Duchenne MD Boys on chronic Glucocorticoid Therapy

27 – (ENDO 2011, Boston, 6-9 June) Puberty Is Delayed or Absent in Duchenne Muscular Dystrophy Boys on chronic Glucocorticoid Therapy

MM Rutter, SR Rose, J Collins, H Sucherew, B Wong. Cincinnati Children’s Hospital medical center and university of Cincinnati, Cincinnati, OH;

Background: Duchenne Muscular Dystrophy (DMD) is a progressive neuromuscular dysfunction affecting one in 3500 boys. chronic glucocorticoid (GC) cure is deemed normal therapy, and slows disease progression. However, GCs induce osteoporosis, growth failure, obesity, insulin resistance and pubertal delay, compromising quality of life. Absent or delayed puberty is definitely an important problem which negatively impacts bone health, growth, self esteem and muscle strength. The prevalence in DMD is not known, and it is actually often under-recognized and untreated.
Objective: To ascertain the prevalence of delayed or absent puberty in DMD on GC therapy.
Methods: clients aged 13 to 19 years with confirmed DMD on chronic daily GC treated with the Cincinnati Neuromuscular extensive treatment center have been studied. clinical information was obtained from an IRB-approved database and chart review. Pubertal status was assessed annually by measuring earlier morning hours total testosterone concentrations and/ or pubertal examination by an endocrinologist.
Results: Of 64 boys aged ≥ 13 years, 56 (88%) had no proof of puberty. Of 49 boys aged ≥ 14 years, 41(84%) showed no symptoms of puberty. Overall, 3 boys had pubertal arrest with pretty low testosterone ranges for get older or testicular size. Only five boys have been in puberty; of these, 2 had 4 mL testes at get older 14 years and 2 showed no major development over 2 years. Of 22 boys aged ≥ 16 years, 21 (95%) still had absent or arrested puberty. 47/59 (80%) boys with absent/ arrested puberty had severe osteoporosis. Absent puberty was distressing to 25 boys who as a outcome have been treated with testosterone, 14 using topical gel.
Conclusions: Most DMD boys on chronic daily GC will not produce spontaneous puberty, or fail to progress. The problem doesn’t tend to improve with age. principal companies might need an increased awareness to facilitate timely suggestion to an endocrinologist. We recommend clinical assessment and discussion by get older twelve and suggestion by get older 14 years. management should be proactive and interdisciplinary, with awareness to associated co-morbidities for instance osteoporosis.

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