The effect of steroid treatment on weight in nonambulatory males with Duchenne muscular dystrophy
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.
Lamb, M. M., Cai, B., Royer, J., Pandya, S., Soim, A., Valdez, R., ... Muscular Dystrophy Surveillance, Research, and Tracking Network (MD STARnet) (2018). The effect of steroid treatment on weight in nonambulatory males with Duchenne muscular dystrophy. American Journal of Medical Genetics. Part A, 176(11), 2350-2358. https://doi.org/10.1002/ajmg.a.40517