Pediatric and Neonatology Department, Hospital "F. Del Ponte", University of Insubria, Varese, Italy.
Pediatric Department, ASST-Lariana, Sant'Anna Hospital, San Fermo della Battaglia, Como, Italy.
Am J Med Genet A. 2021 Feb;185(2):390-396. doi: 10.1002/ajmg.a.61966. Epub 2020 Nov 10.
Williams-Beuren syndrome (WBS) is caused by an haploinsufficiency of the 7q11.2 region which involves the elastin gene (ELN). A deficiency of elastin is a known pathophysiological mechanism of emphysema/chronic obstructive pulmonary disease (COPD). A previous study hypothesized a higher risk of COPD in WBS patients. Herein, this phenomenon was further investigated looking for a possible correlation between COPD and WBS. Dynamic lung volumes (forced vital capacity [FVC], FEV1, FEV1/FVC) were measured in 22 patients (age range 18.9 ± 7.4 years) affected with WBS, genetically confirmed, correlating these parameters to respiratory risk factors. Dyspnea, cough and wheezing were detected in 6/22 (27%) patients. Obstructive and restrictive patterns were identified in 6/22 (27%) and 2/22 (9%) cases, respectively with no evidence of irreversible obstruction. CVF, FEV1 and FEV1/CVF mean values were all normal, with values of 91.3% (n.v. > 80%), 84.2% (n.v. > 80%) and 0.82 (n.v. > 0.7), respectively. The severity of the comorbidities did not show a cause-effect relation with the respiratory patterns, nevertheless patients treated with anti-hypertensive drugs had poorer pulmonary function. Our findings are in accordance with previous observations, showing that emphysema/COPD is not a typical finding in young patients with WBS. However, a respiratory function assessment should be included in the follow-up of WBS patients, especially in adolescents/young adults under treatment with anti-hypertensive drugs.
威廉姆斯-比伦综合征(WBS)是由 7q11.2 区域的单倍体不足引起的,该区域涉及弹性蛋白基因(ELN)。弹性蛋白缺乏是肺气肿/慢性阻塞性肺疾病(COPD)的已知病理生理学机制。先前的研究假设 WBS 患者患 COPD 的风险更高。在此,进一步研究了这种现象,寻找 COPD 与 WBS 之间可能的相关性。对 22 名(年龄范围 18.9±7.4 岁)经基因证实患有 WBS 的患者进行了动态肺量(用力肺活量 [FVC]、FEV1、FEV1/FVC)测量,将这些参数与呼吸危险因素相关联。22 名患者中有 6/22(27%)出现呼吸困难、咳嗽和喘息。6/22(27%)和 2/22(9%)的患者分别存在阻塞性和限制性模式,没有不可逆性阻塞的证据。CVF、FEV1 和 FEV1/CVF 的平均值均正常,分别为 91.3%(n.v.>80%)、84.2%(n.v.>80%)和 0.82(n.v.>0.7)。合并症的严重程度与呼吸模式没有因果关系,但接受抗高血压药物治疗的患者肺功能较差。我们的研究结果与先前的观察结果一致,表明肺气肿/COPD 不是患有 WBS 的年轻患者的典型发现。然而,在 WBS 患者的随访中,应包括呼吸功能评估,特别是在接受抗高血压药物治疗的青少年/年轻成年人中。