Influence of gait analysis on decision-marketing for lower extremity surgery

The Pediatric Quality of Life Inventory in Duchenne muscular dystrophy: correlations with disease progression

E K HENRICSON A, C M MCDONALD A, J J HAN A, R T ABRESCH A,
D M ESCOLAR B, AND THE CINRG INVESTIGATORS

  1. 1. University of California Davis, Davis, CA;
  2. 2. Children’s National Medical Center, Washington, DC, USA

Background: The Pediatric Quality of Life Inventory (PedsQL) 1, is a self-assessment tool that measures physical and psychosocial function in pediatric populations. It is validated in general and disease-specific scales in many pediatric disorders, and normative data is available demonstrating that the device detects disease progression and effect of therapies.

Objectives: In the muscular dystrophies, there is academic and professional consensus on the need to define whether existing quantitative strength efficacy assessments used as study endpoints relate to clinically meaningful  changes in self-reported quality of life or functional assessments.

Design: This is a cross-sectional analysis of quality of life and strength data collected during baseline screening visits of clinical trials and a longitudinal natural history study of Duchenne muscular dystrophy.

Participants: We evaluated 79 children with confirmed Duchenne muscular dystrophy between the ages of 5 and 18 years at 11 centres of the Cooperative International Neuro-muscular Research Group (CINRG).

Method: Participants completed age-appropriate PedsQL general assessments. They were grouped according to device age ranges as young children (YC, 2-4y; n=10), children (c, 5-12y, n=55), or adolescents (A, 13-18y; n=14). Subscale and total scores were calculated and compared between groups. Participants underwent timed motor performance evaluation (time to stand, climb four steps, walk/run 10m) and manual muscle testing (MMT) using a modified MRC scale.

Results: PedsQL physical function mean (SD) score differed significantly between all groups (YC, 62.18 [SD 5.99]), (C, 45.08 [SD 2.55]), (A, 31.69 [SD 5.06]; p=0.00-0.02).  PedsQL psychosocial function score differed significantly between young children (72.66 [SD 4.45]) and children (61.66 [SD 1.90], p=0.02). PedsQL total score for young children (69.02 [SD 4.65]) was significantly different from scores for children and adolescents (C, 55.89 [SD 1.98], p=0.01), (A, 55.74 [SD 3.91], p=0.03). The PedsQL physical function subscale correlated moderately with time to stand (r=0.41) and time to clim,b steps (r=-0.42), and showed a weaker relationship with time to walk 10m (r=-0.28). The physical function sub-scale showed a weak relationship with MMT (r=0.165).

Conclusions: The PedsQL instrument shows a significant differences primarily associated with decrease in physical function due to disease progression in Duchenne muscular dystrophy. Physical function differences contribute to a statistically significant difference in total score. Lesser differences exist in psychosocial function. The physical function subscale of the instrument correlates moderately with timed motor performance evaluation.

Reference: Varni JW, Seid M, Rode CA. (1999) The PedsQL: measurement model for the pediatric quality of life inventory. Med Care 37: 126-139.

 
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