Bai Yin, Zhang Jing, You Shaohua, Ji Lingchao, Jia Jingjie, Wang Hongtian
Department of Otorhinolaryngology Head Neck Surgery, People Liberation Army General Hospital, Beijing 100853, China.
Department of Otorhinolaryngology Head Neck Surgery, People Liberation Army General Hospital, Beijing 100853, China. Email:
Zhonghua Er Bi Yan Hou Tou Jing Wai Ke Za Zhi. 2014 Feb;49(2):115-20.
To analyze the clinical characteristics of primary ciliary dyskinesia(PCD) so as to improve the diagnostic level of this rarely seen disease.
Ten patients with PCD were retrospectively reviewed, the medical history, symptoms, signs, lung CT or chest X-ray, rhino-sinus CT scan, nasal nitric oxide (NO) levels, nasal ciliary ultrastructure, DNAH5 and DNAH11 genetic mutation, as well as treatment outcome were analyzed.
All 10 patients had recurrent chronic sinusitis, otitis media, bronchitis/bronchiectasis since childhood. Nine cases with translocation of heart and big vessels were diagnosed as Kartagener syndrome. One woman was suffering from barrenness and one man sterility after marriage for long time without birth control. Nasal NO levels were significantly lower in 2 patients with PCD but it was almost normal in one patient. Ciliary ultrastructure investigated by transmission electron microscope were almost normal in 4 cases without missing of inner or outer dynein arms. Two cases taking exome capture sequencing showed that mutations happened in DNAH5 and DNAH11. Five subjects underwenting sanger sequencing on 6 common exon fragments of DNAH5 and DNAH11 did not show any abnormality. Ten cases took medication therapy, while 5 patients once underwent functional endoscope sinus surgery. All of the 10 patients had improvement of their symptoms and signs after treatment.
The PCD is so rare in clinic that it is easily misdiagnosed. Clinical characteristics, nasal NO levels, ciliary ultrastructure and genetic testing are significant for clinical diagnosis.
分析原发性纤毛运动障碍(PCD)的临床特征,以提高对这种罕见疾病的诊断水平。
回顾性分析10例PCD患者的病史、症状、体征、肺部CT或胸部X线、鼻窦CT扫描、鼻一氧化氮(NO)水平、鼻纤毛超微结构、DNAH5和DNAH11基因突变以及治疗结果。
10例患者自幼均有反复慢性鼻窦炎、中耳炎、支气管炎/支气管扩张。9例心脏和大血管移位患者被诊断为卡塔格内综合征。1名女性婚后长期不孕,1名男性婚后长期不育且未采取避孕措施。2例PCD患者鼻NO水平显著降低,但1例患者鼻NO水平基本正常。透射电子显微镜观察的纤毛超微结构在4例患者中基本正常,未见内、外动力臂缺失。2例采用外显子捕获测序显示DNAH5和DNAH11发生突变。5例对DNAH5和DNAH11的6个常见外显子片段进行桑格测序的受试者未显示任何异常。10例患者接受药物治疗,5例患者曾接受功能性鼻内镜鼻窦手术。10例患者治疗后症状和体征均有改善。
PCD在临床上极为罕见,易被误诊。临床特征、鼻NO水平、纤毛超微结构及基因检测对临床诊断具有重要意义。