The effect of steroid treatment on weight in nonambulatory males with Duchenne muscular dystrophy

Abstract

To describe the long‐term effect of steroid treatment on weight in nonambulatory males with Duchenne Muscular Dystrophy (DMD), we identified 392 males age 7–29 years with 4,512 weights collected after ambulation loss (176 steroid‐naïve and 216 treated with steroids ≥6 months) from the Muscular Dystrophy Surveillance, Tracking, and Research Network (MD STARnet). Comparisons were made between the weight growth curves for steroid‐naïve males with DMD, steroid‐treated males with DMD, and the US pediatric male population. Using linear mixed‐effects models adjusted for race/ethnicity and birth year, we evaluated the association between weight‐for‐age and steroid treatment characteristics (age at initiation, dosing interval, cumulative duration, cumulative dose, type). The weight growth curves for steroid‐naïve and steroid‐treated nonambulatory males with DMD were wider than the US pediatric male growth curves. Mean weight‐for‐age z scores were lower in both steroid‐naïve (mean = −1.3) and steroid‐treated (mean = −0.02) nonambulatory males with DMD, compared to the US pediatric male population. Longer treatment duration and greater cumulative dose were significantly associated with lower mean weight‐for‐age z scores. Providers should consider the effect of steroid treatment on weight when making postambulation treatment decisions for males with DMD.

The Relationship between Obesity and Clinical Outcomes in Young People with Duchenne Muscular Dystrophy

“…The response to steroids may also play a role in protecting against a slower walk/run but also causing a higher BMI and short stature which may have functional benefits [22,23]. It is well documented in our cohort and in existing literature that steroid-treated boys with DMD have short stature from early in childhood and that finding is exacerbated with increasing age [10,11,42] As obesity becomes more severe, weight may have a greater effect on functional mobility, but we were unable to stratify the obesity category further according to obesity severity due to small sample sizes.…”

“…Long-term corticosteroid 2 of 15 treatment is the mainstay therapy for DMD, with benefits including prolongation of ability to independently ambulate from 10 years in steroid-naïve boys to 13 years in steroid-treated boys [8,9]. Short stature and susceptibility to both over-and underweight are well documented in DMD [10,11]. Body mass index (BMI) is typically higher in both steroid-treated and steroidnaïve boys with DMD compared to typically developing children [12,13].…”

When age matters: boys with Duchenne muscular dystrophy have growth delay and a fat mass accumulation, as they get older

“…In our study, the W/A z-core was higher in G1 than G2 (p = 0.025), demonstrating that the weight changes resulting from the disease may not appear in early childhood. Lamb et al observed that longer duration and greater cumulative dose of glucocorticoids are significantly associated with lower W/A z-scores (Lamb et al, 2018).…”

Direct costs of adhering to selected Duchenne muscular dystrophy Care Considerations: Estimates from a midwestern state

“…23,25 In addition, glucocorticoids (0.75 mg mg/kg per day of prednisone or 0.90 mg/kg/day of deflazacort) and prophylactic cardiac medication (lisinopril: 2.5 mg/day for those up to 40 kg, 5 mg/day for those ≥40 kg) 26 were assumed to be initiated at 8 years of age. We used published average weight data for patients with DMD treated with glucocorticoids 27 to estimate medication costs in combination with the recommended dose per body weight and unit price per dose.…”