Stewart B, Zabner J, Shuber A P, Welsh M J, McCray P B
Department of Pediatrics, Howard Hughes Medical Institute, University of Iowa, College of Medicine, Iowa City 52242.
Am J Respir Crit Care Med. 1995 Mar;151(3 Pt 1):899-903. doi: 10.1164/ajrccm/151.3_Pt_1.899.
Since its introduction in 1959, the sweat test has remained the "gold standard" diagnostic test for cystic fibrosis (CF). It is apparent that CF encompasses a wide spectrum of disease, from meconium ileus and severe respiratory compromise in infants to the presentation of mild pulmonary symptoms and no evidence of gastrointestinal disease in adults. In patients with lung disease that might otherwise be consistent with CF, normal sweat chloride (Cl-) values have tended to exclude the diagnosis. In this report we describe two patients from two families with the compound heterozygotic CF mutations delta F508/3849 + 10 kb C-->T. These patients had mild manifestations of disease, including clinical pancreatic sufficiency (normal growth without pancreatic enzyme supplementation) and absence of azoospermia. Sweat Cl- values were in the normal range. However, both patients developed bronchiectasis, progressive obstructive lung disease, and colonization with Pseudomonas. The diagnosis of CF was made using nasal transepithelial voltage measurements and genotyping. These cases emphasize the need to maintain a high index of suspicion of CF in atypical cases, and to pursue alternative diagnostic tests to confirm a diagnosis of CF suspected on clinical grounds, despite normal sweat test results.
自1959年引入以来,汗液测试一直是囊性纤维化(CF)的“金标准”诊断测试。显然,CF涵盖了广泛的疾病范围,从胎粪性肠梗阻和婴儿严重的呼吸功能不全到成人出现轻度肺部症状且无胃肠道疾病证据。在可能与CF相符的肺部疾病患者中,正常的汗液氯化物(Cl-)值往往排除了该诊断。在本报告中,我们描述了来自两个家庭的两名患者,他们具有复合杂合CF突变delta F508/3849 + 10 kb C→T。这些患者有轻度疾病表现,包括临床胰腺功能充足(无需补充胰酶即可正常生长)且无无精子症。汗液Cl-值在正常范围内。然而,两名患者均出现支气管扩张、进行性阻塞性肺病以及铜绿假单胞菌定植。通过鼻上皮电压测量和基因分型做出了CF诊断。这些病例强调,在非典型病例中需要高度怀疑CF,并采用替代诊断测试来确认基于临床怀疑但汗液测试结果正常的CF诊断。