Endocrine Abstracts

Objectives: Current international Care Considerations for Duchenne Muscular Dystrophy (DMD) recommend initiation of bisphosphonate following first fracture. Given the extent and burden of osteoporosis, some have advocated initiating therapy prior to fracture, although this is not standard practice. Our objective was to investigate the current clinical practice of clinicians managing osteoporosis and their opinion on treatment prior to fracture in DMD.

Methods: An online survey was circulated to paediatric clinicians involved in management of osteoporosis in DMD via patient groups in four countries (UK, USA, Italy, Israel).

Results: A total of 51/105 (48%) responses were received. The commonest indications for starting bisphosphonate were vertebral fracture (VF) of any grade without back pain (44/51,86%) or long bone fracture (34/51,67%). 18% (9/51) would initiate treatment without any fracture. IV Zoledronate was the most common agent (44/51,86%) used following fracture, with 7/51 (14%) responders opting to prescribe oral bisphosphonate. 9/51 (18%) currently use other non-bisphosphonate agents (Denosumab, Teriparatide, Romosozumab) following fracture. Of those who prescribe IV bisphosphonate, 26 (52%) prescribe sick-day steroid with first infusion and 18 (36%) with subsequent infusions; 44 (88%) prescribe oral calcium supplements with first infusion and 39 (78%) with subsequent infusions; 32 (64%) prescribe anti-pyretics with first infusion and 19 (38%) with subsequent infusions. 28 (56%) adjust the dose of subsequent infusions and 30 (60%) alter frequency of subsequent infusions based on bone density. Of those who transitioned patient care to adult bone specialists (n = 36), 21 (58%) continue bisphosphonate during this process, 4 (11%) stop treatment and 7 (19%) develop individualised plans in transition review with an adult bone specialist. Of the 51 responders, 20 (39%) are of the opinion that bisphosphonates should be initiated prior to fracture, 24 (47%) feel there may be a role and 7 (14%) do not see a role.

Conclusions: This international clinician survey identified variation in management of osteoporosis in DMD. Expert recommendations on management of side-effects of IV bisphosphonate, the role of non-bisphosphonate therapies, management during transition and longer-term osteoporosis treatment in adulthood is greatly needed. There was no consistent view on initiation of osteoporosis therapy prior to fracture in DMD.