Endocrine Abstracts

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.

Aim(s): To evaluate changes in growth parameters: height-SDS, weight-SDS, body mass index (BMI)-SDS following initiation of GC in DMD.

Methods: Between 2013-2019, 26 boys with DMD were commenced on daily GC. Growth parameters at baseline, 1-year and 2-years were compared. Data were expressed as mean (SD). P<0.05 was accepted as statistical significance.

Results: Of the 26 boys, 1 was excluded due to insufficient growth data at follow-up. All 25 boys were initiated on daily GC (15 Deflazacort, 10 Prednisolone), and remained on the same GC regimen/type during the 2-year follow-up. Mean age at initiation of GC was 5.5 years. All boys remained ambulant all throughout the 2-year follow-up. Mean height-SDS prior to initiation of GC at baseline was -1.15 (1.12). Mean height-SDS at 1-year was -1.57 (1.10) [P<0.001 vs baseline] and was -1.87 (1.01) at 2-year [P<0.001 vs baseline and 1-year]. Mean BMI-SDS at baseline was +0.67 (0.97). Mean BMI-SDS at 1-year was +1.08 (1.1) [P=0.009 vs baseline] and was +1.46 (0.96) at 2-year [P<0.001 vs baseline; P=0.002 vs 1-year]. At baseline, BMI-SDS in the overweight, obese or severely obese category was noted in 5/25 (20%) whereas this was noted in 9/25 (36%) boys at 1-year and 13/25 (52%) at 2-years of GC treatment. The proportion of boys with severe obesity at baseline was 1/25 (4%) and 3/25 (12%) at 1-year and 2-years, respectively. Investigations for metabolic complications were not performed in overweight, obese or severely obese boys.

Conclusion: Significant increase in BMI occurs early following initiation of daily GC in young boys with DMD. Routine structured nutritional input should be part of clinical care at the time of initiation of GC. Current management strategies of childhood obesity and its complications are not suitable for boys with DMD (e.g. exercise and use of statins contraindicated). National clinical pathways of evaluation and management of obesity-metabolic complications in DMD should now be developed.