Children's Hospital, Helsinki University Central Hospital, University of Helsinki, FI-00029 Helsinki, Finland.
Orphanet J Rare Dis. 2011 Jun 17;6:41. doi: 10.1186/1750-1172-6-41.
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.
Herein, we investigated epidemiological, clinical, and genetic features of KS in Finland.
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.
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.
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。
在此,我们研究了芬兰 KS 的流行病学、临床和遗传特征。
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。
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。