Related Experiment Video
Updated: Aug 15, 2026
Measurements of Motor Function and Other Clinical Outcome Parameters in Ambulant Children with Duchenne Muscular Dystrophy
Published on: January 12, 2019
Corticosteroid treatment and functional improvement in Duchenne muscular dystrophy: long-term effect
Birol Balaban1, Dennis J Matthews, Gerald H Clayton
1Department of Physical Medicine and Rehabilitation, Gulhane Military Medical Academy, Etlik-Ankara, Turkey.
Insights
Prednisone and deflazacort significantly improve function and slow disease progression in boys with Duchenne muscular dystrophy. These steroids offer similar benefits for ambulation, pulmonary function, and scoliosis, but carry risks of side effects.
Area of Science:
- Pediatric Neurology
- Pharmacology
- Genetics
Background:
- Duchenne muscular dystrophy (DMD) is a progressive genetic disorder.
- Current treatments aim to manage symptoms and slow disease progression.
- Corticosteroids like prednisone and deflazacort are commonly used, but their long-term comparative effects require further investigation.
Purpose of the Study:
- To compare the long-term effects of prednisone and deflazacort on functional status in children with DMD.
- To evaluate the impact of these corticosteroids on motor function, pulmonary capacity, and need for scoliosis surgery.
Main Methods:
- Retrospective review of 49 boys with DMD (aged 12-15 years) over 7 years.
- Groups included prednisone treatment (n=18), deflazacort treatment (n=12), and no drug treatment (n=19).
- Comparison of limb motor function, pulmonary function, scoliosis surgery rates, and side effects.
Main Results:
- Steroid-treated groups showed significantly better functional status and test performance compared to the untreated group (P < 0.05).
- No significant difference in efficacy was observed between prednisone and deflazacort (P > 0.05).
- Treated boys had significantly less scoliosis surgery and better pulmonary function; side effects included cataracts, hypertension, and weight gain.
Conclusions:
- Prednisone and deflazacort significantly slow DMD progression, prolonging ambulation and upper limb function.
- Both corticosteroids demonstrate similar potency in improving pulmonary function and delaying spinal interventions.
- Therapeutic profiles are comparable, but potential serious side effects must be monitored.
Objective:
To determine and compare the long-term effects of prednisone and deflazacort on the functional status of children with Duchenne muscular dystrophy.
Design:
A total of 49 boys with Duchenne muscular dystrophy, between the age of 12 and 15 yrs, who were observed over a 7-yr period were reviewed retrospectively. Eighteen had been treated with prednisone, 12 with deflazacort, and 19 had no drug treatment. All boys treated with steroids received medication for >2 yrs before losing their ambulation. Lower and upper limb motor functions, pulmonary function, prevalence of surgery for scoliosis, and side effects were compared.
Results:
Boys in the steroid groups were significantly more functional and performed better on all tests than boys not treated (P < 0.05). There was no significant difference between the deflazacort- and prednisone-treated groups (P > 0.05). The number of boys having scoliosis surgery in treated groups was significantly less than nontreated boys (P < 0.05). The control group's pulmonary capacity was decreasing and significantly less than both prednisone- and deflazacort-treated boys. Both deflazacort and prednisone had beneficial effect on pulmonary function and scoliosis. Cataracts, hypertension, behavioral changes, excessive weight gain, and vertebral fracture were noted as serious side effects.
Conclusions:
Prednisone and deflazacort have a significant beneficial effect on slowing the disease progress. Their usage in Duchenne muscular dystrophy may prolong ambulation and upper limb function with similar potency. Both steroids also improve pulmonary function, in addition to delaying the need for spinal interventions, with similar therapeutic profiles.
Related Concept Videos
01:21Drugs for Treatment of Crohn's Disease in IBD Using Glucocorticoids
01:29Drugs for Treatment of Crohn's Disease in IBD Using Immunomodulatory Agents
01:21Satellite Stem Cells and Muscular Dystrophy
01:20Myasthenia Gravis: Overview and Treatment
These antibodies interfere with the function of the nicotinic receptors in three ways: by binding to the receptor and disrupting acetylcholine binding; by causing cross-linking of receptors which leads...