Marthin June Kehlet
Pediatric Clinic I, Juliane Marie Centret, Rigshospitalet, Blegdamsvej 9, 2100 Kobenhavn Ø, Denmark.
Dan Med Bull. 2010 Aug;57(8):B4174.
Primary ciliary dyskinesia (PCD) is a rare, usually autosomal recessive inherited disorder, characterized by abnormalities in ciliary structure and/or function. Frequent, intermittent or chronic airway infections precipitated by impaired airway mucociliary clearance may cause permanent lung damage and reduced lung function. Early diagnosis is considered important for the prevention of lung damage, but diagnosis is probably often delayed or even missed since diagnosis of PCD is both complex and time consuming, and yet not always exact. The aims of this PhD thesis were to evaluate the discriminative capacity and "real-life" clinical application of two candidates for supplemental diagnostic testing for PCD: Nasal nitric oxide (nNO) measurement placed as a first line test to point out probable PCD patients for further investigation or exclude patients, regardless of age, Pulmonary radioaerosol mucociliary clearance (PRMC) as a second line test for PCD investigation in children from 5 years of age. And additionally, Proposing an algorithm for the pathway of diagnosing PCD based on these two studies and recommendations from the literature. Nasal NO and PRMC demonstrated to be two highly valid supplementary diagnostic tools to be placed in each end of the diagnostic pathway when investigating selected patients referred for PCD work up. Nasal NO measurement demonstrated to have an obvious place as a first line test in the pathway of PCD investigation and PRMC as second line test as a supplement to ciliary function test and EM-test in cases of difficult diagnoses. Neither of these tests can stand alone in diagnosis or excluding of PCD. PCD remains to be a diagnosis that should be made at a tertiary PCD centre, as clinical evaluation of referred patients is crucial before excluding the disease.
原发性纤毛运动障碍(PCD)是一种罕见的、通常为常染色体隐性遗传的疾病,其特征是纤毛结构和/或功能异常。气道黏液纤毛清除功能受损引发的频繁、间歇性或慢性气道感染可能导致永久性肺损伤和肺功能下降。早期诊断被认为对预防肺损伤很重要,但PCD的诊断往往会延迟甚至漏诊,因为其诊断既复杂又耗时,而且并不总是准确的。本博士论文的目的是评估两种PCD补充诊断测试方法的鉴别能力和“实际”临床应用:鼻一氧化氮(nNO)测量作为一线测试,用于指出可能患有PCD的患者以便进一步检查或排除患者,无论其年龄大小;肺放射性气溶胶黏液纤毛清除率(PRMC)作为二线测试,用于对5岁及以上儿童进行PCD调查。此外,基于这两项研究以及文献中的建议,提出一种PCD诊断途径的算法。鼻一氧化氮和肺放射性气溶胶黏液纤毛清除率被证明是两种高度有效的补充诊断工具,在对转诊进行PCD检查的特定患者进行调查时,应置于诊断途径的两端。鼻一氧化氮测量在PCD调查途径中作为一线测试有明显的作用,而肺放射性气溶胶黏液纤毛清除率作为二线测试,在诊断困难的情况下作为纤毛功能测试和电子显微镜检查的补充。这两种测试都不能单独用于诊断或排除PCD。PCD仍需在三级PCD中心进行诊断,因为在排除该疾病之前,对转诊患者进行临床评估至关重要。