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Table 5 Characteristics, assessment, and results of structural validity papers in DMD samples for measures included in the review

From: Measuring quality of life in Duchenne muscular dystrophy: a systematic review of the content and structural validity of commonly used instruments

PROM Reference Country (language) Patient characteristics COSMIN Quality Rating Analysis – model Results (summary)
N Age (yr, M ± SD) % ambulatory PROM score
PedsQL 4.0 GCS Lim et al. 2014 [72] USA (English) 63 boys with DMD (and up to 50 parents, not necessarily matched) 10.2 ± 2.5 95.24 Child: M = 64.5, SD = 15.3.
Parent: M = 56.2, SD = 12.9.
Doubtful Rasch (model not specified) Model misfit for items determined with infit > 1.4 and outfit > 2.0 MnSq values and standardized scores > 2.0. All items fit in parent proxy-reports of physical health scale and child self-reports of psychosocial health scale. 2 out of 8 items showed high infit statistics in child self-reports of the physical health scale (taking a bath or shower; doing chores around the house). In addition 2 out of 15 items showed high infit for the parent proxy-reports of the psychosocial health scale (trouble sleeping; keep up with school work).
PedsQL 3.0 NMM Landfeldt et al. 2018 [66] UK / USA
(English)
278 (95 UK) 16 ± 7 40% not “full-time wheelchair dependent” Not reported Very good Rasch PCM Eight items displayed inadequate fit (χ2: p > 0.01). Six items had fit residuals ≤ −2.5 or ≥ 2.5 (4 significant at p < .05). Inadequate overall fit (χ2 item-trait interaction: p = < .001). Disordered thresholds for 22 of 25 items. Suboptimal targeting.
  1. PROM = patient reported outcome measure; COSMIN = COnsensus-based Standards for the selection of health Measurement INstruments