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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