PURPOSE: In tins retrospective study, positive results and side effects of long-term steroid treatment for Duchenne muscular dystrophy were analyzed. Results of an average follow-up period of 65 months (range, 49-79 mo) are described. METHODS: Nineteen male patients receiving steroids (0.9 mg/kg deflazacort) were compared with the natural history of Duchenne muscular dystrophy given in the literature. Additionally, 13 of these 19 patients were compared with an age-matched control group of 13 patients with Duchenne muscular dystrophy who were not receiving steroid treatment. They were not treated because of loss of independent walking before their first examination in our department or after informed consent with the parents. The same investigators followed all patients. RESULTS: Muscle strength, functional grade according to Vignos scale, and timed functional tests were significantly better in patients on steroids. The vital capacity was significantly improved in treated versus untreated boys; cardiologic examinations revealed no obvious difference. The main side effects of steroids in patients with Duchenne muscular dystrophy were obesity (2 of 13), cataracts (6 of 13), and short stature (II of 13). Rates of infectious diseases or of bone fractures resulting from osteoporosis were not increased. CONCLUSION: Long-term steroid treatment in Duchenne muscular dystrophy (average. 65 mo) proved beneficial m terms of muscle strength, function, and tolerable side effects, as compared with untreated patients with Duchenne muscular dystrophy.
PURPOSE: In tins retrospective study, positive results and side effects of long-term steroid treatment for Duchenne muscular dystrophy were analyzed. Results of an average follow-up period of 65 months (range, 49-79 mo) are described. METHODS: Nineteen male patients receiving steroids (0.9 mg/kg deflazacort) were compared with the natural history of Duchenne muscular dystrophy given in the literature. Additionally, 13 of these 19 patients were compared with an age-matched control group of 13 patients with Duchenne muscular dystrophy who were not receiving steroid treatment. They were not treated because of loss of independent walking before their first examination in our department or after informed consent with the parents. The same investigators followed all patients. RESULTS: Muscle strength, functional grade according to Vignos scale, and timed functional tests were significantly better in patients on steroids. The vital capacity was significantly improved in treated versus untreated boys; cardiologic examinations revealed no obvious difference. The main side effects of steroids in patients with Duchenne muscular dystrophy were obesity (2 of 13), cataracts (6 of 13), and short stature (II of 13). Rates of infectious diseases or of bone fractures resulting from osteoporosis were not increased. CONCLUSION: Long-term steroid treatment in Duchenne muscular dystrophy (average. 65 mo) proved beneficial m terms of muscle strength, function, and tolerable side effects, as compared with untreated patients with Duchenne muscular dystrophy.
Authors: Apurva Sarathy; Ryan D Wuebbles; Tatiana M Fontelonga; Ashley R Tarchione; Lesley A Mathews Griner; Dante J Heredia; Andreia M Nunes; Suzann Duan; Paul D Brewer; Tyler Van Ry; Grant W Hennig; Thomas W Gould; Andrés E Dulcey; Amy Wang; Xin Xu; Catherine Z Chen; Xin Hu; Wei Zheng; Noel Southall; Marc Ferrer; Juan Marugan; Dean J Burkin Journal: Mol Ther Date: 2017-04-05 Impact factor: 11.454
Authors: Andreas R Baudy; Erica K M Reeves; Jesse M Damsker; Christopher Heier; Lindsay M Garvin; Blythe C Dillingham; John McCall; Sree Rayavarapu; Zuyi Wang; Jack H Vandermeulen; Arpana Sali; Vanessa Jahnke; Stephanie Duguez; Debra DuBois; Mary C Rose; Kanneboyina Nagaraju; Eric P Hoffman Journal: J Pharmacol Exp Ther Date: 2012-06-28 Impact factor: 4.030
Authors: Eric P Hoffman; Erica Reeves; Jesse Damsker; Kanneboyina Nagaraju; John M McCall; Edward M Connor; Kate Bushby Journal: Phys Med Rehabil Clin N Am Date: 2012-11 Impact factor: 1.784