TY - JOUR
T1 - Corticosteroid treatment and growth patterns in ambulatory males with duchenne muscular dystrophy
AU - Lamb, Molly M.
AU - West, Nancy A.
AU - Ouyang, Lijing
AU - Yang, Michele
AU - Weitzenkamp, David
AU - James, Katherine
AU - Ciafaloni, Emma
AU - Pandya, Shree
AU - Diguiseppi, Carolyn
AU - Cunniff, Christopher M
AU - Meaney, John
AU - Andrews, Jennifer
AU - Pettit, Kathleen
AU - Pettygrove, Sydney
AU - Miller, Lisa
AU - Matthews, Dennis
AU - Montgomery, April
AU - Donnelly, Jennifer
AU - Bolen, Julie
AU - Street, Natalie
AU - Lyles, Bobby
AU - Mann, Sylvia
AU - Romitti, Paul
AU - Mathews, Katherine
AU - Caspers Conway, Kristin
AU - Puzhankara, Soman
AU - Foo, Florence
AU - Westfield, Christina
AU - Druschel, Charlotte
AU - Campbell, Kim
AU - Fox, Deborah
N1 - Publisher Copyright:
© 2016 Elsevier Inc. All rights reserved.
PY - 2016/6/1
Y1 - 2016/6/1
N2 - 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.
AB - 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.
KW - BMI
KW - height
KW - weight
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U2 - 10.1016/j.jpeds.2016.02.067
DO - 10.1016/j.jpeds.2016.02.067
M3 - Article
C2 - 27039228
AN - SCOPUS:84962094373
SN - 0022-3476
VL - 173
SP - 207-213.e3
JO - Journal of Pediatrics
JF - Journal of Pediatrics
ER -