Bahi-Buisson Nadia, Nectoux Juliette, Rosas-Vargas Haydeé, Milh Mathieu, Boddaert Nathalie, Girard Benoit, Cances Claude, Ville Dorothée, Afenjar Alexandra, Rio Marlène, Héron Delphine, N'guyen Morel Marie Ange, Arzimanoglou Alexis, Philippe Christophe, Jonveaux Philippe, Chelly Jamel, Bienvenu Thierry
Pediatric Neurology, Department of Pediatrics, Necker Enfants Malades Hospital, AP-HP, Paris V, Paris, France.
Brain. 2008 Oct;131(Pt 10):2647-61. doi: 10.1093/brain/awn197. Epub 2008 Sep 12.
Mutations in the human X-linked cyclin-dependent kinase-like 5 (CDKL5) gene have been shown to cause infantile spasms as well as Rett syndrome (RTT)-like phenotype. To date, less than 25 different mutations have been reported. So far, there are still little data on the key clinical diagnosis criteria and on the natural history of CDKL5-associated encephalopathy. We screened the entire coding region of CDKL5 for mutations in 183 females with encephalopathy with early seizures by denaturing high liquid performance chromatography and direct sequencing, and we identified in 20 unrelated girls, 18 different mutations including 7 novel mutations. These mutations were identified in eight patients with encephalopathy with RTT-like features, five with infantile spasms and seven with encephalopathy with refractory epilepsy. Early epilepsy with normal interictal EEG and severe hypotonia are the key clinical features in identifying patients likely to have CDKL5 mutations. Our study also indicates that these patients clearly exhibit some RTT features such as deceleration of head growth, stereotypies and hand apraxia and that these RTT features become more evident in older and ambulatory patients. However, some RTT signs are clearly absent such as the so called RTT disease profile (period of nearly normal development followed by regression with loss of acquired fine finger skill in early childhood and characteristic intensive eye communication) and the characteristic evolution of the RTT electroencephalogram. Interestingly, in addition to the overall stereotypical symptomatology (age of onset and evolution of the disease) resulting from CDKL5 mutations, atypical forms of CDKL5-related conditions have also been observed. Our data suggest that phenotypic heterogeneity does not correlate with the nature or the position of the mutations or with the pattern of X-chromosome inactivation, but most probably with the functional transcriptional and/or translational consequences of CDKL5 mutations. In conclusion, our report show that search for mutations in CDKL5 is indicated in girls with early onset of a severe intractable seizure disorder or infantile spasms with severe hypotonia, and in girls with RTT-like phenotype and early onset seizures, though, in our cohort, mutations in CDKL5 account for about 10% of the girls affected by these disorders.
人类X连锁细胞周期蛋白依赖性激酶样5(CDKL5)基因突变已被证明可导致婴儿痉挛以及雷特综合征(RTT)样表型。迄今为止,报道的不同突变少于25种。到目前为止,关于关键临床诊断标准以及CDKL5相关脑病自然史的数据仍然很少。我们通过变性高效液相色谱法和直接测序,对183例有早期癫痫发作的脑病女性患者的CDKL5整个编码区进行了突变筛查,在20名无亲缘关系的女孩中鉴定出18种不同突变,包括7种新突变。这些突变在8例具有RTT样特征的脑病患者、5例婴儿痉挛患者和7例难治性癫痫脑病患者中被发现。发作间期脑电图正常的早期癫痫和严重肌张力减退是识别可能存在CDKL5突变患者的关键临床特征。我们的研究还表明,这些患者明显表现出一些RTT特征,如头围生长减速、刻板动作和手部失用症,并且这些RTT特征在年龄较大且能行走的患者中更为明显。然而,一些RTT体征明显不存在,如所谓的RTT疾病特征(近乎正常发育阶段,随后在幼儿期出现发育倒退并丧失获得性精细手指技能以及特征性的频繁眼神交流)以及RTT脑电图的特征性演变。有趣的是,除了CDKL5突变导致的总体刻板症状(发病年龄和疾病演变)外,还观察到了CDKL5相关病症的非典型形式。我们的数据表明,表型异质性与突变的性质或位置或X染色体失活模式无关,但很可能与CDKL5突变的功能性转录和/或翻译后果有关。总之,我们的报告表明,对于严重难治性癫痫发作或伴有严重肌张力减退的婴儿痉挛早期发作的女孩,以及具有RTT样表型和早期癫痫发作的女孩,应进行CDKL5突变检测,不过,在我们的队列中,CDKL5突变约占受这些疾病影响女孩的10%。