Kisla Ekinci Rabia Miray, Kilic Konte Elif, Akay Nergis, Gul Umit
Department of Pediatric Rheumatology, Adana City Training and Research Hospital, Adana, Türkiye.
Department of Pediatric Rheumatology, İstanbul University-Cerrahpaşa Cerrahpaşa Medical Faculty, İstanbul, Türkiye.
Turk Arch Pediatr. 2024 Nov 1;59(6):527-534. doi: 10.5152/TurkArchPediatr.2024.24188.
Familial Mediterranean fever (FMF) is the most prevalent monogenic autoinflammatory disorder, characterized by recurrent fever and serositis. It primarily affects individuals of Mediterranean descent, including Arabs, Armenians, Turks, and Jews. The Mediterranean fever (MEFV) gene, responsible for FMF, was discovered in 1997. Biallelic pathogenic variants lead to excessive activation of the pyrin inflammasome, resulting in inflammation. Clinical manifestations include recurrent fever, abdominal pain, and joint involvement, with attacks typically lasting 12-72 hours. Diagnosis relies on clinical criteria and is supported by genetic testing. Colchicine is the primary treatment to reduce attack frequency and prevent the complications like renal amyloidosis. Despite advancements in understanding FMF, including its genetic basis and treatment options, challenges remain in distinguishing it from other autoinflammatory diseases. Co-existing conditions such as juvenile idiopathic arthritis and inflammatory bowel disease are common among FMF patients. Ongoing research should aim to clarify the development of the disease, enhance diagnostic accuracy, and address its clinical presentation and genetic variability, with a focus on identifying new genetic mutations and epigenetic factors that contribute to its pathogenesis.
家族性地中海热(FMF)是最常见的单基因自身炎症性疾病,其特征为反复发热和浆膜炎。它主要影响地中海血统的个体,包括阿拉伯人、亚美尼亚人、土耳其人和犹太人。导致FMF的地中海热(MEFV)基因于1997年被发现。双等位基因致病性变异导致吡啉炎性小体过度激活,从而引发炎症。临床表现包括反复发热、腹痛和关节受累,发作通常持续12至72小时。诊断依赖临床标准,并通过基因检测得到支持。秋水仙碱是减少发作频率和预防肾淀粉样变性等并发症的主要治疗方法。尽管在理解FMF方面取得了进展,包括其遗传基础和治疗选择,但在将其与其他自身炎症性疾病区分开来方面仍存在挑战。幼年特发性关节炎和炎症性肠病等并存疾病在FMF患者中很常见。正在进行的研究应旨在阐明该疾病的发展过程,提高诊断准确性,并解决其临床表现和遗传变异性问题,重点是识别导致其发病机制的新基因突变和表观遗传因素。