阿那白那治疗儿童风湿病的疗效:我们的单中心经验。

IF 1.1 4区 医学 Q4 Medicine
Ferhat Demir, Eda Gürler, Betül Sözeri
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引用次数: 1

摘要

目的:介绍重组白细胞介素-1 (IL-1)受体拮抗剂anakinra治疗小儿风湿病的临床经验及疗效。患者及方法:2016年7月1日至2020年7月1日,共33例儿科患者(男18例,女15例;平均年龄:6±3.4岁;回顾性分析4至13岁的儿童风湿病患者使用阿那白胺治疗的情况。治疗期1个月以上且随访1年以上的患者纳入。在基线、治疗3个月和12个月时收集人口统计学和临床结果、结局、不良事件、既往和/或额外治疗。结果:研究组共有33例小儿风湿病患者,其中系统性幼年特发性关节炎[sJIA]合并巨噬细胞活化综合征[MAS] 11例,高免疫球蛋白- d综合征6例,低温蛋白相关周期综合征5例,家族性地中海热5例,特发性复发性心包炎4例,nlrp12相关周期热综合征1例,未分类的全身性自身炎症性疾病1例。治疗3个月后,69.7%的患者完全缓解,24.2%的患者部分缓解,6.1%的患者无缓解。一年后,45.5%的药物缓解患者和18.2%的药物缓解患者达到非活动性疾病状态。51.5%的患者(n=17)将Anakinra转为其他生物治疗。在这些患者中,分别有70.6%和29.4%的患者生物切换到canakinumab和tocilizumab。除局部反应(n=2)外,所有患者均未发生不良反应。结论:Anakinra似乎是一种很有前途的治疗选择,因为它在自身炎症条件下的半衰期短,效果迅速。虽然短期治疗对于合并MAS的sJIA似乎是足够的,但系统性自身炎症性疾病的患者维持一个更依赖阿那金的过程。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Efficacy of anakinra treatment in pediatric rheumatic diseases: Our single-center experience.

Objectives: This study aims to present our experience on anakinra, a recombinant interleukin-1 (IL-1) receptor antagonist, and efficacy results in pediatric rheumatic diseases in our clinic.

Patients and methods: Between July 1st, 2016 and July 1st, 2020, a total of 33 pediatric patients (18 males, 15 females; mean age: 6±3.4 years; range 4 to 13 years) with pediatric rheumatic diseases who were treated with anakinra were retrospectively analyzed. The patients with over one-month treatment period and followed for at least one year were included. Demographic and clinical findings, outcomes, adverse events, prior and/or additional treatments were collected at baseline, at 3 and 12 months of therapy.

Results: There were 33 patients with different pediatric rheumatic diseases (11 with systemic juvenile idiopathic arthritis [sJIA] complicated by macrophage activation syndrome [MAS], six with hyperimmunoglobulin-D syndrome, five with cryopyrin-associated periodic syndrome, five with familial Mediterranean fever, four with idiopathic recurrent pericarditis, one with NLRP12-associated periodic fever syndrome and one with unclassified systemic autoinflammatory disease), in the study group. The complete response was observed 69.7% of patients, partial response in 24.2%, and no response in 6.1% at three months of treatment. Inactive disease status was achieved in 45.5% of the patients with remission-on medication and 18.2% of the patients with remission-off medication at the end of a year. Anakinra was switched to other biological treatments in 51.5% of patients (n=17). Biological switch to canakinumab and tocilizumab were observed in 70.6% and 29.4% of these patients. Except for local reactions (n=2), no adverse events were observed in any of the patients.

Conclusion: Anakinra appears to be a promising treatment alternative owing to its rapid effect as a result of its short half-life in autoinflammatory conditions. While short-term therapy seems to be sufficient for the sJIA complicated by MAS, the patients with systemic autoinflammatory diseases maintenance a more anakinra-dependent course.

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来源期刊
Archives of rheumatology
Archives of rheumatology Medicine-Rheumatology
CiteScore
2.00
自引率
9.10%
发文量
15
期刊介绍: The Archives of Rheumatology is an official journal of the Turkish League Against Rheumatism (TLAR) and is published quarterly in March, June, September, and December. It publishes original work on all aspects of rheumatology and disorders of the musculoskeletal system. The priority of the Archives of Rheumatology is to publish high-quality original research articles, especially in inflammatory rheumatic disorders. In addition to research articles, brief reports, reviews, editorials, letters to the editor can also be published. It is an independent peer-reviewed international journal printed in English. Manuscripts are refereed by a "double-blind peer-reviewed" process for both referees and authors. Editorial Board of the Archives of Rheumatology works under the principles of The World Association of Medical Editors (WAME), the International Council of Medical Journal Editors (ICMJE), and Committee on Publication Ethics (COPE).
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