摘要
家族性地中海热(FMF)是一种罕见的常染色体隐性遗传性自身炎症性疾病,包括先天性免疫并且几乎完全影响地中海起源的人口。临床表现包括发热、白细胞增多反复发作、浆膜炎(腹膜炎、胸膜炎、关节炎)、肌痛或丹毒样皮肤病变,持续12-72小时。MEFV基因突变在染色体16p13.3编码异常PYRIN(marenostrin),异常表达的蛋白质的粒细胞、单核细胞、浆膜、滑膜成纤维细胞和参与激活caspase-1和处理及炎性β释放IL-1。自1972第一次报告,三环中性生物碱与秋水仙碱维持治疗,保留了对FMF患者的所有症状的治疗效果,因为它减少了疾病的活动性和防止发展继发性淀粉样变性和肾损害。辅助治疗包括非甾类化合物的秋水仙碱抗炎药和糖皮质激素。在一个小组或不能耐受秋水仙碱的秋水仙碱耐药FMF病人,选择性的治疗必须考虑。不断发展的经验都集中在生物制剂的潜在效能作为TNF-α抑制剂(依那西普英夫利昔单抗),白介素IL-1(rilonacept),IL-1抑制剂(anakinra canakinumab)和IL-6受体抗体(tocilizumab)。干扰素-α和沙利度胺也可用于FMF病人。然而,临床试验常常主要是不受控制和限制的案例,因此需要明确的结论。在罕见的FMF病中,旧的和新的治疗方法都被进行了讨论。我们需要知道,任何理想的治疗必须经得起时间的考验。
关键词: 阿那白滞素,自身炎症性疾病,人抗IL-β单克隆抗体,秋水仙素,染色体16p,依那西普,白细胞介素1,炎性体,利洛纳塞,继发性淀粉样变性。
Current Medicinal Chemistry
Title:Colchicine, Biologic Agents and More for the Treatment of Familial Mediterranean Fever. The Old, the New, and the Rare
Volume: 23 Issue: 1
Author(s): P. Portincasa
Affiliation:
关键词: 阿那白滞素,自身炎症性疾病,人抗IL-β单克隆抗体,秋水仙素,染色体16p,依那西普,白细胞介素1,炎性体,利洛纳塞,继发性淀粉样变性。
摘要: Familial Mediterranean Fever (FMF) is a rare autosomal recessive autoinflammatory disorder involving the innate immunity and affecting almost exclusively populations with Mediterranean origin. Clinical features include recurrent episodes of fever, leukocitosis, serositis (peritonitis or pleuritis, arthritis), myalgia or erysipelas-like skin lesions, lasting 12-72 hrs. The MEFV gene mutations on chromosome 16p13.3 encodes the abnormal pyrin (marenostrin), a protein expressed in granulocytes, monocytes, serosal and synovial fibroblasts and involved in the activation of caspase-1 and the processing and release of active pro-inflammatory IL-1β. Since the first report in 1972, maintenance therapy with colchicine, a tricyclic neutral alkaloid, remains the mainstay of treatment in symptomatic FMF patients since it reduces the disease activity and prevents the development of secondary amyloidosis and renal damage. Adjunctive symptomatic therapy to colchicine includes nonsteroideal antinflammatory drugs and corticosteroids. In a small group of colchicine-intolerant or colchicine-resistant FMF patients, alternative treatments must be considered. Evolving experiences have focussed on the potential effectiveness of biologic agents working as TNF-α inhibitors (etanercept, infliximab), IL-1 trap (Rilonacept), IL-1 inhibitors (Anakinra, Canakinumab) and IL-6 receptor antibody (Tocilizumab). Interferon-α and thalidomide have also been employed in FMF patients. Still, clinical trials are mainly uncontrolled and restricted to few cases, thus requiring definitive conclusions. Old, and new treatments are discussed in the rare FMF disease, with the concept that any ideal treatment has to stand the test of time.
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P. Portincasa , Colchicine, Biologic Agents and More for the Treatment of Familial Mediterranean Fever. The Old, the New, and the Rare, Current Medicinal Chemistry 2016; 23 (1) . https://dx.doi.org/10.2174/0929867323666151117121706
DOI https://dx.doi.org/10.2174/0929867323666151117121706 |
Print ISSN 0929-8673 |
Publisher Name Bentham Science Publisher |
Online ISSN 1875-533X |
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