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秋水仙碱治疗可在选择的一组儿科 FMF 患者中停药。

Colchicine treatment can be discontinued in a selected group of pediatric FMF patients.

机构信息

Sackler Faculty of Medicine, Tel-Aviv University, Tel-Aviv, Israel.

Paediatric Rheumatology unit, Edmond and Lily Safra Children's Hospital, Sheba Medical Centrer, 52621, Ramat-Gan, Tel Hashomer, Israel.

出版信息

Pediatr Rheumatol Online J. 2023 Jan 4;21(1):2. doi: 10.1186/s12969-022-00780-w.

Abstract

OBJECTIVES

Familial Mediterranean Fever (FMF) patients are required to adhere to a life-long treatment with colchicine, primarily for preventing amyloidosis. As some patients may be asymptomatic for long periods of time, it remains unclear whether it is possible to discontinue colchicine treatment in a selective group of patients. We aimed to identify predictive characteristics for a successful cessation of colchicine therapy.

METHODS

Out of 646 FMF pediatric patients followed in our referral FMF clinic, colchicine treatment was discontinued in 51 patients. In this study we compared the genetic, demographic, and clinical characteristics between patients for whom a successful cessation of therapy was made (Group 1; n = 21) and patients for whom cessation of therapy was deemed a failure (Group 2; n = 30) and consequently had to resume colchicine therapy.

RESULTS

Patients for whom a successful cessation of therapy was achieved had no biallelic pathogenic MEFV mutations, were less likely to have "severe attacks" (two or more FMF characteristic symptoms) (24% vs 80%; P = 0.000067) and did not require higher than 1 mg/day of colchicine, prior to the drug cessation. Remission duration under colchicine treatment was significantly higher in group 1 compared with group 2 (4.36 years ±2.12 vs 2.53 years ±2; P = 0.0036).

CONCLUSION

This study supports the concept of colchicine free remission in a minority of FMF patients (3%). Holding treatment, under close monitoring, may be reasonable when selecting the appropriate patients.

摘要

目的

家族性地中海热 (FMF) 患者需要终身接受秋水仙碱治疗,主要是为了预防淀粉样变性。由于一些患者可能长时间无症状,因此尚不清楚是否可以在选择性患者群体中停止秋水仙碱治疗。我们旨在确定成功停止秋水仙碱治疗的预测特征。

方法

在我们的转诊 FMF 诊所中,对 646 名 FMF 儿科患者进行了随访,其中 51 名患者停止了秋水仙碱治疗。在这项研究中,我们比较了成功停止治疗的患者(第 1 组;n=21)和停止治疗被认为失败的患者(第 2 组;n=30)之间的遗传、人口统计学和临床特征,并且随后不得不恢复秋水仙碱治疗。

结果

成功停止治疗的患者没有双等位基因致病性 MEFV 突变,不太可能发生“严重发作”(两种或多种 FMF 特征症状)(24%比 80%;P=0.000067),并且在停药前不需要高于 1 毫克/天的秋水仙碱。与第 2 组相比,第 1 组在秋水仙碱治疗下的缓解持续时间明显更长(4.36 年±2.12 比 2.53 年±2;P=0.0036)。

结论

这项研究支持在少数 FMF 患者(3%)中无秋水仙碱缓解的概念。在选择合适的患者时,密切监测下的维持治疗可能是合理的。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/9034/9811732/056b37aecff5/12969_2022_780_Fig1_HTML.jpg

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Familial Mediterranean fever in childhood: a single-center experience.儿童期家族性地中海热:单中心经验。
Rheumatol Int. 2018 Jan;38(1):67-74. doi: 10.1007/s00296-017-3796-0. Epub 2017 Aug 21.

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