Jorissen M, Willems T, Van der Schueren B, Verbeken E, De Boeck K
ENT Department, UZ GHB, Leuven.
Acta Otorhinolaryngol Belg. 2000;54(3):343-56.
During the period 1990-1999 84 PCD patients were identified and characterized. The expression of inherited abnormalities in primary ciliary dyskinesia after ciliogenesis was investigated in 41 patients with dynein deficiency, 6 patients with absence of the central pair of microtubules and 24 PCD patients with normal ultrastructure. In patients with dynein deficiency, the outer dynein arms counts were 1.9 +/- 1.0 in the biopsies and 1.6 +/- 0.7 after ciliogenesis. Secondary abnormalities were found in 15.8 +/- 20.4% of the transverse sections of cilia and only in 1.0 +/- 1.3% after ciliogenesis. Ciliary orientation was 28 +/- 11 degrees and 24 +/- 10 degrees respectively in biopsies and cultures. In patients with absence of the central pair this was found in 15 +/- 16% in biopsies and 21 +/- 19% after ciliogenesis. The values for the outer dynein arm were 8.4 +/- 0.3 and 8.7 +/- 0.2 and for the secondary abnormalities were 11.7 +/- 7.3% and 0.5 +/- 1.3% in the biopsies, respectively after ciliogenesis. In patients with normal ultrastructure the scores for the dynein arms were similar. Secondary abnormalities were found in 12.2 +/- 11.7% in the biopsies and 0.6 +/- 0.9% after ciliogenesis while ciliary orientation was respectively 21 +/- 7 degrees and 25 +/- 8 degrees. In conclusion, inherited abnormalities in primary ciliary dyskinesia are expressed after ciliogenesis, while secondary abnormalities are virtually absent, thereby facilitating the ultrastructural diagnosis.
在1990年至1999年期间,共识别并表征了84例原发性纤毛运动障碍(PCD)患者。对41例动力蛋白缺乏患者、6例中央微管对缺失患者以及24例超微结构正常的PCD患者,研究了纤毛生成后原发性纤毛运动障碍中遗传异常的表达情况。在动力蛋白缺乏的患者中,活检时外动力蛋白臂计数为1.9±1.0,纤毛生成后为1.6±0.7。在纤毛横切面中,15.8±20.4%发现有继发性异常,而纤毛生成后仅1.0±1.3%有继发性异常。活检和培养时纤毛方向分别为28±11度和24±10度。对于中央微管对缺失的患者,活检时为15±16%,纤毛生成后为21±19%。活检时外动力蛋白臂的值分别为8.4±0.3和8.7±0.2,继发性异常在活检时分别为11.7±7.3%和纤毛生成后0.5±1.3%。超微结构正常的患者中,动力蛋白臂评分相似。活检时12.2±11.7%发现有继发性异常,纤毛生成后为0.6±0.9%,而纤毛方向分别为21±7度和25±8度。总之,原发性纤毛运动障碍中的遗传异常在纤毛生成后表达,而继发性异常几乎不存在,从而便于进行超微结构诊断。