Donkervoort S, Bonnemann C G, Loeys B, Jungbluth H, Voermans N C
Am J Med Genet C Semin Med Genet. 2015 Mar;169C(1):23-42. doi: 10.1002/ajmg.c.31433.
Joint hypermobility is the defining feature of various inherited connective tissue disorders such as Marfan syndrome and various types of Ehlers-Danlos syndrome and these will generally be the first conditions to be considered by geneticists and pediatricians in the differential diagnosis of a patient presenting with such findings. However, several congenital and adult-onset inherited myopathies also present with joint hypermobility in the context of often only mild-to-moderate muscle weakness and should, therefore, be included in the differential diagnosis of joint hypermobility. In fact, on the molecular level disorders within both groups represent different ends of the same spectrum of inherited extracellular matrix (ECM) disorders. In this review we will summarize the measures of joint hypermobility, illustrate molecular mechanisms these groups of disorders have in common, and subsequently discuss the clinical features of: 1) the most common connective tissue disorders with myopathic or other neuromuscular features: Ehlers-Danlos syndrome, Marfan syndrome and Loeys-Dietz syndrome; 2) myopathy and connective tissue overlap disorders (muscle extracellular matrix (ECM) disorders), including collagen VI related dystrophies and FKBP14 related kyphoscoliotic type of Ehlers-Danlos syndrome; and 3) various (congenital) myopathies with prominent joint hypermobility including RYR1- and SEPN1-related myopathy. The aim of this review is to assist clinical geneticists and other clinicians with recognition of these disorders.
关节过度活动是多种遗传性结缔组织疾病的典型特征,如马方综合征和各种类型的埃勒斯-当洛综合征,在对出现此类症状的患者进行鉴别诊断时,遗传学家和儿科医生通常会首先考虑这些疾病。然而,一些先天性和成人起病的遗传性肌病在通常仅伴有轻至中度肌肉无力的情况下也会出现关节过度活动,因此也应纳入关节过度活动的鉴别诊断。事实上,在分子水平上,这两组疾病都代表了遗传性细胞外基质(ECM)疾病同一谱系的不同端点。在本综述中,我们将总结关节过度活动的测量方法,阐明这些疾病组共有的分子机制,随后讨论以下疾病的临床特征:1)具有肌病或其他神经肌肉特征的最常见结缔组织疾病:埃勒斯-当洛综合征、马方综合征和洛伊迪茨综合征;2)肌病和结缔组织重叠疾病(肌肉细胞外基质(ECM)疾病),包括与胶原蛋白VI相关的营养不良和与FKBP14相关的脊柱后侧凸型埃勒斯-当洛综合征;3)各种具有明显关节过度活动的(先天性)肌病,包括与RYR1和SEPN1相关的肌病。本综述的目的是帮助临床遗传学家和其他临床医生识别这些疾病。