Longitudinal pulmonary function testing outcome measures in Duchenne muscular dystrophy: Long-term natural history with and without glucocorticoids
Longitudinal pulmonary function testing outcome measures in Duchenne muscular dystrophy: Long-term natural history with and without glucocorticoids
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DOI：
10.1016/j.nmd.2018.07.004
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发表时间：
2018-11-01
影响因子：
2.8
通讯作者：
Abresch, Richard T.
中科院分区：
文献类型：
--
作者：
McDonald, Craig M.;Gordish-Dressman, Heather;Abresch, Richard T.
我们描述了一项多中心前瞻性队列研究--国际合作研究组杜氏自然史研究中，与未接受糖皮质激素治疗的患者相比，接受糖皮质激素（GC）治疗> 1年的杜氏肌营养不良患者肺功能指标随时间的变化。397名参与者在长达10年的时间内接受了2799次肺功能评估。将53名GC初治参与者（< 1个月暴露）与322名累积GC治疗> 1年的受试者进行比较。测定用力肺活量（FVC）、最大呼气流速（PEFr）、最大吸气压和呼气压，并计算为预测百分比（%p）。在7-9.9岁的患者中，GC治疗减缓了肺功能下降的速率（通过FVC%p测量）。在基线FVC%p < 80- 34%的患者中，GC治疗在更大程度上减缓了12个月和24个月预测肺功能百分比的进展。GC治疗导致FVC和PEFr绝对峰值较高，下降开始较晚。GC治疗延迟了绝对FVC < 1升的进展。FVC低于1 L的患者死亡的可能性增加4.1倍（p=0.017）。长期糖皮质激素治疗可延缓Duchenne营养不良患者的肺部疾病进展(C)2018作者由爱思唯尔公司出版
We describe changes in pulmonary function measures across time in Duchenne muscular dystrophy patients treated with glucocorticoids (GCs) > 1 year compared to GC naive patients in the Cooperative International Research Group Duchenne Natural History Study, a multicenter prospective cohort study. 397 participants underwent 2799 pulmonary function assessments over a period up to 10 years. Fifty-three GC naive participants (< 1 month exposure) were compared to 322 subjects with > 1 year cumulative GC treatment. Forced vital capacity (FVC), peak expiratory flow rate (PEFr), maximal inspiratory and expiratory pressures were performed and calculated as a percent predicted (%p). GC treatment slowed the rate of pulmonary decline as measured by FVC%p, in patients aged 7-9.9 years. GC treatment slowed 12 and 24-month progression of percent predicted spirometry to a greater degree in those with baseline FVC%p from < 80-34%. GC treatment resulted in higher peak absolute FVC and PEFr values with later onset of decline. Progression to an absolute FVC < 1 liter was delayed by GC treatment. Patients who reached a FVC below 1 L were 4.1 times more likely to die (p=0.017). Long-term glucocorticoid treatment slows pulmonary disease progression in Duchenne dystrophy throughout the lifespan. (C) 2018 The Authors. Published by Elsevier B.V.