Castori Marco, Dordoni Chiara, Valiante Michele, Sperduti Isabella, Ritelli Marco, Morlino Silvia, Chiarelli Nicola, Celletti Claudia, Venturini Marina, Camerota Filippo, Calzavara-Pinton Piergiacomo, Grammatico Paola, Colombi Marina
Division of Medical Genetics, Department of Molecular Medicine, Sapienza University, San Camillo-Forlanini Hospital, Rome, Italy.
Am J Med Genet A. 2014 Dec;164A(12):3010-20. doi: 10.1002/ajmg.a.36805. Epub 2014 Oct 22.
Joint hypermobility syndrome (JHS) and Ehlers-Danlos syndrome, hypermobility type (EDS-HT) are two markedly overlapping heritable connective tissue disorders. The cumulative frequency of JHS and EDS-HT seems high, but their recognition remains an exclusion diagnosis based on different sets of diagnostic criteria. Although proposed by a panel of experts, clinical identity between JHS and EDS-HT is still a matter of debate due to unknown molecular basis. We present 23 families with three or more individuals with a diagnosis of JHS and/or EDS-HT. Rough data from the 82 individuals were used to assess the frequency of major and minor criteria, as well as selected additional features. A series of statistical tools were applied to assess intrafamilial and interfamilial variability, emphasizing intergenerational, and intersex differences. This study demonstrates marked heterogeneity within and between families in terms of agreement of available diagnostic criteria. In 21 pedigrees affected individuals belong to two or three phenotypic sub-categories among JHS, EDS-HT, and JHS + EDS-HT overlap. Intergenerational analysis depicts a progressive shifting, also within the same pedigree, from EDS-HT in childhood, to JHS + EDS-HT in early adulthood and JHS later in life. Female-male ratio is 2.1:1, which results lower than previously observed in unselected patients' cohorts. In these pedigrees, JHS, EDS-HT, and JHS + EDS-HT segregate as a single dominant trait with complete penetrance, variable expressivity, and a markedly evolving phenotype. This study represents a formal demonstration that EDS-HT and JHS contitute the same clinical entity, and likely share the same genetic background, at least, in familial cases.
关节过度活动综合征(JHS)和高活动型埃勒斯-当洛综合征(EDS-HT)是两种明显重叠的遗传性结缔组织疾病。JHS和EDS-HT的累积发病率似乎很高,但它们的诊断仍基于不同的诊断标准集进行排除性诊断。尽管由专家小组提出,但由于分子基础不明,JHS和EDS-HT之间的临床一致性仍是一个有争议的问题。我们展示了23个家庭,每个家庭中有三个或更多被诊断为JHS和/或EDS-HT的个体。来自82名个体的粗略数据用于评估主要和次要标准的频率,以及选定的其他特征。应用了一系列统计工具来评估家族内和家族间的变异性,重点是代际和性别差异。这项研究表明,在可用诊断标准的一致性方面,家庭内部和家庭之间存在明显的异质性。在21个家系中,受影响的个体属于JHS、EDS-HT和JHS + EDS-HT重叠中的两个或三个表型亚类。代际分析描绘了一种渐进性的转变,即使在同一个家系中,也从儿童期的EDS-HT转变为成年早期的JHS + EDS-HT,再到生命后期的JHS。女性与男性的比例为2.1:1,低于先前在未选择的患者队列中观察到的比例。在这些家系中,JHS、EDS-HT和JHS + EDS-HT作为单一显性性状分离,具有完全外显率、可变表达率和明显演变的表型。这项研究正式证明,至少在家族性病例中,EDS-HT和JHS构成相同的临床实体,并且可能具有相同的遗传背景。