Grody W W, Dunkel-Schetter C, Tatsugawa Z H, Fox M A, Fang C Y, Cantor R M, Novak J M, Bass H N, Crandall B F
Department of Pathology, University of California, Los Angeles 90095-1732, USA.
Am J Hum Genet. 1997 Apr;60(4):935-47.
As the most common lethal autosomal recessive disorder in North America, cystic fibrosis (CF) is an obvious candidate for general population carrier screening. Although the identification of the causative gene has made detection of asymptomatic carriers possible, the extreme heterogeneity of its mutations has limited the sensitivity of the available DNA screening tests and has called into question their utility when they are applied to patients with no family history of the disease. The purpose of this study was to determine the technical feasibility, patient acceptance and understanding, and psychosocial impact of large-scale CF carrier screening in an ethnically diverse pregnant population. A total of 4,739 pregnant women attending prenatal clinics located in both an academic medical center and a large HMO were invited in person to participate. Of this group, 3,543 received CF instruction and assessments of knowledge and mood, and 3,192 underwent DNA testing for the six most common CF mutations, by means of a noninvasive PCR-based reverse-dot-blot method. Overall participation rates (ranging from 53% at the HMO to 77% at the academic center) and consent rates for DNA testing after CF instruction (>98%) exceeded those of most other American studies. The PCR-based screening method worked efficiently on large numbers of samples, and 55 carriers and one at-risk couple were identified. Understanding of residual risk, anxiety levels, and overall satisfaction with the program were acceptable across all ethnic groups. Our strategy of approaching a motivated pregnant population in person with a rapid and noninvasive testing method may provide a practical model for developing a larger CF screening program targeting appropriate high-risk groups at the national level, and may also serve as a paradigm for population-based screening of other genetically heterogeneous disorders in the future.
作为北美最常见的致死性常染色体隐性疾病,囊性纤维化(CF)显然是普通人群携带者筛查的合适对象。尽管致病基因的鉴定使得检测无症状携带者成为可能,但其突变的极端异质性限制了现有DNA筛查测试的敏感性,并使人对其应用于无家族病史患者时的效用产生质疑。本研究的目的是确定在种族多样化的孕妇群体中进行大规模CF携带者筛查的技术可行性、患者接受度和理解程度以及社会心理影响。共有4739名在学术医疗中心和大型健康维护组织(HMO)的产前诊所就诊的孕妇被亲自邀请参与。在这组人群中,3543人接受了CF指导以及知识和情绪评估,3192人通过基于非侵入性PCR的反向点杂交方法对六种最常见的CF突变进行了DNA检测。总体参与率(从HMO的53%到学术中心的77%)以及CF指导后DNA检测的同意率(>98%)超过了大多数其他美国研究。基于PCR的筛查方法在大量样本上有效工作,共识别出55名携带者和一对高危夫妇。所有种族群体对残余风险的理解、焦虑水平以及对该项目的总体满意度都处于可接受范围。我们采用快速、非侵入性检测方法亲自接触有积极性的孕妇群体的策略,可能为在国家层面制定针对适当高危群体的更大规模CF筛查项目提供一个实用模型,也可能成为未来基于人群筛查其他基因异质性疾病的范例。