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Endocrine Abstracts (2021) 78 OC9.5 | DOI: 10.1530/endoabs.78.OC9.5

BSPED2021 Oral Communications Oral Communications 9 (6 abstracts)

Variability of advice and education for steroid sick day dosing in Duchenne Muscular Dystrophy: Results of a UK patient survey

S Thakrar 1 , MS Cheung 2 , A Johnson 1 , H Katugampola 3 , A Manzur 4 , F Muntoni 4 , T Mushtaq 5 , C Turner 6 , C Wood 7 , M Guglieri 6 & SC Wong 8


1Duchenne UK, London, United Kingdom; 2Department of Endocrinology and Diabetes, Evelina London Children’s Hospital, London, United Kingdom; 3Department of Paediatric Endocrinology, Great Ormond Street Hospital for Children, London, United Kingdom; 4Dubowitz Neuromuscular Centre, Great Ormond Street Hospital for Children, London, United Kingdom; 5Department of Paediatric Endocrinology, Leeds Teaching Hospital, Leeds, Leeds, United Kingdom; 6John Walton Muscular Dystrophy Research Centre, Newcastle, United Kingdom; 7Department of Paediatric Endocrinology, Royal Victoria Infirmary, Newcastle, United Kingdom; 8Department of Paediatric Endocrinology, Royal Hospital for Children, Glasgow, United Kingdom


Introduction: The use of long-term oral corticosteroids in DMD leads to secondary adrenal insufficiency. The 2018 international care consensus recognizes this important issue and recommends emergency plans to be in place.

Aim: This online UK-wide patient survey aims to determine the advice/education given for emergency stress dose plans and the impact of the COVID-19 pandemic in corticosteroid-treated young people with DMD.

Methods: The survey was circulated between November 2020 and January 2021. Results were presented as median (range).

Results: A total of 130 responses were obtained from 129 parents/guardians of a person with DMD. One parent provided responses for twins. Median age of the person with DMD was 11.5 years (5, 26). Eighty-three of 130(64%) were on daily corticosteroids. Sixty of the 130(46%) had access to hydrocortisone for injection. Of those 60, 42/60(70%) had received some form of training. The training involved a practice injection in 34/42(81%). Thirty-four of 42(81%) who received training felt confident about administering an injection as opposed to 9/18(50%) of those who did not receive any training. Forty-one of 42(98%) who received training were clear on when injectable hydrocortisone is needed as opposed to 14/18(78%) of those who did not receive training. Prior to the COVID-19 pandemic [February 2020], 43/130(33%) were provided with an oral emergency stress dose plan for mild-moderate acute illness. After February 2020, the number of patients who were given an oral stress dose plan increased to 66/130(51%). A total of seven oral stress dose plans were identified for those on daily corticosteroid and a greater number of plans in those on the intermittent regimen (during the on and off days).

Conclusion: Despite the known risk of adrenal crisis in patients who are treated with long-term corticosteroid and recent international guidance, emergency sick day plans were not consistently in place in this national survey of boys with DMD. Solutions must take a multi-dimensional and multi-disciplinary approach. Guidance at a national level and the development of structured education for patients and hospital clinicians maybe steps towards improving clinical outcomes.

Volume 78

48th Meeting of the British Society for Paediatric Endocrinology and Diabetes

Online, Virtual
24 Nov 2021 - 26 Nov 2021

British Society for Paediatric Endocrinology and Diabetes 

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