Department of Pediatrics, Lausanne University Hospital and University of Lausanne, Lausanne, Switzerland.
Am J Med Genet C Semin Med Genet. 2012 Aug 15;160C(3):154-64. doi: 10.1002/ajmg.c.31331. Epub 2012 Jul 12.
The so-called "enchondromatoses" are skeletal disorders defined by the presence of ectopic cartilaginous tissue within bone tissue. The clinical and radiographic features of the different enchondromatoses are distinct, and grouping them does not reflect a common pathogenesis but simply a similar radiographic appearance and thus the need for a differential diagnosis. Recent advances in the understanding of their molecular and cellular bases confirm the heterogeneous nature of the different enchondromatoses. Some, like Ollier disease, Maffucci disease, metaphyseal chondromatosis with hydroxyglutaric aciduria, and metachondromatosis are produced by a dysregulation of chondrocyte proliferation, while others (such as spondyloenchondrodysplasia or dysspondyloenchondromatosis) are caused by defects in structure or metabolism of cartilage or bone matrix. In other forms (e.g., the dominantly inherited genochondromatoses), the basic defect remains to be determined. The classification, proposed by Spranger and associates in 1978 and tentatively revised twice, was based on the radiographic appearance, the anatomic sites involved, and the mode of inheritance. The new classification proposed here integrates the molecular genetic advances and delineates phenotypic families based on the molecular defects. Reference radiographs are provided to help in the diagnosis of the well-defined forms. In spite of advances, many cases remain difficult to diagnose and classify, implying that more variants remain to be defined at both the clinical and molecular levels.
所谓“软骨瘤病”是指骨骼疾病,其特征是骨组织内存在异位软骨组织。不同软骨瘤病的临床和影像学特征明显不同,将它们分组并不是反映共同的发病机制,而只是反映了相似的影像学表现,因此需要进行鉴别诊断。对其分子和细胞基础的理解的最新进展证实了不同软骨瘤病的异质性。一些疾病,如 Ollier 病、Maffucci 病、羟戊二酸尿症性骺软骨病和软骨发育不良,是由软骨细胞增殖失调引起的,而另一些疾病(如脊椎骨骺发育不良或骨软骨发育不良)是由软骨或骨基质的结构或代谢缺陷引起的。在其他形式中(例如,显性遗传性软骨瘤病),基本缺陷仍有待确定。由 Spranger 等人于 1978 年提出并两次暂定修订的分类是基于放射影像学表现、受累的解剖部位和遗传方式。这里提出的新分类整合了分子遗传学进展,并根据分子缺陷描绘了表型家族。提供了参考射线照片以帮助诊断明确的形式。尽管取得了进展,但许多病例仍然难以诊断和分类,这意味着在临床和分子水平上仍有更多的变体有待定义。