source: American Journal of Medical Genetics
Lamb MM, Cai B, Royer J, Pandya S, Soim A, Valdez R, DiGuiseppi C, James K, Whitehead N, Peay H, Venkatesh SY, Matthews D
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.
Colorado School of Public Health, USA; Arnold School of Public Health, USA; South Carolina Revenue and Fiscal Affairs Office, USA; University of Rochester, USA; New York State Department of Health, USA; Centers for Disease Control and Prevention, USA; RTI International, North Carolina; University of South Carolina School of Medicine, USA; Children's Hospital Colorado, USA
10.1002/ajmg.a.40517 read more