title: Long-Term Outcome of Interdisciplinary Management of Patients with Duchenne Muscular Dystrophy Receiving Daily Glucocorticoid Treatment.
abstract: To evaluate clinical outcomes and steroid side effects in a cohort of patients with Duchenne muscular dystrophy (DMD) treated with long-term daily glucocorticoid therapy. Although daily glucocorticoid therapy has been shown to extend ambulatory function in DMD, less frequent dosing is often used because of side effect concerns.
  
  
  
  Retrospective study of 97 patients with DMD aged 10 to <16 years treated with daily glucocorticoid (89% on deflazacort) for a mean of 8.5 years. Outcome measures were motor, pulmonary, and cardiac function, and scoliosis. Side effects were growth failure and weight gain, facial fullness, blood pressure, bone health, cataracts, gastrointestinal symptoms, behavior, hypertrichosis, and need for medication interventions.
  
  
  
  For 13- to 16-year-old patients, 40% could rise from the floor and 50% could perform the 30-foot run test. Forced vital capacity for the entire cohort was well preserved. Thirteen percent of younger (10- to <13-year-old) and 21% of older patients had findings of left ventricle systolic dysfunction. Six percent (all aged 16 years) developed scoliosis (Cobb angle >20 degrees). Eighty-six percent had normal weight velocities; 30% had no increased facial fullness; 72% had short stature; and 19% had asymptomatic cataracts. Asymptomatic spine compression deformities were noted in 76% and long bone fractures in 30%. One patient stopped glucocorticoid because of behavioral concerns.
  
  
  
  With evidence for improved outcomes and manageable side effects, we recommend use of daily glucocorticoid therapy for patients with DMD with anticipatory management of side effects and a coordinated interdisciplinary care approach.
authors.affiliation: Comprehensive Neuromuscular Center, Cincinnati Children's Hospital Medical Center, Cincinnati, OH. Electronic address: brenda.wong@cchmc.org.
authors.forename: Brenda L
authors.initials: BL
authors.lastname: Wong
authors.affiliation: Comprehensive Neuromuscular Center, Cincinnati Children's Hospital Medical Center, Cincinnati, OH.
authors.forename: Irina
authors.initials: I
authors.lastname: Rybalsky
authors.affiliation: Comprehensive Neuromuscular Center, Cincinnati Children's Hospital Medical Center, Cincinnati, OH.
authors.forename: Karen C
authors.initials: KC
authors.lastname: Shellenbarger
authors.affiliation: Comprehensive Neuromuscular Center, Cincinnati Children's Hospital Medical Center, Cincinnati, OH.
authors.forename: Cuixia
authors.initials: C
authors.lastname: Tian
authors.affiliation: Comprehensive Neuromuscular Center, Cincinnati Children's Hospital Medical Center, Cincinnati, OH; Division of Physical Medicine and Rehabilitation, Cincinnati Children's Hospital Medical Center, Cincinnati, OH.
authors.forename: Mary A
authors.initials: MA
authors.lastname: McMahon
authors.affiliation: Comprehensive Neuromuscular Center, Cincinnati Children's Hospital Medical Center, Cincinnati, OH; Division of Endocrinology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH.
authors.forename: Meilan M
authors.initials: MM
authors.lastname: Rutter
authors.affiliation: Comprehensive Neuromuscular Center, Cincinnati Children's Hospital Medical Center, Cincinnati, OH; Division of Pulmonary Medicine, Cincinnati Children's Hospital Medical Center, Cincinnati, OH.
authors.forename: Hemant
authors.initials: H
authors.lastname: Sawnani
authors.affiliation: Comprehensive Neuromuscular Center, Cincinnati Children's Hospital Medical Center, Cincinnati, OH; The Heart Institute, Cincinnati Children's Hospital Medical Center, Cincinnati, OH.
authors.forename: John L
authors.initials: JL
authors.lastname: Jefferies
mesh_terms: D000293:Adolescent; D000367:Age Factors; D002648:Child; D015331:Cohort Studies; D004305:Dose-Response Relationship, Drug; D004334:Drug Administration Schedule; D005081:Exercise Therapy / Q000379:methods; D005500:Follow-Up Studies; D050723:Fractures, Bone / Q000139:chemically induced / Q000503:physiopathology; D005938:Glucocorticoids / Q000008:administration & dosage* / Q000009:adverse effects*; D006801:Humans; D007333:Insulin Resistance; D008134:Long-Term Care; D008297:Male; D020388:Muscular Dystrophy, Duchenne / Q000175:diagnosis / Q000188:drug therapy* / Q000534:rehabilitation; D010024:Osteoporosis / Q000139:chemically induced / Q000503:physiopathology; D010348:Patient Care Team / Q000458:organization & administration*; D011241:Prednisone / Q000008:administration & dosage / Q000009:adverse effects; D011282:Pregnenediones / Q000008:administration & dosage / Q000009:adverse effects; D012189:Retrospective Studies; D018570:Risk Assessment; D012720:Severity of Illness Index; D016896:Treatment Outcome; D015430:Weight Gain
chemical_list: D005938:Glucocorticoids; D011282:Pregnenediones; C021988:deflazacort; D011241:Prednisone
keywords: coordinated comprehensive care; deflazacort; multidisciplinary management; prednisone