Brenda L Wong1, Irina Rybalsky2, Karen C Shellenbarger2, Cuixia Tian2, Mary A McMahon3, Meilan M Rutter4, Hemant Sawnani5, John L Jefferies6. 1. Comprehensive Neuromuscular Center, Cincinnati Children's Hospital Medical Center, Cincinnati, OH. Electronic address: brenda.wong@cchmc.org. 2. Comprehensive Neuromuscular Center, Cincinnati Children's Hospital Medical Center, Cincinnati, OH. 3. 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. 4. Comprehensive Neuromuscular Center, Cincinnati Children's Hospital Medical Center, Cincinnati, OH; Division of Endocrinology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH. 5. Comprehensive Neuromuscular Center, Cincinnati Children's Hospital Medical Center, Cincinnati, OH; Division of Pulmonary Medicine, Cincinnati Children's Hospital Medical Center, Cincinnati, OH. 6. Comprehensive Neuromuscular Center, Cincinnati Children's Hospital Medical Center, Cincinnati, OH; The Heart Institute, Cincinnati Children's Hospital Medical Center, Cincinnati, OH.
Abstract
OBJECTIVE: 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. STUDY DESIGN: 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. RESULTS: 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. CONCLUSIONS: 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.
OBJECTIVE: 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. STUDY DESIGN: 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. RESULTS: 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. CONCLUSIONS: 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: Savant S Thakur; Kristy Swiderski; James G Ryall; Gordon S Lynch Journal: Philos Trans R Soc Lond B Biol Sci Date: 2018-01-19 Impact factor: 6.237
Authors: Ali Asma; Armagan Can Ulusaloglu; Michael Wade Shrader; William G Mackenzie; Robert Heinle; Mena Scavina; Jason J Howard Journal: Spine Deform Date: 2022-06-13
Authors: Erika Finanger; Krista Vandenborne; Richard S Finkel; H Lee Sweeney; Gihan Tennekoon; Sabrina Yum; Maria Mancini; Pradeep Bista; Andrew Nichols; Hanlan Liu; Angelika Fretzen; Joanne M Donovan Journal: J Neuromuscul Dis Date: 2019