Endocrine Abstracts

Background: Osteoporosis and vertebral fracture development are common in young people with Duchenne muscular dystrophy (DMD) and can lead to chronic back pain and reduced quality of life. The current standards of care recommend initiation of bisphosphonate treatment following identification of fractures. Given the extent of fractures in these young people, initiation of treatment prior to fractures seems logical. This study aimed to explore the opinions of young people with DMD, parents/carers and healthcare professionals on the acceptability of bisphosphonates prior to first fracture.

Methods: Young people with DMD (aged 7-18 years) could choose how to share their views including; activity booklets, semi-structured interviews and arts-based augmented interviews. Parents/carers were involved in semi-structured interviews and healthcare professionals participated in online focus groups. Data were analysed using a framework analysis approach.

Results: Fifty-one participants shared their views, including 4 young people (4 male, mean age 15.8 years, range 15-17 years), 20 parents/carers and 27 healthcare professionals. Three themes were identified representing a continuum of opinions towards the endorsement of preventative treatment: 1) Endorsement of preventative bisphosphonate treatments, 2) Uncertainty about preventative bisphosphonate treatments and 3) Reluctance to use bisphosphonate treatments preventatively. Generally, young people and parents were keen to be offered treatments that could protect their child’s bone health and delay or prevent fractures. Professionals commented that preventative treatment ‘made sense’. Young people, parents and professionals were uncertain whether treatments would benefit all young people with DMD (e.g. depending on clinical status). Parents and healthcare professionals felt that further evidence on the risks, benefits and efficacy of preventative bisphosphonates would address uncertainties about using this approach. Some parents and healthcare professionals expressed concerns about side effects and recommended a holistic approach to bone protection (e.g. managing nutrition, puberty and mobility).

Discussion: Findings highlight a continuum of opinions regarding provision of bisphosphonate treatment prior to fracture. Acceptance of preventative bisphosphonate treatment is a personal decision influenced by the individual circumstances of each family. However, there was a consistent call for further research into the risk, benefits and efficacy of using preventative bisphosphonates in this population to inform clinical practice.