Levine Hagit, Goldfarb Itai, Katz Julia, Carmeli Moriah, Shochat Tzippy, Mussaffi Huda, Aharoni Sharon, Prais Dario, Nevo Yoram
Pulmonary Institute, Schneider Children's Medical Center, Petah-Tikva, Israel.
Sackler Faculty of Medicine, Tel Aviv University, Tel Aviv, Israel.
Acta Paediatr. 2023 Apr;112(4):854-860. doi: 10.1111/apa.16653. Epub 2023 Jan 16.
In Duchenne muscular dystrophy (DMD), lung disease contributes significantly to morbidity and mortality. This study aimed to assess the usefulness of various pulmonary function tests in evaluating DMD severity.
This retrospective study analysed lung function tests of patients with DMD-treated in the multidisciplinary respiratory neuromuscular clinic at Schneiders' Children Medical Center of Israel. Data were analysed according to age, ambulatory status and glucocorticoid treatment.
Among 90 patients with DMD, 40/63 (63.5%) ambulatory patients and 22/27 (81.5%) nonambulatory patients successfully performed spirometry. Significant annual declines were demonstrated among nonambulatory patients, in percentile predicted forced vital capacity (3.8%) and in total lung capacity (5.5%) per year. The decline correlated with age and loss of ambulation but not with steroid treatment. Peak cough flow values were randomly distributed and did not correlate with age, ambulation or treatment. In nonambulatory patients, transcutaneous carbon dioxide measurement correlated significantly with age (r = 0.55, p = 0.02).
Forced vital capacity, total lung capacity and transcutaneous carbon dioxide correlated with the clinical severity of disease in children with DMD. These measures may be useful in follow-up and clinical trials. A comparable correlation was not found for peak cough flow.
在杜氏肌营养不良症（DMD）中，肺部疾病对发病率和死亡率有显著影响。本研究旨在评估各种肺功能测试在评估DMD严重程度方面的实用性。
这项回顾性研究分析了在以色列施耐德儿童医学中心多学科呼吸神经肌肉诊所接受治疗的DMD患者的肺功能测试。根据年龄、行走状态和糖皮质激素治疗情况对数据进行分析。
在90例DMD患者中，63例（63.5%）能行走的患者中有40例，27例（81.5%）不能行走的患者中有22例成功完成了肺活量测定。不能行走的患者中，预计用力肺活量百分比（每年3.8%）和肺总量（每年5.5%）出现显著的年度下降。这种下降与年龄和行走能力丧失相关，但与类固醇治疗无关。峰值咳嗽流速值呈随机分布，与年龄、行走能力或治疗无关。在不能行走的患者中，经皮二氧化碳测量值与年龄显著相关（r = 0.55，p = 0.02）。
用力肺活量、肺总量和经皮二氧化碳与DMD患儿的临床疾病严重程度相关。这些测量方法可能对随访和临床试验有用。未发现峰值咳嗽流速有类似的相关性。
