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美国儿童家族性地中海热:密歇根州东南部队列中的临床表型谱和MEFV基因型

Childhood Familial Mediterranean Fever in the United States: Spectrum of Clinical Phenotypes and MEFV Genotypes in a Cohort From Southeast Michigan.

作者信息

Fathalla Basil M, Thomas Ronald

机构信息

From the Department of Allergy, Immunology, and Rheumatology, Children's Hospital of Michigan, Central Michigan University, Detroit, MI.

Department of Biostatistics, Children's Research Institute, Central Michigan University School of Medicine, Mount Pleasant, MI.

出版信息

J Clin Rheumatol. 2025 Apr 1;31(3):99-106. doi: 10.1097/RHU.0000000000002183. Epub 2024 Dec 3.

Abstract

OBJECTIVES

The aim of this study was to report the spectrum of Familial Mediterranean Fever (FMF) in children living in Southeast Michigan.

METHODS

We reviewed prerecorded data in medical records of FMF patients. Statistical analysis of the data included Fisher exact test, Pearson χ 2 procedure, parametric independent samples t test, and parametric analysis of variance using SPSS Version 29.0, IBM Inc.

RESULTS

The study included 29 males and 21 females. The mean age at presentation was 4.63 ± 3.66 years, and the mean time to diagnosis was 2.1 ± 2.18 years. A slight majority presented in the first 3 years of age (54%). Family history of FMF was reported in only 58% of patients. Clinical manifestations included fever (84%), gastrointestinal (84%), musculoskeletal (64%; including chronic arthritis, sacroiliitis, and nonbacterial osteomyelitis), chest (28%), cutaneous (14%), and other manifestations (16%). Fever without other manifestations was reported only in patients presenting at ≤3 years of age ( p = 0.016), whereas older patients reported more gastrointestinal manifestations ( p = 0.04). Reported MEFV variants included p.M694V (n = 26), p.V726A (n = 23), p.M694I (n = 13), and others (n = 10). Homozygote and compound heterozygote patients had more gastrointestinal manifestations ( p < 0.001), whereas fever was more common in the heterozygote patients ( p = 0.04). The mean follow-up period was 5.34 ± 4.13 years with no renal disease.

CONCLUSIONS

We report the largest childhood FMF cohort in the United States. A negative family history should not preclude consideration of FMF as a cause of periodic fever. Recurrent fever can be the only manifestation, particularly in young patients with FMF. The absence of fever and chronic progressive musculoskeletal manifestations can uncommonly occur.

摘要

目的

本研究旨在报告居住在密歇根州东南部儿童的家族性地中海热(FMF)情况。

方法

我们回顾了FMF患者病历中的预先记录数据。使用IBM公司的SPSS 29.0版本对数据进行统计分析,包括Fisher精确检验、Pearson卡方检验、参数独立样本t检验和参数方差分析。

结果

该研究纳入了29名男性和21名女性。就诊时的平均年龄为4.63±3.66岁,平均诊断时间为2.1±2.18年。略多数患者在3岁前就诊(54%)。仅58%的患者报告有FMF家族史。临床表现包括发热(84%)、胃肠道症状(84%)、肌肉骨骼症状(64%;包括慢性关节炎、骶髂关节炎和非细菌性骨髓炎)、胸部症状(28%)、皮肤症状(14%)和其他症状(16%)。仅≤3岁就诊的患者报告有无其他症状的发热(p = 0.016),而年龄较大的患者报告有更多胃肠道症状(p = 0.04)。报告的MEFV变异包括p.M694V(n = 26)、p.V726A(n = 23)、p.M694I(n = 13)和其他变异(n = 10)。纯合子和复合杂合子患者有更多胃肠道症状(p < 0.001),而异合子患者发热更常见(p = 0.04)。平均随访期为5.34±4.13年,无肾脏疾病。

结论

我们报告了美国最大的儿童FMF队列。家族史阴性不应排除将FMF视为周期性发热病因的考虑。反复发热可能是唯一表现,特别是在患有FMF的年轻患者中。无发热和慢性进行性肌肉骨骼症状的情况罕见。

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