#AANAM – Corticosteroids Aid Muscle Mass in BMD and LGMD, Trials Needed

Editor’s note: The Muscular Dystrophy News Today team is providing in-depth coverage of the 2021 Virtual AAN Annual Meeting, April 17–22. Go here to read the latest stories from the conference.

Once-weekly corticosteroid treatment was safe and well-tolerated in people with Becker muscular dystrophy (BMD) or limb-girdle muscular dystrophy (LGMD) in a small clinical trial, and early evidence suggests that corticosteroids can improve muscle mass and muscle function in these patients.

These findings warrant further trials to assess corticosteroid’s potential benefits in treating BMD and LGMD, according to its researchers.

Data were presented in the poster, “24-week open label clinical trial to test safety and efficacy of oral weekly prednisone in adults with Becker (BMD) and Limb-Girdle Muscular Dystrophy (LGMD),” at the 2021 American Academy of Neurology annual meeting.

Corticosteroids (sometimes referred to as “steroids”) are a class of medication that decrease inflammation and closely resemble cortisol, a hormone produced by the adrenal glands. Corticosteroid treatment is common for people with Duchenne muscular dystrophy, and it has been shown to improve walking ability and survival.

Researchers at Northwestern University conducted a small, 24-week Phase 2 clinical trial (NCT04054375) to examine the safety and tolerability of once-weekly corticosteroid use in people with BMD or LGMD.

A total of 20 patients completed the trial: one with BMD and 19 with LGMD. Participants ranged from 18 to 63 years old; about two-thirds were male. Six were able to walk.

Patients were given a corticosteroid called prednisone at doses of 0.75 mg/kg or 1 mg/kg depending on body weight, taken after the last meal every Monday for about six months.

Before and after treatment, they underwent a battery of laboratory and imaging tests. For most of these assessments, values did not significantly differ between the trial’s start and end. The researchers highlighted that these findings indicate that treatment did not cause any noteworthy problems in areas such as weight, metabolism, blood pressure, and breathing ability.

Corticosteroid use, however, led to an increase in lean muscle mass, as well as a decrease in levels of creatine kinase, which is a marker of muscle damage.

Among the six patients able to walk, a trend toward a better walking ability was seen.

Corticosteroid treatment was generally safe and well-tolerated. The most common events were increased anxiety on Monday nights and facial acne. No serious adverse events were reported, and no one discontinued treatment due to side effects. All but one of these 20 patients elected to continue taking weekly corticosteroids after finishing the study.

“Additional multicenter randomized clinical trials are needed to better understand how weekly steroids may benefit patients with BMD and LGMD,” the researchers concluded.