hrp0094p2-5 | Adrenals and HPA Axis | ESPE2021

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

Hariri Caleb , Choong Wong Sze ,

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 glucoc...

hrp0097p1-410 | Adrenals and HPA Axis | ESPE2023

Patient education for management of sick day episodes in adrenal insufficiency: A systematic review of structured education and online patient resources

Bradford Anna , Mason Avril , Choong Wong Sze

Background: Management of adrenal insufficiency(AI) during sick-day episodes require adjustment of oral glucocorticoid therapy or administration of intramuscular injection to prevent adrenal crisis. Education of families of a young person with AI on management during sick day episode is therefore critical.Aim: To critically appraise patient education of sick-day episode management of adrenal insufficiency by conducting</...

hrp0097p1-581 | Sex Differentiation, Gonads and Gynaecology, and Sex Endocrinology | ESPE2023

A dual centre evaluation of discontinuation of testosterone therapy in boys with Duchenne Muscular Dystrophy

Choong Wong Sze , Loughnan Myles , Zacharin Margaret

Background: The 2018 international standards of care for DMD recommend initiating testosterone for management of delayed puberty commencing at a low dose, gradually increasing to adult replacement. No recommendations exist regarding longer term use of testosterone during transition and adulthood.Aim(s): To report long-term use of testosterone in DMD with outcomes of gonadal function and pubertal development in those who ...

hrp0092rfc2.6 | Bone, Growth Plate and Mineral Metabolism Session 1 | ESPE2019

Impact of Pubertal Suppression on Body Composition and Bone Mineral Density in Adolescents with Gender Dysphoria

Shepherd Sheila , Dewantoro Dickson , Gerasimidis Konstantinos , Shaikh M Guftar , Mason Avril , Choong Wong Sze , Kyriakou Andreas

Introduction: Pubertal suppression with gonadotrophin releasing hormone (GnRH) analogue is introduced after the onset of puberty in adolescents with gender dysphoria (GD). As puberty is a critical period for bone accrual and changes in body composition, alterations in body composition and bone mass may be observed during treatment.Methods: Thirty-eight adolescents (32/38 assigned females at birth) with GD had dual energy...

hrp0094p1-11 | Bone A | ESPE2021

Bone and endocrine monitoring in boys with Duchenne Muscular Dystrophy

Harley Gemma , Dunner Jennifer , Joseph Shuko , Horrocks Iain , Choong Wong Sze ,

Background: In 2020, a UK wide collaborative project between clinicians and patient organisations (Funding from Duchenne UK) called DMD Care UK was launched. The project aims to define the care standards which should be implemented as a priority in all UK centres. Adapting recommendations from the 2018 international care consensus, items were highlighted and used as standards for this audit in one tertiary centre. The standards are that all boys with DMD<p...

hrp0097p1-449 | Fat, Metabolism and Obesity | ESPE2023

Body composition following initiation of daily glucocorticoid in Duchenne Muscular Dystrophy: Identifying the timing of increase of fat mass to introduce intensive weight management strategies

McQuillan Erin , Dunne Jennifer , Mochrie Rachel , Horrocks Iain , Joseph Shuko , Choong Wong Sze

Background: Glucocorticoid (GC) therapy is standard of care of management of Duchenne Muscular Dystrophy (DMD) but its use is associated with a range of side-effects. Weight gain leading to significant obesity is common in GC treated boys. There are limited studies evaluating body composition in DMD following initiation of GC, and the timing of increase in fat mass is not known.Aim(s): To evaluate changes in growth param...

hrp0097p1-573 | Sex Differentiation, Gonads and Gynaecology, and Sex Endocrinology | ESPE2023

Testosterone therapy in Duchenne Muscular Dystrophy and longitudinal bone growth with metacarpophalangeal index

McCauley Cara , Dunne Jennifer , Horrocks Iain , Joseph Shuko , Choong Wong Sze

Background: Testosterone therapy is recommended for management of puberty from the age of 12 years in boys with Duchenne Muscular Dystrophy(DMD) in accordance with the 2018 international standards of care. The majority of boys loose ambulation in mid to late adolescence. Height measurement is problematic in these adolescents s as lower limb contracture can be common and estimated height from segmental body part measurements generally over-estimate height.<...