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Is ongoing testosterone required after pubertal induction in Duchenne muscular dystrophy?

Lookup NU author(s): Dr Claire Wood, Dr Kieren Hollingsworth, Edrina Bokaie, Dr Eric Hughes, Robert Muni Lofra, Dr Anna Mayhew, Professor Michela GuglieriORCiD, Professor Timothy Cheetham, Professor Volker StraubORCiD



This work is licensed under a Creative Commons Attribution 4.0 International License (CC BY 4.0).


Glucocorticoids (GCs) reduce inflammation and preserve muscle fu nction in boys withDuchenne muscular dystrophy (DMD) but cause pubertal delay. Pub ertal induction withtestosterone is recommended but longer-term outcome is unknown.Objective: To assess hypothalamic–pituitary–gonadal axis, muscle volume a nd function 5years after pubertal induction.Methods: A prospective observational follow-up of a clinical study was conducted. 15GC-treated males with DMD were treated with incremental testost erone for 2 years(end of regimen +2 years) then evaluated at +2.5 years and +5 y ears (final follow-up ~3years after last injection). Data collected included testicular volume (TV), gonadotrophin,testosterone, inhibin B, muscle function, and limb muscle MRI.Results: Participants were 18.7 years (s.d. 1.6) at the final follow-up and had been on GCfor 11.2 years (s.d. 2.2). Testosterone levels were similar at +2.5 years (8.6 nmol /L (s.d.3.4) and 5 years (11.0 nmol/L (s.d. 6.1). TV increased from 2.8 mL ( s.d. 0.9) at +2 years to7.1 mL (s.d. 1.8) then 10.6 mL (s.d. 3.5) at +2.5 years and +5.0 years (P < 0.001). InhibinB levels increased from 55.6 pg/mL (s.d. 47.0) at baseline to 158.2 pg/mL ( s.d. 87.6),P =0.004 at 5 years but remained lower than reference values (mean 305 pg/mL). Musclecontractile bulk decreased.Interpretation: Pubertal induction with testosterone in DMD is associated with HPG axisactivation and ongoing increases in inhibin B, TV, and testoste rone concentrations.Some patients have normal levels which is promising regarding f uture fertility. Given thebeneficial impact of testosterone on bone health, muscle, and we ll-being, monitoringtestosterone levels in this population and supplementation of s ub-optimal levels isimportant.

Publication metadata

Author(s): Wood CL, Hollingsworth KG, Bokaie E, Hughes E, Muni-Lofra R, Mayhew A, Mitchell RT, Guglieri M, McElvaney J, Cheetham TD, Straub V

Publication type: Article

Publication status: Published

Journal: Endocrine Connections

Year: 2023

Volume: 12

Issue: 12

Pages: e230245

Print publication date: 26/10/2023

Online publication date: 28/09/2023

Acceptance date: 28/09/2023

Date deposited: 14/11/2023

ISSN (electronic): 20493614

Publisher: Bioscientifica Ltd


DOI: 10.1530/EC-23-0245

PubMed id: 37768006


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Funder referenceFunder name
UKRI Medical Research Council; UKRI Medical Research Counci