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芬兰 Kallmann 综合征的发病率、表型特征和分子遗传学。

Incidence, phenotypic features and molecular genetics of Kallmann syndrome in Finland.

机构信息

Children's Hospital, Helsinki University Central Hospital, University of Helsinki, FI-00029 Helsinki, Finland.

出版信息

Orphanet J Rare Dis. 2011 Jun 17;6:41. doi: 10.1186/1750-1172-6-41.

Abstract

BACKGROUND

Kallmann syndrome (KS), comprised of congenital hypogonadotropic hypogonadism (HH) and anosmia, is a clinically and genetically heterogeneous disorder. Its exact incidence is currently unknown, and a mutation in one of the identified KS genes has only been found in ~30% of the patients.

METHODS

Herein, we investigated epidemiological, clinical, and genetic features of KS in Finland.

RESULTS

The minimal incidence estimate of KS in Finland was 1:48 000, with clear difference between males (1:30 000) and females (1:125 000) (p = 0.02). The reproductive phenotype of 30 probands (25 men; 5 women) ranged from severe HH to partial puberty. Comprehensive mutation analysis of all 7 known KS genes (KAL1, FGFR1, FGF8, PROK2, PROKR2, CHD7, and WDR11) in these 30 well-phenotyped probands revealed mutations in KAL1 (3 men) and FGFR1 (all 5 women vs. 4/25 men), but not in other genes.

CONCLUSIONS

Our results suggest that Finnish KS men harbor mutations in gene(s) yet-to-be discovered with sex-dependent penetrance of the disease phenotype. In addition, some KS patients without CHD7 mutations display CHARGE-syndrome associated phenotypic features (e.g. ear or eye anomalies), possibly implying that, in addition to CHD7, there may be other genes associated with phenotypes ranging from KS to CHARGE.

摘要

背景

卡尔曼综合征(KS)由先天性促性腺激素低下性性腺功能减退症(HH)和嗅觉缺失组成,是一种临床表现和遗传均具有异质性的疾病。其确切发病率目前尚不清楚,在已鉴定的 KS 基因中,只有约 30%的患者存在基因突变。

方法

在此,我们研究了芬兰 KS 的流行病学、临床和遗传特征。

结果

芬兰 KS 的最小发病率估计为 1:48000,男性(1:30000)和女性(1:125000)之间存在明显差异(p=0.02)。30 名先证者(25 名男性;5 名女性)的生殖表型从严重 HH 到部分青春期不等。对这 30 名表型明确的先证者的所有 7 个已知 KS 基因(KAL1、FGFR1、FGF8、PROK2、PROKR2、CHD7 和 WDR11)进行全面的突变分析,发现 KAL1 突变(3 名男性)和 FGFR1 突变(所有 5 名女性,而非 4/25 名男性),但在其他基因中未发现突变。

结论

我们的结果表明,芬兰 KS 男性携带尚未发现的基因(s)突变,且疾病表型具有性别依赖性外显率。此外,一些没有 CHD7 突变的 KS 患者表现出 CHARGE 综合征相关的表型特征(如耳或眼异常),这可能意味着除了 CHD7 之外,可能还有其他基因与从 KS 到 CHARGE 的表型相关。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/1534/3143089/68d06a1f2d99/1750-1172-6-41-1.jpg

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