P.11.11 Pulmonary function characteristics of boys with Duchenne Muscular Dystrophy by age groups, ambulatory status and steroid use

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• 作者：R.T. Abresch ; C.M. McDonald ; E.K. Henricson ; N. Gustavo ; F. Hu ; T. Duong ; N. Joyce ; J. Mah
• 刊名：Neuromuscular Disorders
• 出版年：2013
• 出版时间：October, 2013
• 年：2013
• 卷：23
• 期：9-10
• 页码：801-802
• 全文大小：56 K

文摘

The lack of natural history data and well-characterized outcome measures limits the ability to conduct therapeutic clinical trials in Duchenne Muscular Dystrophy (DMD). This study describes pulmonary function tests (PFTs) from 341 participants with confirmed DMD from the Cooperative International longitudinal study. The objective of this study is to describe the pulmonary function characteristics of individuals with DMD and to determine whether steroid therapy is associated with improvements in respiratory function. Participants in this study ranged from 6-28 years of age from 20 centers and 10 countries. PFT measures included forced vital capacity (FVC, % FVC), forced expiratory volume in 1 s (FEV1, % FEV1), peak expiratory flow rate (PEFR, % PEFR), maximum inspiratory pressure (MIP), maximum expiratory pressure (MEP), and peak cough flow (PCF). The PFT measures were evaluated by current corticosteroid use, age groups (<10, 10-14, >15), and ambulatory status (ambulatory, part-time wheelchair user, full-time wheelchair user). All PFT measures except PCF were significantly different in the 3 age groups (p < 0.02). All PFT measures were significantly different when compared to ambulatory status (p < 0.01). All PFT measures except FVC, FEV1, and PEFR were significantly higher in steroid users than non-users (p < 0.01). Overall, all measures were most highly correlated in the oldest age group and least correlated in the youngest group. FEV1 and FVC were strongly correlated across age and ambulatory status (r = 0.9-0.99). PEFR was also strongly correlated with FEV1 (r = 0.77-0.92). PCF was the most variable and least correlated with other PFT measures (r = 0.16-0.71). Steroid use significantly affects respiratory muscles and performance of pulmonary function measures. There is clear delineation between steroid use, age, ambulatory status and change in PFTs suggesting PFTs are a suitable outcome measure to detect change over time for clinical trials in DMD.