Prednisone

Prednisone is a synthetic corticosteroid currently used off-label to delay muscle weakness progression in Duchenne muscular dystrophy (DMD) patients.

The treatment was approved by the Food and Drug Administration in the 1950s and is prescribed due to its anti-inflammatory properties.

How prednisone works

Prednisone mimics the action of a naturally occurring corticosteroid called cortisol, which is produced by the adrenal glands that sit on top of the kidneys.

The treatment is metabolized into prednisolone by the liver to induce therapeutic effects. Prednisolone can easily pass through the cell membrane and binds to the glucocorticoid receptor inside the cell. Once the glucocorticoid receptor-prednisolone complex reaches the cell nucleus, it regulates gene activity and stifles the production of inflammatory mediators.

The precise mechanisms through which corticosteroids work in the muscle are not fully known, but they have been reported to inhibit muscle breakdown, stimulate muscle repair, stabilize muscle fiber membranes, and have anti-inflammatory and immunosuppressive effects.

Based on this, and according to American Academy of Neurology guidelines, prednisone should be made available to DMD patients to improve muscle strength and lung function, reduce the need for scoliosis surgery, and delay the onset of cardiomyopathy, or heart muscle disease.

Prednisone in clinical trials

A six-month clinical trial in 103 DMD boys, ages 5 to 15, showed that prednisone administered at 0.75 mg per kg of body weight per day or 1.5 mg/kg/day increased muscle strength and function of patients relative to a placebo. Specifically, participants improved on the times needed to rise from a supine to a standing position, to walk a set distance and climb four stairs, and to lift a weight. They also showed better lung function.

Two successive six-month trials found that 0.75 mg/kg/day was the optimal dose for those with DMD. When this dose was given to 93 boys for three years, a significant delay in loss of muscle strength was observed and several boys actually improved motor function performance, although side effects caused a dose reduction in some.

Another double-blind trial (NCT00110669) compared different prednisone dosing regimens in DMD patients. The study assessed daily prednisone 0.75 mg/kg/day (and a placebo on weekends) versus weekend-only prednisone at 5 mg/kg/day plus a daily placebo, over a year. A total of 64 boys between the ages of 4 and 10 were enrolled in the study, which assessed muscle strength in the arms and legs, and mean body mass index (BMI, a measure of body fat), among other measures. Results showed that weekend prednisone dosing is as safe and effective as a daily regimen in maintaining muscle strength and preventing BMI increases.

Other information

Prednisone is an oral treatment available as a tablet (immediate- and delayed-release, sold as Rayos), as a solution (liquid), and as a concentrated solution. The treatment is usually taken with food one to four times a day or once every other day.

The therapy comes in several strengths: 1, 2, 2.5, 5, 10, 20, and 50 mg (tablets); 5 mg/5 mL and 5 mg/mL (oral solutions). Recommended starting dose is usually up to 60 mg daily, depending on specific medical condition and age.

Common side effects include aggression, agitation, blurred vision, dizziness, and a fast and irregular heartbeat. Shortness of breath, weight gain, and trouble thinking may occur. Other possible complications include abdominal pain, cough, dry mouth, diarrhea, pain in the back, arms and legs, trouble sleeping, sweating, vomiting, fluid retention, and increased hunger. Metabolic, cardiovascular, and hormone-related side effects also may occur, as well as skin issues, ocular problems, and allergic reactions.

Prednisone may cause new infections and aggravate underlying infections. The treatment may mask signs of infection.

It also may decrease bone density, which may lead to weak and brittle bones, or osteoporosis. Calcium and vitamin D supplementation is recommended for long-term prednisone treatment.

Children should be cautiously monitored as prednisone may negatively affect growth and development. Use during pregnancy may harm the fetus, particularly in the first trimester.

 

Last updated: Feb. 17, 2022, by Teresa Carvalho MS

 


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