STEROIDS
page 1
January, 2005
(IN PRESS: Arch.Neurol.2005;62:128-32) Intermittent Prednisone Therapy in Duchenne Muscular Dystrophy - A Randomized Controlled Trial NEW
Ernesto A. C. Beenakker; Johanna M. Fock; Marja J. Van Tol; Natalia M. Maurits; Hendrik M. Koop; Oebele F. Brouwer; Johannes H. Van der Hoeven - Netherland
Background Prednisone treatment is used to prolong ambulation in patients with Duchenne muscular dystrophy (DMD). However, since severe adverse effects often accompany prednisone treatment, it is debatable whether the benefits of prednisone treatment outweigh its adverse effects.
Objectives To study the effects of prednisone on muscle function and to determine the extent of steroid-related adverse effects and their influence on the quality of life of ambulant patients with DMD.
Design A randomized, placebo-controlled, crossover trial with 6 months of treatment: prednisone or placebo (0.75 mg/kg daily) during the first 10 days of each month. After a washout period of 2 months, patients received the other regimen for an additional 6 months.
Setting University hospital and rehabilitation center in the Netherlands.
Patients Seventeen ambulant patients with DMD aged 5 to 8 years.
Main Outcome Measure Change in muscle function assessed by timed functional testing: running 9 m, climbing 4 standard-sized stairs, and rising from the floor to a standing position.
Results The increase in time needed to run 9 m (P = .005) and to climb 4 standard-sized stairs (P = .02) was significantly lower during the prednisone period.
Conclusions Prednisone slowed deterioration of muscle function and muscle force in ambulant patients with DMD. Although adverse effects were present, patient quality of life was not affected. Therefore, short-term prednisone treatment can be recommended to preserve motor functions in ambulant patients with DMD.
January, 2005
CORTICOSTEROIDS FOR DUCHENNE MUSCULAR DYSTROPHY
April, 2004
European Neuro Muscular Centre (ENMC)
Steroids and Duchenne Muscular Dystrophy (DMD) - some questions and answers.
April, 2004
Deflazacort treatment in Duchenne Muscular Dystrophy, by Doug Biggar
http://www.parentprojectmd.org/news/latest/deflazacort_biggar.pdf
March, 2004:
Written by Ana Lùcia Langer
What muscular dystrophy patients and their parents should know about steroid therapy
March, 2004:
Steroid Treatment and the Development of Scoliosis in Males with Duchenne Muscular Dystrophy
Conclusions: Steroid treatment slows the progression of scoliosis in males with Duchenne muscular dystrophy; however, longer-term evaluation will be necessary to determine if the treatment prevents the development of scoliosis or just delays its onset. At the very least, steroid treatment delays the need for spinal surgery.
http://www.ejbjs.org/cgi/content/abstract/86/3/519
Report on the Round table on steroids in Duchenne Muscular Dystrophy, held in Monte Carlo on june 6, 2003
http://www.parentprojectuk.com/SteroidReport.pdf
Deflazacort prevents heart disease in Duchenne's, study finds http://www.bloorviewmacmillan.on.ca/aboutus/stories.htm#heart
25/03/03: IMPORTANT: Effects of Deflazacort on Left Ventricular Function in Patients With Duchenne Muscular Dystrophy
Cardiac involvement in Duchenne muscular dystrophy (DMD) is well described. Although the effect of deflazacort, an oxazolone derivative of prednisone, on skeletal and pulmonary muscle function has been described, there is no information on the effects of deflazacort on myocardial function. The purpose of this report is to examine the effect of deflazacort on cardiac function in patients with DMD. This is a retrospective cohort study of patients seen at the Bloorview MacMillan Children’s Center, Toronto, Canada. Patients with DMD between the ages of 10 and 18 years were included in the study. Administration with deflazacort was begun at a mean age of 8.4 + 2 years. The initial dose of deflazacort was 0.9 mg/kg/day along with daily oral supplements of vitamin D and calcium. As per standard protocol, the dose of deflazacort decreased with the duration of treatment (at 10 years, the mean dose of deflazacort was 0.76 + 0.19 mg/kg/day; at 15 years 0.61+ 0.20 mg/kg/day; and at 18 years 0.59 + 0.15mg/kg/day). All patients took deflazacort for at least 3years.This is the first published study examining the effects of deflazacort treatment on cardiac function in patients with DMD. We found that patients who had been receiving deflazacort for > 3 years were more likely to have preserved cardiac function than those who had not received the medication. Preservation of cardiac muscle function was associated with better pulmonary and skeletal muscle function. Cardiac dysfunction did not correlate with patient age, blood pressure, or weight. Significant adverse effects from the medication were uncommon apart from growth limitation and asymptomatic cataracts. Long-term studies are important to determine the duration of benefit on gross motor, pulmonary, and cardiac function.
