Endocrine Abstracts

Background: Osteoporosis commonly occurs as a result of long-term glucocorticoid use and muscle weakness in individuals with Duchenne muscular dystrophy (DMD), rendering them highly susceptible to fragility fractures of vertebrae and long bones. Existing clinical guidelines for the management of osteoporosis in DMD primarily focus on paediatric management. In particular, management during transition from paediatric to adult care is not clarified in current international guidance.

Objective: A UK working group was set up to establish a national clinical consensus for the management of osteoporosis in adult patients with DMD with focus on transition from paediatric to adult care.

Method: An expert panel comprising of adult and paediatric bone specialists, adult neuromuscular clinicians, and patient representatives was assembled to develop the clinical consensus through a series of online meetings. Focus groups involving patients and their caregivers were organised to gather feedback on their experience and the management of bone health. Systematic and scoping reviews on osteoporosis therapies for DMD, international consensus guidelines for glucocorticoid-induced osteoporosis in adults, and DXA predictions of fractures in DMD were conducted to inform the expert panel. In the initial round, the expert panel proposed areas of clinical care for which consensus statements will be formulated via an online survey. The final consensus statements will be developed using a three-to-four-round e-Delphi technique.

Results: Of the 22 clinicians invited to participate, 19 (86%) provided their suggestions in the first survey round. The suggested areas were categorised into eight main domains, covering various aspects of osteoporosis risk assessment, treatment, and monitoring. The findings from the three literature reviews confirmed the paucity of evidence regarding bone health management in adults with DMD. The results of these reviews and the outcomes of the focus groups were shared with the expert panel in subsequent online meetings before the voting on the consensus statements commences in September 2023. The final clinical consensus is expected to be published in the spring of 2024.

Conclusion: Employing an e-Delphi-based systematic approach, this study endeavours to develop national guidance for managing osteoporosis in adults with DMD in the UK and to guide management during transition.