BSPED2021 Oral Communications Oral Communications 9 (6 abstracts)
1Duchenne UK, London, United Kingdom; 2Department of Endocrinology and Diabetes, Evelina London Childrens 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.