P.2.19 Long term growth hormone therapy in Duchenne Muscular Dystrophy (DMD): A case report

Abstract

Long term glucocorticoid (GC) treatment compromises linear growth in DMD. Growth hormone (GH) therapy improves height, yet the impact on motor and cardiopulmonary function has not been studied in current DMD research. To report clinical outcomes in a DMD patient on long term GH for GC-induced linear growth failure. Retrospective review of motor, cardiac, and pulmonary outcomes pre- and post-GH therapy. Subject was a 19.6-year-old male with DMD, GC-induced linear growth failure, and GH deficiency (peak stimulated GH of 3.3ng/mL, normal >10), treated with daily GC for 9.9y and GH for 6.3y. GH treatment was associated with improved height growth (pre-GH height growth 1.25cm per year compared to 4.3cm per year on GH, reaching 151cm at age 18.8y). Motor function improved initially and stabilized until age 14.7y, followed by a gradual decline; subject lost the ability to rise from the floor independently at age 16.2y, to climb stairs unsupported at age 17.5y, and to ambulate independently at age 18.8 y. Cardiac function remained stable after GC initiation (pre-GH, LVEF=54.0%; post-GH, LVEF=64.8%). Pulmonary function was also relatively unaffected by GH therapy (pre-GH, FVC=1.42L (72% of predicted), MIP=64cm H20, MEP=64cm H20, PCF=200L/min; post-GH, FVC=2.3 L (82% of predicted), MIP=76cm H20, MEP=76cm H20, PCF=300L/min). Subject had no significant side effects of long term GH treatment. This single case report of a DMD patient with GC-induced growth failure improved with long term GH treatment suggests that GH did not adversely affect motor and cardiopulmonary function. Additional research on functional outcomes in GH-treated DMD patients is indicated.