Mundra Vishal, Taxel Pamela
Department of Internal Medicine, Cleveland Clinic, Weston, Florida 33331, USA.
Conn Med. 2012 Jan;76(1):33-7.
Camurati-Engelmann disease or progressive diaphyseal dysplasia is a rare autosomal dominant disease characterized by cortical thickening of the long-bone diaphyses accompanied by waddling gait, muscular weakness, hearing loss and chronic skeletal pain. We describe two cases of Camurati-Engelmann disease with differing presentations and review of the literature on several therapeutic options.
We present two cases of Camurati-Engelmann disease which responded to different medical therapies. Various diagnostic tests including radiographs, bone scan and genetic analysis are also described.
These cases responded differently to the treatment options and provide an insight into the variable response of the disease.
Diagnosis of this disorder is based on the clinical history, family history, clinical examination and imaging results. Recently genetic testing has become available for TGF-beta1 mutation Several therapeutic agents including biphosphonates, NSAIDs, prednisone and losartan have been described as therapeutic options with mixed results, as described in our cases.
卡穆拉蒂 - 恩格尔曼病或进行性骨干发育异常是一种罕见的常染色体显性疾病,其特征为长骨干皮质增厚,伴有摇摆步态、肌肉无力、听力丧失和慢性骨骼疼痛。我们描述了两例表现不同的卡穆拉蒂 - 恩格尔曼病病例,并回顾了有关几种治疗选择的文献。
我们展示了两例对不同药物治疗有反应的卡穆拉蒂 - 恩格尔曼病病例。还描述了各种诊断测试,包括X线片、骨扫描和基因分析。
这些病例对治疗选择的反应不同,为该疾病的可变反应提供了见解。
该疾病的诊断基于临床病史、家族史、临床检查和影像学结果。最近,已可进行转化生长因子β1(TGF-beta1)突变的基因检测。如我们的病例中所述,包括双膦酸盐、非甾体抗炎药、泼尼松和氯沙坦在内的几种治疗药物已被描述为治疗选择,但结果不一。