Clybouw C, Desmyttere S, Bonduelle M, Piepsz A
Department of Pediatrics, Vrije Universiteit Brussel, Belgium.
Genet Couns. 1994;5(2):195-8.
Progressive diaphyseal dysplasia (Camurati-Engelmann disease) is a rare hereditary disorder of bone characterized by progressive, bilaterally symmetrical diaphyseal sclerosis of the long bones. Severely affected patients show muscle weakness, waddling gait and severe pain in the extremities. However, clinical and radiological investigations in families with Camurati-Engelmann disease demonstrate a wide variability in expression of the manifestations. Asymptomatic patients were in several instances diagnosed only after radiologic screening of relatives. Although considered an autosomal dominant disorder, families are described where neither clinical nor radiological manifestations can be demonstrated in direct ancestors of patients. Combining roentgenographic examination with bone scintigraphy seems therefore necessary in confirming or ruling out progressive diaphyseal dysplasia in each family member.
进行性骨干发育异常(卡穆拉蒂 - 恩格尔曼病)是一种罕见的遗传性骨病,其特征为长骨进行性、双侧对称性骨干硬化。严重受累患者表现出肌肉无力、蹒跚步态和四肢剧痛。然而,对卡穆拉蒂 - 恩格尔曼病家族的临床和放射学调查显示,其临床表现的表达存在很大差异。在多个病例中,无症状患者仅在对亲属进行放射学筛查后才被诊断出来。尽管该病被认为是常染色体显性疾病,但也有一些家族的患者直系祖先中既无临床症状也无放射学表现。因此,对每个家庭成员进行X线检查与骨闪烁显像相结合,对于确诊或排除进行性骨干发育异常似乎是必要的。