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September 19, 2025Journal of Pediatric Endocrinology and Metabolism0 citations

Testosterone in Duchenne muscular dystrophy: effects on puberty and growth

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CMCara McCauleyRSRachel SmillieJDJennifer Dunne

Key Points

  • Testosterone therapy enhanced long bone growth in boys with DMD but did not achieve full catch-up growth.
  • At baseline, 91% of boys were pre-pubertal, with 77.3% reaching early puberty stages after therapy.
  • Both intramuscular and topical testosterone promoted secondary sexual characteristics without significant side effects.
  • Boys included in the study enjoyed mean testosterone initiation at age 14.05, ensuring timely interventions.

Abstract

Abstract Objectives Testosterone therapy is recommended for management of puberty from the age of 12 years in boys with Duchenne muscular dystrophy (DMD). Height measurement is problematic in these adolescent boys as lower limb contracture can be common and estimated height from segmental body part measurements may over-estimate height. Methods Bone growth was assessed using metacarpal and phalangeal measurements from hand radiographs. Raw length measurements were converted to Z-scores based on published normative data and compared by calculating the mean of each patient as ‘composite bone length Z-score’. Biochemical data was also collected to monitor the safety of testosterone therapy. Results A total of 22 boys with DMD on daily glucocorticoid were included in the study. Mean age at commencement of testosterone therapy was 14.05 (SD 1.20). 13 boys were commenced on intramuscular testosterone therapy and nine were commenced on topical testosterone therapy. At baseline, 20/22 (91 %) were pre-pubertal (G1). At follow-up, 16/22 (77.3 %) were at early stages of puberty (G2 or G3) and 6/22 (22.7 %) were at late stages of puberty (G4 or G5). At baseline and follow up, composite bone length Z-scores for chronological age were significantly lower than zero, −2.7 (SD 1.07) (p<0.001 vs. zero) and −3.3 (SD 1.17), (p<0.001 vs. zero), respectively. There were no statistically significant differences between the topical and IM group at baseline (p=0.61) or 1 year (p=0.40). Conclusions Measurements of metacarpophalangeal bone length confirms that testosterone therapy enhanced long bone growth in boys with DMD but does not achieve full catch-up growth. Both IM and topical testosterone effectively promote secondary sexual characteristics with no significant side effects.

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Cite This Study

McCauley et al. (2025) studied this question.

synapsesocial.com/papers/68d464ea31b076d99fa63ef1https://doi.org/10.1515/jpem-2025-0329
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