Bach John R, Martinez Daniel, Saulat Bilal
Department of Physical Medicine and Rehabilitation, University Hospital, UMDNJ-The New Jersey Medical School, Newark, 07103, USA.
Am J Phys Med Rehabil. 2010 Aug;89(8):620-4. doi: 10.1097/PHM.0b013e3181e72207.
To describe the effect of glucocorticoid treatment on age at wheelchair dependence and at dependence on part-time (<23 hrs/day) and continuous noninvasive mechanical ventilation.
In this retrospective study, patients with Duchenne muscular dystrophy who received glucocorticoid therapy were compared with those who did not for ages at wheelchair dependence and when beginning part-time (nocturnal) and continuous noninvasive intermittent positive pressure ventilation (NIV). Respiratory symptoms, end-tidal carbon dioxide, oximetry, and vital capacity were noted every 4-12 mos, and NIV was initiated for symptomatic nocturnal hypoventilation. The daily NIV use increased until some required it continuously to survive.
The 117 untreated patients became wheelchair-dependent at 9.7 +/- 1.3 yrs of age. Four then died from cardiac failure, and five were older than 19 yrs without using NIV. The remaining 108 began nocturnal NIV at 19.2 +/- 3.7 yrs of age. Ninety of the 108 became continuously NIV-dependent at 21.9 +/- 4.5 yrs of age, and the 17 treated with glucocorticoid therapy became wheelchair-dependent significantly later at 10.8 +/- 1.3 yrs of age (P = 0.02). Three died from cardiac failure, and three were older than 19 yrs without using NIV. The remaining 11 began nocturnal NIV at 22.9 +/- 5.3 yrs of age (P = 0.05). Eight of the 11 became continuously NIV-dependent at age 28.9 +/- 7.3 yrs (P = 0.005).
Intermittent glucocorticoid therapy delays wheelchair dependence and may delay ventilator dependence for patients with Duchenne muscular dystrophy.
描述糖皮质激素治疗对杜氏肌营养不良症患者出现轮椅依赖、部分时间（<23小时/天）依赖和持续无创机械通气依赖的年龄的影响。
在这项回顾性研究中，将接受糖皮质激素治疗的杜氏肌营养不良症患者与未接受治疗的患者在出现轮椅依赖、开始部分时间（夜间）和持续无创间歇正压通气（NIV）的年龄方面进行比较。每4 - 12个月记录呼吸症状、呼气末二氧化碳、血氧饱和度和肺活量，对于有症状的夜间通气不足则开始使用NIV。每日NIV使用量逐渐增加，直至部分患者需要持续使用以维持生命。
117名未接受治疗的患者在9.7±1.3岁时出现轮椅依赖。其中4人死于心力衰竭，5人年龄超过19岁未使用NIV。其余108人在19.2±3.7岁时开始夜间NIV。108人中的90人在21.9±4.5岁时开始持续依赖NIV，而17名接受糖皮质激素治疗的患者在10.8±1.3岁时出现轮椅依赖的时间显著更晚（P = 0.02）。3人死于心力衰竭，3人年龄超过19岁未使用NIV。其余11人在22.9±5.3岁时开始夜间NIV（P = 0.05）。11人中的8人在28.9±7.3岁时开始持续依赖NIV（P = 0.005）。
间歇性糖皮质激素治疗可延迟杜氏肌营养不良症患者的轮椅依赖，并可能延迟其对呼吸机的依赖。
