Department of Pathology and Laboratory Medicine, UCLA Medical Center, Los Angeles, California 90049, USA.
Genet Med. 2011 Mar;13(3):263-9. doi: 10.1097/GIM.0b013e31820e27b1.
Familial Mediterranean fever is an autosomal recessive genetic disorder characterized by recurrent febrile polyserositis, especially prevalent in individuals of Mediterranean descent. Familial Mediterranean fever can have nonspecific manifestations that mimic many common acquired disorders such as infections, acute appendicitis, cholecystitis, and arthritis, which can delay diagnosis for many years and subject patients to extensive evaluations and even unnecessary surgery. Untreated familial Mediterranean fever can result in serious complications such as end-stage renal disease and malabsorption secondary to amyloid deposition in the kidneys and digestive tract, male and female infertility, and growth retardation in children. These significant sequelae, along with the episodic acute attacks, are readily preventable by treatment with oral colchicine and underscore the necessity of early detection and treatment from a medical, psychosocial, and economic standpoint. We describe our comprehensive approach to the accurate diagnosis and effective management of this disorder by means of a dedicated familial Mediterranean fever clinic that incorporates medical genetics on equal footing with general medicine. In addition to providing the clinician with the presenting features of familial Mediterranean fever, methods of diagnosis including molecular testing, and current management based on our extensive experience with hundreds of affected individuals, we also advance this approach as a model for the incorporation of medical genetics practice into the more traditional domains of general medicine.
家族性地中海热是一种常染色体隐性遗传疾病,其特征为反复发作的发热性多浆膜炎,尤其常见于地中海血统的人群。家族性地中海热可能具有非特异性表现,类似于许多常见的后天性疾病,如感染、急性阑尾炎、胆囊炎和关节炎,这可能导致多年的误诊,并使患者接受广泛的评估,甚至进行不必要的手术。未经治疗的家族性地中海热可能导致严重的并发症,如肾脏和消化道淀粉样物质沉积引起的终末期肾病和吸收不良、男性和女性不孕以及儿童生长迟缓。这些严重的后果,加上间歇性的急性发作,通过口服秋水仙碱治疗是可以预防的,这从医学、心理社会和经济角度强调了早期发现和治疗的必要性。我们通过专门的家族性地中海热诊所,将医学遗传学与普通医学平等结合,描述了我们对这种疾病进行准确诊断和有效管理的综合方法。除了为临床医生提供家族性地中海热的表现特征、包括分子检测在内的诊断方法以及基于我们对数百名受影响个体的丰富经验的当前管理方法外,我们还将这种方法作为将医学遗传学实践纳入更传统的普通医学领域的模型进行推广。