of the Study:
- To identify proxy-reported outcome measures for asthma in children aged 6 years and younger.
- To assess the measurement properties of these instruments.
- To determine their suitability as endpoints in pediatric asthma clinical trials.
Main Methods:
- A two-phase literature search was conducted using databases like PubMed and EMBASE.
- Instruments were assessed against predefined criteria for relevance and measurement properties.
- A total of 631 records were initially screened, identifying 20 instruments.
Main Results:
- Seven pediatric asthma proxy-reported outcome instruments met the inclusion criteria.
- These included the Childhood Asthma Control Test and the Pediatric Asthma Caregiver Diary.
- Three instruments were deemed suitable for clinical trials, with the Pediatric Asthma Caregiver Diary showing the strongest properties.
Conclusions:
- The Pediatric Asthma Caregiver Diary is a robust measure for assessing asthma in young children.
- This instrument demonstrates strong measurement properties, supporting its use in clinical trials.
- Further validation of pediatric asthma outcome measures is essential for advancing research.
Abstract:
Which proxy-reported outcome measures have been developed for use with children aged 6 years and younger to assess asthma symptoms, asthma control, and asthma-specific health-related quality of life, and do these questionnaires' measurement properties support their use as end-points in clinical trials? A two-phase literature search was conducted: 1) studies describing relevant questionnaires were identified, and the questionnaires were assessed against predefined criteria; 2) studies providing information on the measurement properties of questionnaires meeting the predefined inclusion criteria were identified. Literature sources included PubMed and EMBASE databases, scientific conference proceedings, a clinical trial registry, and a quality of life instrument database. The initial search of literature databases and conference abstracts identified 631 records. 20 paediatric asthma proxy-reported outcome instruments were identified; seven met the inclusion criteria: Childhood Asthma Control Test, Control de Asma en Niños Questionnaire, Pediatric Asthma Caregiver Diary, Pediatric Asthma Control Tool, PedsQL 3.0 Short-Form 22 Asthma Module, PedsQL Asthma Symptoms Scale, and Test for Respiratory and Asthma Control in Kids. Three proxy-reported outcome instruments were considered suitable for use as end-points in paediatric asthma clinical trials; the Pediatric Asthma Caregiver Diary possesses the strongest measurement properties of the three.
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