Endocrine Abstracts

Objectives: DMD Care UK (www.dmdcareuk.org) is a national project initiated and funded by Duchenne UK in collaboration with Newcastle University and the UK North Star clinical network. The main aim is to facilitate implementation of care standards across the UK national health system. In 2021, DMD Care UK conducted a comprehensive online family survey of all aspects of care of boys with DMD with the aim of capturing the patients experience. Here, we present results relating to bone-endocrine care.

Methods: An online survey was circulated in May 2021.

Results: 164 parents/carers responded to the survey. Median age of the young person with DMD was 11 years (Range 2,46). 32/164(20%) were 18 years and older. 78/164(48%) have been seen by an endocrinologist or recently referred. Vitamin D levels were not checked regularly in 27/164(16%). 61/164(37%) have not ever had lateral spine imaging to screen for VF. 48/164(29%) undergo spine imaging every 12 months,14/164(9%) every 2 years, 34/164(21%) every 3 years or longer. 30/164(18%) have never had a DXA for assessment of bone density. Only 6/62(10%) of adolescents aged 13-18 years had received testosterone therapy. 13/124(11%) on steroids were not aware or unsure of emergency steroid sick day dosing plans. 66/164(40%) were very satisfied or satisfied with endocrine/bone care, with 18/164(11%) who were dissatisfied or very dissatisfied. Feedback on areas that are important to the patients and influences satisfaction include a regular bone healthmonitoring programme, timely assessment of puberty, open discussions of hormone treatment for puberty/growth and clear instructions on steroid sick day dosing plans.ConclusionThis first national survey of bone-endocrie management in DMD demonstrates variability despite the 2018 international guidance. A consistent bone health monitoring programme, timely assessment of puberty, open discussions of hormone treatment (testosterone and growth hormone) and clear instructions on management of steroid during illness are of great importance to patients in this bone-endocrine care. These are priorities to be addressed by DMD Care UK, and important points to be considered in service development.