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Improvements in health status and utility associated with ataluren for the treatment of nonsense mutation Duchenne muscular dystrophy
Author(s) -
Landfeldt Erik,
Lindberg Christopher,
Sejersen Thomas
Publication year - 2020
Publication title -
muscle and nerve
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.025
H-Index - 145
eISSN - 1097-4598
pISSN - 0148-639X
DOI - 10.1002/mus.26787
Subject(s) - medicine , ambulatory , duchenne muscular dystrophy , physical therapy , nonsense mutation , visual analogue scale , neuromuscular disease , health care , ambulatory care , pediatrics , disease , mutation , biochemistry , chemistry , missense mutation , economic growth , economics , gene
Little is known of the impact of ataluren on health status and utility in patients with nonsense mutation Duchenne muscular dystrophy (nmDMD). In this work we sought to investigate the clinical expert consensus of these topics in a Delphi panel study. Methods Six Swedish neuromuscular experts participated in this study. Consensus was investigated for responses to the Health Utilities Index (HUI) and a visual analog scale (VAS) for ambulatory and nonambulatory patients treated with ataluren plus best supportive care vs best supportive care alone. Results Consensus was obtained after three panel rounds across treatments and disease stages, except for HUI question 10 (ability to use hands and fingers) for nonambulatory patients. Utility differences between treatments were 0.31 for ambulatory patients, and 0.15 to 0.18 for nonambulatory patients, respectively. The corresponding VAS differences were 12 and 13. Discussion The outcomes of this study support the association of ataluren for the treatment of nmDMD with improved health status and utility.