ESPE Abstracts

Background: Long-term glucocorticoid therapy with Prednisolone or Deflazacort has improved outcomes in patients with Duchenne Muscular Dystrophy (DMD), however recommended dosages suppress the hypothalamic-pituitary-adrenal axis, leading to adrenal insufficiency. All boys prescribed glucocorticoid therapy should be assumed to have adrenal suppression, and therefore at risk of adrenal crisis during illness or stress (eg. surgery, bisphosphonate infusions). The updated DMD Care Considerations consensus document (2018) recommends implementation of emergency plans. Despite this, no unified national or international guidance for illness management for this cohort exists. Previously emergency sick day plans were not consistently in place within our own specialist centre, and on national survey of 6 other Trusts, it was clear that advice and education for sick day steroid regimens was variable.

Aim: We sought to develop a weight-based steroid emergency regimen for DMD patients, many of whom are non-ambulant, and in whom accurate measurements for body surface area can be challenging.

Methods: A novel weight-based regimen of stress doses of oral hydrocortisone given 6 hourly was devised. This was to be implemented when needed, in addition to the glucocorticoid regimen the patient was taking for disease (prednisolone/deflazacort, daily/pulsed therapy).

Results: Approval from the Drugs and Therapeutics committee was obtained for a weight-based stress dose regimen of hydrocortisone, which was implemented in this cohort from April 2020. Additionally, training and education was provided to increase awareness of adrenal insufficiency among patients and families. All DMD patients with prescribed steroid therapy have written documentation of an emergency steroid plan to be followed during acute illness. All families and boys are educated about stress dosing plans, recognising signs/symptoms of adrenal crisis, and have been trained to give IM Hydrocortisone injections. Adrenal ‘Flags’ have been added to our electronic patient records alerting staff that these patients are at risk of adrenal crisis.

Conclusion: We are currently reviewing the results of our practice change. Anecdotally families report faster recovery times from illness since using stress-doses of hydrocortisone alongside their usual glucocorticoid. Previous regimens utilising a patient’s usual disease-modifying glucocorticoid may not have taken into account the pharmacokinetics of these drugs; Deflazacort for example has a rapid plasma elimination half-life of 1-2 hours. The development of a unified, consistent approach to illness management, alongside structured education for DMD patients and families, are initial actions towards improving clinical outcomes. Further guidance at a national and international level is needed.