Corticosteroid Treatment and Growth Patterns in Ambulatory Males with Duchenne Muscular Dystrophy - 25/05/16

Doi : 10.1016/j.jpeds.2016.02.067

Molly M. Lamb, PhD ^1,^⁎ , Nancy A. West, PhD ^1,², Lijing Ouyang, PhD ³, Michele Yang, MD ⁴, David Weitzenkamp, PhD ⁴, Katherine James, PhD ¹, Emma Ciafaloni, MD ⁵, Shree Pandya, DPT ⁵, Carolyn DiGuiseppi, MD, PhD ¹
on behalf of the
Muscular Dystrophy Surveillance, Research, and Tracking Network (MD STARnet)^{^∗}
List of additional members of MD STARnet is available at www.jpeds.com (Appendix).
Chris Cunniff, MD : Epi PI, John Meaney, MD : Epi PI, Jennifer Andrews, MBA : Project Manager, Kathleen Pettit : Data Manager, Sydney Pettygrove, PhD : Interview Coordinator, Lisa Miller, MD : PI, Dennis Matthews, MD : Clinical PI, April Montgomery, MHA : Program Coordinator, Jennifer Donnelly : Data Manager, Julie Bolen, PhD : Epi PI, Natalie Street, MS : Program Coordinator, Bobby Lyles : Data Manager, Sylvia Mann, MS : Epi PI, Paul Romitti, PhD : Epi PI, Katherine Mathews, MD : Clinical PI, Kristin Caspers Conway, PhD : Co-Investigator, Soman Puzhankara, MS : Data Coordinator, Florence Foo, MA : Data Manager, Shree Pandya, PT, MS : Clinical PI, Christina Westfield, RN : Abstractor, Data QA/QC Coordinator, Charlotte Druschel, MD, MPH : Epi PI, Kim Campbell, PhD : Data Manager, Deborah Fox, MS : Study Coordinator

¹ Colorado School of Public Health, Aurora, CO
² University of Mississippi Medical Center, Jackson, MS
³ Centers for Disease Control and Prevention, Atlanta, GA
⁴ Children's Hospital Colorado, Aurora, CO
⁵ University of Rochester, Rochester, NY

^∗Reprint requests: Molly M. Lamb, PhD, Colorado School of Public Health, University of Colorado Denver, Building 500, 13001 E. 17th Place, Mail Stop C245, Aurora, CO 80045.Colorado School of Public HealthUniversity of Colorado DenverBuilding 50013001 E. 17th PlaceMail Stop C245AuroraCO80045

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.

Le texte complet de cet article est disponible en PDF.

Keywords : weight, height, BMI

Abbreviations : BMI, CDC, DMD, MD STARnet

Plan

Growth Curve Analysis

Linear Mixed-Effects Modeling

Discussion

Supported by the Centers for Disease Control and Prevention (DD000187, DD000189, DD000190, and DD000191). Its contents are solely the responsibility of the authors and do not necessarily represent the official views of the Centers for Disease Control and Prevention or the Department of Health and Human Services. The authors declare no conflicts of interest.

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Vol 173

P. 207 - juin 2016 Retour au numéro

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Corticosteroid Treatment and Growth Patterns in Ambulatory Males with Duchenne Muscular Dystrophy - 25/05/16

Abstract

Objectives

Study design

Results

Conclusion

Plan

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