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. 2025 Apr 25;38(8):807-814.
doi: 10.1515/jpem-2025-0039. Print 2025 Aug 26.

The use of bisphosphonate and testosterone in young people with Duchenne muscular dystrophy: an international clinician survey

Sarah McCarrison  1   2 , Shima Abdelrahman  1 , Justin H Davies  3 , Talat Mushtaq  4 , Raja Padidela  5   6 , Vrinda Saraff  7 , Claire L Wood  8 , Sze Choong Wong  1   2
Affiliations

The use of bisphosphonate and testosterone in young people with Duchenne muscular dystrophy: an international clinician survey

Sarah McCarrison et al. J Pediatr Endocrinol Metab. .

Abstract

Objectives: Delayed puberty and osteoporosis are significant challenges in boys with Duchenne muscular dystrophy (DMD). The current International Care Considerations for DMD recommend bisphosphonate after first fracture and testosterone for delayed puberty from 12 years. This study aimed to investigate the current clinical practices of clinicians managing osteoporosis and testosterone treatment in DMD.

Methods: An online survey was circulated to paediatric clinicians involved in the management of bone health and puberty in DMD via patient groups (UK, USA, Italy, Israel).

Results: A total of 51/105 (48 %) responses were received. For osteoporosis, vertebral fracture of any grade (86 %) and long bone fracture (67 %) were the most common indications for starting bisphosphonates, with IV zoledronate being the most used agent (86 %). Approaches varied in managing bisphosphonate side effects, and transitioning care to adult specialists. Opinions differed on starting bisphosphonate before fracture. Fourty-nine clinicians managing pubertal disorders reported initiating testosterone for delayed puberty typically between 12 and 14 years, with majority prescribing intramuscular injections (96 %). Duration of testosterone therapy varied, with some using short-term courses and others continuing until adulthood.

Conclusions: This international survey highlights variability in the management of osteoporosis and delayed puberty in DMD. Recommendations on management of side-effects of IV bisphosphonate, osteoporosis therapy during transition, and longer-term treatment into adulthood is required. There is a need for further guidance on testosterone therapy, particularly in regard to monitoring after discontinuation and the threshold for re-initiation of treatment.

Keywords: Duchenne muscular dystrophy; bisphosphonate; delayed puberty; osteoporosis; testosterone.

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