Searchable abstracts of presentations at key conferences in endocrinology
Endocrine Abstracts (2023) 95 OC6.1 | DOI: 10.1530/endoabs.95.OC6.1

BSPED2023 Oral Communications Oral Communications 6 (5 abstracts)

An audit on improving the endocrine management of patients with Duchenne Muscular Dystrophy

Martha McKenna , Kim Stevenson , Emma Campbell , Isobel Douglas , Karen Thompson , Noina Abid & Sandya Tirupathi

Royal Belfast Hospital for Sick Children, Belfast, United Kingdom

Introduction: Duchenne Muscular Dystrophy (DMD) is an X-linked recessive neuromuscular disorder causing progressive muscular degeneration and weakness. The survival of patients with DMD has improved with multi-disciplinary team input. Sub-specialties have moved towards anticipatory diagnostic and therapeutic strategies which focus on prevention and treatment of modifiable disease complications. Endocrine complications of DMD include impaired growth, delayed puberty, adrenal insufficiency and low bone mineral density (BMD).

Methods: A retrospective review of all patients with DMD in the Belfast Trust was undertaken by reviewing patient records and comparing with the NorthStar recommendations. We reviewed if patients had steroid emergency plan and if a pubertal assessment had taken place. Bone health was assessed by regular vitamin D monitoring and supplementation, surveillance imaging and appropriate treatment of low BMD. A series of actions were made based on 2021 audit findings. This included a standardised referral letter to endocrinology for surveillance of puberty and bone health at 10 years of age. An information letter was produced for patients and parents to educate on adrenal insufficiency with an action plan for sick day rules and training for IM hydrocortisone administration. Re-audit was undertaken in 2023 to assess progressResults*Undertaken in patients referred to the endocrine team

Number of patients4544
Age range (years)5-172-18
Steroids use N(%)40/45(89%)37/44(84%)
Steroid emergency plan/IM hydrocortisone training N(%)2/40(5%)34/37(92%)
Vitamin D N(%) Prescription Regularly monitored Up to date surveillance imaging N(%) DEXA Lateral spine x-ray Compression fracture N(%) Zolendronic acid treatment N(%) 41/45(91%) 11/45(24%) 26/45(58%) 0/45(0%) 5/45(11%) 4/45(9%)41/44(93%) 35/44(80%) 40/44(91%) 37/44(84%) 13/44(30%) 13/44(30%)
Endocrine referral >10 years of age4/39(10%)34/38(89%)
Pubertal assessment N(%)* Treatment for delayed puberty N(%)*3/4(75%) 2/4(50%)34/34(100%) 11/34(32%)
*Undertaken in patients referred to the endocrine team

Conclusion: An improvement has been demonstrated in the management of endocrine complications associated with DMD but there are still areas which require further work. Ongoing multi-disciplinary input and partnership is required to optimise management for patients with DMD.

Volume 95

50th Annual Meeting of the British Society for Paediatric Endocrinology and Diabetes

Manchester, UK
08 Nov 2023 - 10 Nov 2023

British Society for Paediatric Endocrinology and Diabetes 

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