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Lookup NU author(s): Dr Claire Wood, Dr Jonathon Foggin, Professor Michela GuglieriORCiD, Professor Volker StraubORCiD, Professor Timothy Cheetham
This work is licensed under a Creative Commons Attribution 4.0 International License (CC BY 4.0).
© 2021 The AuthorsDuchenne muscular dystrophy (DMD) is the most common muscular dystrophy in childhood. It is associated with progressive muscle function decline and premature death. Long-term oral glucocorticoid use slows muscle weakness but is associated with several side effects including delayed puberty. This study assessed the impact of a 2-year incremental intramuscular testosterone regimen on quality of life (QoL) in a cohort of 15 adolescents with DMD. The Pediatric Quality of Life Inventory (PedsQL) Neuromuscular module was used to assess QoL and was completed by parent-child dyads. Semi-structured interviews were carried out to understand patient views on testosterone therapy. QoL scores increased in 10 of the 15 participants during treatment, with a mean total PedsQL score of 74.6 pre-treatment v 80.2 post treatment (p = 0.04). This was supported by comments in the semi-structured interviews. Parent-reported PedsQL scores were lower than their child's post treatment (p = 0.007). Testosterone therapy for pubertal induction was associated with an improvement in QoL and the observed physical changes during puberty played an important role. Low self-esteem was also a prevailing theme. This data supports the inclusion of testosterone therapy for pubertal induction as a Standard of Care.
Author(s): Wood CL, Page J, Foggin J, Guglieri M, Straub V, Cheetham TD
Publication type: Article
Publication status: Published
Journal: Neuromuscular Disorders
Year: 2021
Volume: 31
Issue: 12
Pages: 1259-1265
Print publication date: 01/12/2021
Online publication date: 25/09/2021
Acceptance date: 21/09/2021
Date deposited: 07/06/2022
ISSN (print): 0960-8966
ISSN (electronic): 1873-2364
Publisher: Elsevier Ltd
URL: https://doi.org/10.1016/j.nmd.2021.09.007
DOI: 10.1016/j.nmd.2021.09.007
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