Schroeder Scott A, Zielen Stefan
Division of Pulmonary Medicine and Allergy, Department of Pediatrics, The Floating Hospital, Tufts Medical Center, 800 Washington St., Boston, Massachusetts, 02111.
Pediatr Pulmonol. 2014 Apr;49(4):389-99. doi: 10.1002/ppul.22817. Epub 2013 Jun 13.
Ataxia-telangiectasia (A-T) is an autosomal recessive multiorgan disease characterized by progressive neurologic deterioration in which the most common causes of death are diseases of the respiratory system and cancer. This retrospective case-series characterizes the pulmonary disease seen in 101 cancer-free A-T patients who were living with chronic respiratory symptoms or who had pulmonary disease listed as the cause of their death. All of the 101 patients developed chronic progressive pulmonary disease with intermittent acute exacerbations and irreversible radiographic changes. Seventy-nine patients had microbiologic analysis of their respiratory secretions and of the patients less than 15 years of age, 25/27 cultured Staphylococcus aureus, Hemophilus influenzae, or Streptococcus pneumoniae and 35/47 older patients cultured Pseudomonas aeruginosa. Opportunistic infections of the lungs were not observed. Thirty-two patients had histologic examination of their lungs and 21 of the specimens had both acute and chronic inflammatory changes. The results of this study describe the natural history of the suppurative lung disease in patients with A-T that often progresses to bronchiectasis and pneumonias and ultimately respiratory failure. The etiology of the progressive deterioration is unknown but the pulmonary disease of A-T described in this study shows similarities to the lung disease seen in cystic fibrosis. Treatment of the lung disease in patients with A-T may benefit from many of the same therapies that have improved the quality of life for patients with cystic fibrosis.
共济失调毛细血管扩张症(A-T)是一种常染色体隐性多器官疾病,其特征为进行性神经功能恶化,最常见的死亡原因是呼吸系统疾病和癌症。本回顾性病例系列描述了101例无癌症的A-T患者的肺部疾病情况,这些患者伴有慢性呼吸道症状或其死亡原因列为肺部疾病。101例患者均出现慢性进行性肺部疾病,伴有间歇性急性加重和不可逆的影像学改变。79例患者对其呼吸道分泌物进行了微生物学分析,15岁以下患者中,27例中有25例培养出金黄色葡萄球菌、流感嗜血杆菌或肺炎链球菌,47例年龄较大的患者中有35例培养出铜绿假单胞菌。未观察到肺部机会性感染。32例患者进行了肺部组织学检查,21份标本同时有急性和慢性炎症改变。本研究结果描述了A-T患者化脓性肺部疾病的自然史,该疾病常进展为支气管扩张和肺炎,最终导致呼吸衰竭。病情进行性恶化的病因尚不清楚,但本研究中描述的A-T肺部疾病与囊性纤维化患者的肺部疾病有相似之处。对A-T患者肺部疾病的治疗可能受益于许多同样改善了囊性纤维化患者生活质量的治疗方法。