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.

• Authors: Molly M Lamb, Bo Cai, Julie Royer, Shree Pandya, Aida Soim, Rodolfo Valdez, Carolyn DiGuiseppi, Katherine James, Nedra Whitehead, Holly Peay, Swamy Y Venkatesh, Dennis Matthews, Muscular Dystrophy Surveillance, Research, and Tracking Network (MD STARnet)
• Journal: American journal of medical genetics. Part A (Am J Med Genet A) Vol. 176 Issue 11 Pg. 2350-2358 (11 2018) ISSN: 1552-4833 [Electronic] United States
• PMID: 30256515 (Publication Type: Journal Article, Research Support, U.S. Gov't, P.H.S.)
• Copyright: © 2018 Wiley Periodicals, Inc.

Chemical References

• Adrenal Cortex Hormones

Topics

• Adolescent
• Adrenal Cortex Hormones (pharmacology, therapeutic use)
• Adult
• Body Weight (drug effects)
• Child
• Humans
• Male
• Muscular Dystrophy, Duchenne (drug therapy, physiopathology)
• Walking
• Young Adult