ESPE Abstracts (2021) 94 P2-5

ESPE2021 ePoster Category 2 Adrenals and HPA Axis (57 abstracts)

Glucocorticoid side-effects in Duchenne Muscular Dystrophy: Systematic review of side effects in published literature and a survey of the concerns of the patient community

Caleb Hariri 1 & Sze Choong Wong 2


1School of Medicine, University of Glasgow, Glasgow, United Kingdom; 2School of Medicine, University of Glasgow, Department of Paediatric Endocrinology, Royal Hospital for Children, Glasgow, Glasgow, United Kingdom


Background: Long-term use of glucocorticoid for management of Duchenne muscular dystrophy (DMD) has been standard of care for over 20 years with documented improvement in outcomes. Side-effects are common particularly endocrine and bone adverse effects. To date, there is limited information on the perception of glucocorticoid side effects by parents/guardians and those living with DMD.

Aims:

• To identify glucocorticoid side-effects investigated in published literature for boys with DMD.

• To identify the patient perception of glucocorticoid side-effects.

Methods: A systematic literature search was performed using MEDLINE, EMBASE and Cochrane Library (January 2021). An online survey was circulated via UK-based patient groups for a period of 5 weeks. Responders were asked to rate their concern of glucocorticoid side-effects on a scale of 0 to 10(0: not concerned to 10: extremely concern). Ethics approval was sought from the University of Glasgow Ethics Committee. Continuous data expressed as median (25th,75th centile).

Results: Sixty-one publications were identified from the systematic search. Fifty-three glucocorticoid side-effects were considered in those publications. The top three side-effects considered were: weight gain/obesity (43/61; 71%), poor growth/short stature (38/61; 62%) and blood pressure abnormalities (25/61; 41%). Thirty-three parents/guardians responded to the online survey. Median age of the person with DMD was 10 years (6,13) with median age of starting glucocorticoid at 5 years (4, 6). Thirty-one out of 33 are still on glucocorticoid; two had discontinued therapy due to concerns about side-effects and three who are still on treatment were no longer happy to continue glucocorticoid. The top three glucocorticoid side-effects experienced by the young person with DMD as reported by parents/guardians were: poor growth/short stature (25/33; 76%), cushingoid features (21/33; 64%), and weight gain/obesity (19/33; 58%). Glucocorticoid side-effects which concern parents/guardian the most were: long bone fracture [median score 9 (7,10)], vertebral fractures [median score 9 (7, 10)], weight gain/obesity [median score 8(7,9)] and delayed puberty [median score 8 (5,9)]. Glucocorticoid side-effects which concern the young person the most were: short stature [median score 7(2,9)], weight gain/obesity [median score 7(1, 8.8)] and cushingoid features [median score 5.5(1, 8.8)].

Conclusion: This study identified for the first time that endocrine/bone side effects concern parents/guardians and young people with DMD greatly. Research on therapies/DMD treatment that may eliminate or minimize such side effects are needed, strongly supported by concerns raised by the patient community.

Volume 94

59th Annual ESPE (ESPE 2021 Online)

Online,
22 Sep 2021 - 26 Sep 2021

European Society for Paediatric Endocrinology 

Browse other volumes

Article tools

My recent searches

No recent searches.