Corticosteroid treatment and growth patterns in ambulatory males with duchenne muscular dystrophy

Molly M. Lamb, Nancy A. West, Lijing Ouyang, Michele Yang, David Weitzenkamp, Katherine James, Emma Ciafaloni, Shree Pandya, Carolyn Diguiseppi, Chris Cunniff, John Meaney, Jennifer Andrews, Kathleen Pettit, Sydney Pettygrove, Lisa Miller, Dennis Matthews, April Montgomery, Jennifer Donnelly, Julie Bolen, Natalie StreetBobby Lyles, Sylvia Mann, Paul Romitti, Katherine Mathews, Kristin Caspers Conway, Soman Puzhankara, Florence Foo, Christina Westfield, Charlotte Druschel, Kim Campbell, Deborah Fox

Research output: Contribution to journalArticlepeer-review

24 Scopus citations

Abstract

Objectives To evaluate growth patterns of ambulatory males with Duchenne muscular dystrophy (DMD) treated with corticosteroids compared with ambulatory, steroid-naïve males with DMD and age-matched unaffected general-population males and to test associations between growth and steroid treatment patterns among treated males. Study design Using data from the Muscular Dystrophy Surveillance, Tracking, and Research Network, we identified a total of 1768 height, 2246 weight, and 1755 body mass index (BMI) measurements between age 2 and 12 years for 324 ambulatory males who were treated with corticosteroids for at least 6 months. Growth curve comparisons and linear mixed-effects modeling, adjusted for race/ethnicity and birth year, were used to evaluate growth and steroid treatment patterns (age at initiation, dosing interval, duration, cumulative dose). Results Growth curves for ambulatory males treated with corticosteroids showed significantly shorter stature, heavier weight, and greater BMI compared with ambulatory, steroid-naïve males with DMD and general-population US males. Adjusted linear mixed-effects models for ambulatory males treated with corticosteroids showed that earlier initiation, daily dosing, longer duration, and greater dosages predicted shorter stature with prednisone. Longer duration and greater dosages predicted shorter stature for deflazacort. Daily prednisone dosing predicted lighter weight, but longer duration, and greater dosages predicted heavier weight. Early initiation, less than daily dosing, longer duration, and greater doses predicted greater BMIs. Deflazacort predicted shorter stature, but lighter weight, compared with prednisone. Conclusion Prolonged steroid use is significantly associated with short stature and heavier weight. Growth alterations associated with steroid treatment should be considered when making treatment decisions for males with DMD.

Original languageEnglish (US)
Pages (from-to)207-213.e3
JournalJournal of Pediatrics
Volume173
DOIs
StatePublished - Jun 1 2016

Keywords

  • BMI
  • height
  • weight

ASJC Scopus subject areas

  • Pediatrics, Perinatology, and Child Health

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