Glorieux Francis H, Ward Leanne M, Rauch Frank, Lalic Ljiljana, Roughley Peter J, Travers Rose
Shriners Hospital for Children, and Department of Surgery, McGill University, Montreal, Quebec, Canada.
J Bone Miner Res. 2002 Jan;17(1):30-8. doi: 10.1359/jbmr.2002.17.1.30.
Osteogenesis imperfecta (OI) is a heritable disease of bone in which the hallmark is bone fragility. Usually, the disorder is divided into four groups on clinical grounds. We previously described a group of patients initially classified with OI type IV who had a discrete phenotype including hyperplastic callus formation without evidence of mutations in type I collagen. We called that disease entity OI type V. In this study, we describe another group of 8 patients initially diagnosed with OI type IV who share unique, common characteristics. We propose to name this disorder "OI type VI." Fractures were first documented between 4 and 18 months of age. Patients with OI type VI sustained more frequent fractures than patients with OI type IV. Sclerae were white or faintly blue and dentinogenesis imperfecta was uniformly absent. All patients had vertebral compression fractures. No patients showed radiological signs of rickets. Lumbar spine areal bone mineral density (aBMD) was low and similar to age-matched patients with OI type IV. Serum alkaline phosphatase levels were elevated compared with age-matched patients with type IV OI (409 +/- 145 U/liter vs. 295 +/- 95 U/liter; p < 0.03 by t-test). Other biochemical parameters of bone and mineral metabolism were within the reference range. Mutation screening of the coding regions and exon/intron boundaries of both collagen type I genes did not reveal any mutations, and type I collagen protein analyses were normal. Qualitative histology of iliac crest bone biopsy specimens showed an absence of the birefringent pattern of normal lamellar bone under polarized light, often with a "fish-scale" pattern. Quantitative histomorphometry revealed thin cortices, hyperosteoidosis, and a prolonged mineralization lag time in the presence of a decreased mineral apposition rate. We conclude that type VI OI is a moderate to severe form of brittle bone disease with accumulation of osteoid due to a mineralization defect, in the absence of a disturbance of mineral metabolism. The underlying genetic defect remains to be elucidated.
成骨不全症(OI)是一种遗传性骨病,其特征是骨脆性增加。通常,根据临床情况将该疾病分为四组。我们之前描述过一组最初被归类为IV型OI的患者,他们具有独特的表型,包括增生性骨痂形成,且I型胶原蛋白无突变证据。我们将该疾病实体称为V型OI。在本研究中,我们描述了另一组8例最初被诊断为IV型OI的患者,他们具有独特的共同特征。我们建议将这种疾病命名为“VI型OI”。骨折首次记录于4至18个月龄之间。VI型OI患者比IV型OI患者发生骨折的频率更高。巩膜呈白色或浅蓝色,均未出现牙本质生成不全。所有患者均有椎体压缩性骨折。无患者表现出佝偻病的放射学征象。腰椎区域骨矿物质密度(aBMD)较低,与年龄匹配的IV型OI患者相似。与年龄匹配的IV型OI患者相比,血清碱性磷酸酶水平升高(409±145 U/升对295±95 U/升;经t检验,p<0.03)。骨和矿物质代谢的其他生化参数在参考范围内。对两个I型胶原蛋白基因的编码区和外显子/内含子边界进行突变筛查未发现任何突变,I型胶原蛋白蛋白分析正常。髂嵴骨活检标本的定性组织学显示,在偏振光下正常板层骨的双折射模式缺失,常呈“鱼鳞”状。定量组织形态计量学显示皮质变薄、类骨质增多,且在矿化沉积率降低的情况下矿化延迟时间延长。我们得出结论,VI型OI是一种中度至重度的脆性骨病,由于矿化缺陷导致类骨质堆积,且不存在矿物质代谢紊乱。潜在的基因缺陷仍有待阐明。