P141 Concordance of patient-reported outcomes measurement information system (PROMIS) questionnaires between caregivers and children with DMD

• Published in: Neuromuscular disorders : NMD 2023-10, Vol.33, p.S82-S83
• Main Authors: Audhya, I., Patel, S., LeReun, C., Alfano, L., Reash, N., Iammarino, M., Lowes, L.
• Format: Article
• Language: English
• ISSN: 0960-8966; 1873-2364
• DOI: 10.1016/j.nmd.2023.07.076

PROMIS includes patient-reported outcomes that quantify the impact of disease on physical, social, or cognitive function. While self-reporting is considered the gold standard, caregivers frequently report on behalf of patients in many disease states, including Duchenne muscular dystrophy (DMD). PROMIS Parent Proxy (PP) questionnaires are being used in DMD studies, with caregivers rating their child's functional ability. Agreement between caregiver and child on the PROMIS Mobility and Upper Extremity (UE) questionnaires remains largely unknown for the DMD community. This analysis evaluated the appropriateness of using caregivers of patients with DMD as proxies for these questionnaires. PROMIS Mobility (v1.0; n=41) and UE (v1.0; n=94) were administered to dyads at one USA health care center. Inter-rater reliability between caregivers and children on overall PROMIS raw scores was assessed using intraclass correlation coefficient (ICC). Degree of agreement between caregiver and child responses on individual PROMIS items was assessed using Gwet coefficient. ICC (95% CI) for overall PROMIS Mobility and UE scores are moderate 0.555 (0.304–0.735) and poor 0.413 (0.231–0.567), respectively. Caregiver and child ratings for each item showed substantial to perfect agreement for 10/23 (43%) and moderate agreement for 8/23 (35%) Mobility items. For UE, 17/29 items (59%) demonstrated substantial to perfect agreement and 7/29 (24%) moderate. Poor ICC observed for the UE overall score is likely attributable to differences in how corresponding items are scored between the PP and child questionnaire versions. Agreement was stronger within caregiver/child dyads when assessed item by item for both questionnaires, indicating substantial to perfect concordance for most items. This analysis found caregivers and their children have related yet unique perspectives. Based on these findings, caregivers can be considered suitable proxies for children when rating PROMIS Mobility and UE function.