Cognitive Debrief of the PROMIS Parent Proxy Mobility Item Bank in Caregivers of Children Living With Duchenne Muscular Dystrophy
Author(s)
Chloe Carmichael, MSc1, Ania Filus, PhD2, Payton Ramsey, MPH1, Alise Nacson, MPH2, Hannah Edge, MRes1, Elizabeth Collins, PhD1, Helen Kitchen, MSc1, Ivana Filipovic Audhya, MSc2.
1Clarivate, London, United Kingdom, 2Sarepta Therapeutics, Inc., Cambridge, MA, USA.
1Clarivate, London, United Kingdom, 2Sarepta Therapeutics, Inc., Cambridge, MA, USA.
OBJECTIVES: Duchenne muscular dystrophy (DMD) is an X-linked, rare, debilitating neuromuscular disease characterized by progressive muscle deterioration. This study explored the content validity of the Patient-Reported Outcomes Measurement Information System (PROMIS) Parent Proxy Mobility item bank (version 2.0) to measure caregiver-reported relevant experiences of pediatric patients with DMD.
METHODS: Caregivers participated in individual, 75-minute, cognitive debriefing interviews to assess the content validity of the PROMIS Parent Proxy Mobility item bank. Framework analysis methodology was used to analyze qualitative data.
RESULTS: Twelve caregivers of children aged 4-14 years old, living with early (n=9) or late (n=3) ambulatory DMD, participated. Caregivers were predominantly female (n=10), and all were White (n=12) and educated at college or associate degree level (n=12). All items were understood and interpreted correctly by at least 80% of caregivers. Most items were conceptually relevant, reflecting activities impacted by the condition. However, some were deemed unsuitable for 4-year-olds (e.g., carrying books in a backpack or running a mile), and others were subject to varied interpretation (e.g., “the activities he/she enjoys the most”, which ranged from physical to sedentary activities). Responses indicated that the activities assessed were observable by caregivers and appropriate for parent proxy reporting. Caregivers also identified several items in the PROMIS Parent Proxy Mobility item bank as particularly bothersome and therefore relevant to their child (e.g., an (in)ability to play with other children, difficulty doing sports, or riding a bike).
CONCLUSIONS: Most items in the PROMIS Parent Proxy Mobility item bank demonstrated adequate content validity and may be fit-for-purpose in an early and/or late ambulatory DMD population. By obtaining direct feedback from caregivers, cognitive debriefing helped confirm comprehension of items and identify potential relevance gaps. These insights can help guide item selection to more effectively capture patient experiences that are relevant to the individual and appropriate for their disease stage.
METHODS: Caregivers participated in individual, 75-minute, cognitive debriefing interviews to assess the content validity of the PROMIS Parent Proxy Mobility item bank. Framework analysis methodology was used to analyze qualitative data.
RESULTS: Twelve caregivers of children aged 4-14 years old, living with early (n=9) or late (n=3) ambulatory DMD, participated. Caregivers were predominantly female (n=10), and all were White (n=12) and educated at college or associate degree level (n=12). All items were understood and interpreted correctly by at least 80% of caregivers. Most items were conceptually relevant, reflecting activities impacted by the condition. However, some were deemed unsuitable for 4-year-olds (e.g., carrying books in a backpack or running a mile), and others were subject to varied interpretation (e.g., “the activities he/she enjoys the most”, which ranged from physical to sedentary activities). Responses indicated that the activities assessed were observable by caregivers and appropriate for parent proxy reporting. Caregivers also identified several items in the PROMIS Parent Proxy Mobility item bank as particularly bothersome and therefore relevant to their child (e.g., an (in)ability to play with other children, difficulty doing sports, or riding a bike).
CONCLUSIONS: Most items in the PROMIS Parent Proxy Mobility item bank demonstrated adequate content validity and may be fit-for-purpose in an early and/or late ambulatory DMD population. By obtaining direct feedback from caregivers, cognitive debriefing helped confirm comprehension of items and identify potential relevance gaps. These insights can help guide item selection to more effectively capture patient experiences that are relevant to the individual and appropriate for their disease stage.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
PCR45
Topic
Patient-Centered Research
Topic Subcategory
Instrument Development, Validation, & Translation, Patient-reported Outcomes & Quality of Life Outcomes
Disease
Musculoskeletal Disorders (Arthritis, Bone Disorders, Osteoporosis, Other Musculoskeletal), Pediatrics, Rare & Orphan Diseases