The NHLBI lymphangioleiomyomatosis registry - Characteristics of 230 patients at enrollment
The NHLBI lymphangioleiomyomatosis registry - Characteristics of 230 patients at enrollment
复制标题
DOI：
10.1164/rccm.200409-1298oc
复制
发表时间：
2006-01-01
影响因子：
24.7
通讯作者：
Fanburg, BL
中科院分区：
文献类型：
--
作者：
Ryu, JH;Moss, J;Fanburg, BL
理论基础：肺淋巴管肌瘤病是一种进行性囊性肺部疾病，与非典型的平滑肌样细胞的渗透有关。目的：描述散发性和结节性硬化性肺淋巴管肌瘤病患者的临床特征。方法：在1998年至2001年的3年间，243例肺淋巴管肌瘤病患者登记在国家登记中；13名已接受肺移植的患者被排除在本报告的研究范围内。测量和主要结果：所有230名受试者均为女性，年龄18-76岁(平均+/-SE，44.5+/-0.65岁)。平均发病年龄为38.9±0.73岁，确诊时平均为41.0±0.65岁。结节性硬化症在14.8%的受试者中出现。肺部表现，最常见的自发性气胸，在86.5%的病例中是导致诊断的主要事件。近55%的受试者正在接受黄体酮衍生物的治疗。57.3%的受试者在肺功能测试中观察到阻塞性模式，而33.9%的受试者肺功能测试结果正常。与散发性形式相比，患有结节性硬化症相关淋巴管肌瘤病的女性更年轻，肺功能受损更少。结论：患有肺淋巴管肌瘤病的女性患者的年龄范围比以前认识的更广泛，肺功能的程度也可能有很大的差异，三分之一的受试者在登记时肺功能正常。
Rationale: Pulmonary lymphangioleiomyomatosis is a progressive cystic lung disease that is associated with infiltration of atypical smooth muscle-like cells. Previous descriptions of clinical characteristics of subjects with lymphangioleiomyomatosis have been based on a limited number of patients.Objectives: To describe the clinical characteristics of subjects with pulmonary lymphangioleiomyomatosis, both sporadic and tuberous sclerosis-related forms.Methods: Over a 3-yr period, from 1998 to 2001, 243 subjects with pulmonary lymphangioleiomyomatosis were enrolled into a national registry; 13 subjects who had already undergone lung transplantation were excluded for the purposes of this report.Measurements and Main Results: All 230 subjects were women, aged 18 to 76 yr (mean +/- SE, 44.5 +/- 0.65 yr). The average age at onset of symptoms was 38.9 +/- 0.73 yr and at diagnosis was 41.0 +/- 0.65 yr. Tuberous sclerosis complex was present in 14.8% of subjects. Pulmonary manifestations, most commonly spontaneous pneumothorax, were the primary events leading to the diagnosis in 86.5% of cases. Nearly 55% of the subjects were being treated with a progesterone derivative. An obstructive pattern on pulmonary function testing was observed in 57.3% of the subjects, whereas 33.9% had normal spirometric results. Women with tuberous sclerosis-related lymphangioleiomyomatosis were younger and had less impaired lung function compared with those with the sporadic form.Conclusions: The age range of women afflicted with pulmonary lymphangioleiomyomatosis is broader than previously appreciated and the degree of pulmonary function can be quite variable, with one-third of subjects having normal spirometry at enrollment into this registry.