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Health‐related quality of life in patients with Duchenne muscular dystrophy: a multinational, cross‐sectional study
Author(s) -
Landfeldt Erik,
Lindgren Peter,
Bell Christopher F,
Guglieri Michela,
Straub Volker,
Lochmüller Hanns,
Bushby Katharine
Publication year - 2016
Publication title -
developmental medicine and child neurology
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.658
H-Index - 143
eISSN - 1469-8749
pISSN - 0012-1622
DOI - 10.1111/dmcn.12938
Subject(s) - medicine , quality of life (healthcare) , ambulatory , duchenne muscular dystrophy , physical therapy , health utilities index , disease , population , cross sectional study , neuromuscular disease , gerontology , pediatrics , public health , health related quality of life , environmental health , pathology , nursing
Aim To estimate health‐related quality of life ( HRQOL ) in patients with Duchenne muscular dystrophy ( DMD ). Method HRQOL was assessed using the Health Utilities Index Questionnaire ( HUI ) and the Pediatric Quality of Life Inventory (Peds QL ) neuromuscular module version 3.0 online. Results were stratified by disease stage (early/late ambulatory/non‐ambulatory) and caregivers’ perceptions of patients’ health and mental status. Results A total of 770 patient–caregiver pairs (173 German, 122 Italian, 191 UK , and 284 USA ) participated. Most caregivers (>84%) perceived their patients as happy/somewhat happy and in excellent/very good/good health, irrespective of current ambulatory class. In contrast, mean patient utility (reflecting public preferences: 0, dead; 1, perfect health) deteriorated with disease course, from 0.75 in early ambulatory males to 0.15 in the most severely affected patients. Mean patient Peds QL scores (0–100, higher score indicating better HRQOL ) decreased from 80 to 57 across ambulatory classes. Interpretation HRQOL in DMD , measured through public preferences, is substantially impaired in relation to the general population and significantly associated with disease progression. Still, most patients are perceived as happy and in good health by their caregivers, indicating that influential domains of HRQOL remain intact through the disease progression. Our findings emphasize the challenges in measuring HRQOL in a rare, progressive childhood condition such as DMD .