Date of Graduation

Summer 8-11-2012

Degree Type

Capstone Project

Degree Name

Master of Science in Physician Assistant Studies

First Advisor

Robert P. Rosenow, Pharm.D., O.D.

Second Advisor

Annjanette Sommers PA-C, MS

Abstract

Background: Duchenne muscular dystrophy (DMD) is a genetic disorder in males resulting in muscle weakness leading to loss of ambulation and progression to death by pulmonary and cardiac dysfunction. It has been documented both steroids deflazacort and prednisone delay muscle degeneration but not many studies outline the differences between the two including side effect profiles. Deflazacort is not yet available in the US. This systematic review compiles recent data to compare deflazacort to prednisone in the treatment of DMD boys.

Method: A full search of medical literature using multiple databases, MEDLINE, CINAHL, Evidence Based Reviews Multifile and Web of Science, and reference lists was conducted with the key words muscular dystrophy, deflazacort and prednisone. Articles that consisted of the outcomes of muscle function, cardiac and pulmonary changes, side effect incidence and type, and gene expression in whole blood were included. The literature was assessed using the GRADE system to rate its quality and importance.

Results: Four articles were selected for review. There were no significant differences between the two steroids in treating muscle functionality, prevention of cardiomyopathy or difference in pulmonary function. In Bonifati et al and Balaban et al, weight gain in prednisone-treated patients was greater than with deflazacort. With regards to gene expression, Lit et al discovered gene probes in the prednisone-treated patients which promote weight gain. On the other hand, deflazacort probes are suspected to prevent obesity.

Conclusion: It is clear both steroids are efficacious in treating DMD but side effects will determine patient and providers’ choice of prescription. Further research with randomized control trails over extended time periods is needed to establish the true difference between side effects of deflazacort and prednisone. Research in gene expression holds promise for future utility in the selection of steroid therapy.

Share

COinS