Development and initial validation of the assessment of caregiver experience with neuromuscular disease.

Background: Orthopaedic intervention can have a wide range of functional and psychosocial effects on children with neuromuscular disease (NMD). In the multihandicapped child (Gross Motor Classification System IV/V), functional status, pain, psychosocial function, and health-related quality of life also have effects on the families of these child. The purpose of this study is to report the development and initial validation of an outcomes instrument specifically designed to assess the caregiver impact experienced by parents raising severely affected NMD children: the Assessment of Caregiver Experience with Neuromuscular Disease (ACEND).

Methods: In the first part of this prospective study, 61 children with NMD and their parents were administered a range of earlier validated pediatric health measures. A framework technique was used to select the most appropriate and relevant subset of questions from this large set. Sensitivity analyses guided the development of a master question list measuring caregiver impact, excluding items with low relevance, and modifying unclear questions. In the second part of the study, the ACEND was administered to the caregivers of 46 children with moderate-to-severe NMD. Statistical analyses were conducted to determine validity of the instrument.

Results: The resulting ACEND instrument included 2 domains, 7 subdomains, and 41 items. Domain 1, examining physical impact, includes 4 subdomains: feeding/grooming/dressing (6 items), sitting/play (5 items), transfers (5 items), and mobility (7 items). Domain 2, which examines general caregiver impact, included 3 subdomains: time (4 items), emotion (9 items), and finance (5 items). Mean overall relevance rating was 6.21 ± 0.37 and clarity rating was 6.68 ± 0.52 (scale 0 to 7). Multiple floor effects in patients with GMFCS V and ceiling effects in patients with GMFCS III were identified almost exclusively in motor-based items. Virtually no floor or ceiling effects were identified in the time, emotion or finance domains across GMFCS level.

Conclusions: The initial validation demonstrated that ACEND is a valid, disease-specific measure to quantify experience on caregivers of children with NMD. Larger groups of patients across NMD disease type are currently being tested to strengthen validity findings. Additionally, the ACEND is now being administered before and after orthopaedic interventions to determine responsiveness, which is critical to health outcomes research. LEVEL OF EVIDENCE/RELEVANCE: IIc.