T.P.50 Outcomes associated with an Interdisciplinary Comprehensive Care program for DMD patients treated with long term glucocorticoids

Abstract

Abstract Glucocorticoid (GC) treatment is the recommended standard of care for patients with Duchenne muscular dystrophy (DMD). The benefit/risk ratio of GC treatment is influenced by adverse effects – cushingoid facies, weight gain, linear growth failure, osteoporosis, etc.; and can be maximized with mitigation of adverse effects. The multi-systemic problems of GC treated patients require a coordinated care approach to improve/stabilize/slow progression of motor function with GC, attain optimal nutritional status for age (low glycemic index diet and treatment of insulin resistance) and prevent contractures (daily home stretch program and night resting ankle braces). Objective: To evaluate the outcomes of integrative care for a cohort of GC treated DMD patients. Study design: Retrospective case series review. Patient characteristics: N = 130; mean age at last visit 9.5 y (±1.9). GC: Daily GC (prednisone – 11; deflazacort – 110; Prednisone change to deflazacort – 9); mean start age of GC 5.0 y (2.6–8.0); daily start dose – 0.75 mg/kg prednisone or 0.9 mg/kg deflazacort. Clinical motor outcomes: (1) Independent ambulators: 7–9 y – 96% ( n = 69); 10–12 y −71% ( n = 44); (2) Ability to climb steps: 7–9 y – 89%; 10–12 y – 62%; (3) Age for point of slope change for NSAA – 11 y ( p = 0.02); timed Gowers 11 y ( p = 0.01), timed 30 ft walk/run – no slope change found up to age 13; (4) Ankle Contractures: 7–9 y – 20.6%; 10–12 y – 43.2%. Growth outcomes at last visit: Height %: 7–9 y 9.4 ± 14.5; 10–12 y 9.0 ± 17.4; weight%: 7–9 yr 47.7 ± 33.2; 10–12 y 52.4 ± 31.3; BMI% 7–9 y 81.3 ± 20.2; 10–12 y 82.4 ± 24.3. Bone health process outcomes: (1) Vitamin D sufficiency: 90% of patients ( n 120); mean 25 OH D – 36.9 ± 13.7 ng/ml; (2) % of patients with normal lumbar spine BMD z scores: 7–9 y 60%; 10–12 y 73%. Our review shows that an integrative care program for DMD patients on long term daily GC is effective in improving motor outcomes with an acceptable side effect profile.