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阿那白滞素治疗家族性地中海热患者:单中心经验。

Anakinra treatment in patients with familial Mediterranean fever: a single-centre experience.

机构信息

Division of Rheumatology, Department of Internal Medicine, Cerrahpasa Medical Faculty, Istanbul University-Cerrahpasa, Istanbul, Turkey.

出版信息

Rheumatology (Oxford). 2021 May 14;60(5):2327-2332. doi: 10.1093/rheumatology/keaa596.

Abstract

OBJECTIVES

Anakinra is proven to be effective in controlled trials in terms of attack frequency and subclinical inflammation in colchicine-resistant patients. The objective of this study was to review the patients followed in our single centre with FMF who received anakinra because of insufficient colchicine response.

METHODS

The study was conducted at a tertiary rheumatology centre experienced in autoinflammatory diseases. The patients were treated for at least 1 month with anakinra. Patients with amyloidosis and pregnancy were not included. Attack frequency, patient global assessment scales of disease severity and acute phase reactants were recorded before and throughout anakinra treatment. Criteria of treatment termination were side effects, disease remission, inadequate response, pregnancy plan and non-compliance.

RESULTS

One hundred and six patients diagnosed with FMF were treated with anakinra; 45.92% of the patients had a homozygous M694V mutation; 83 of the 98 patients tested for MEFV carried at least one copy of M694V. Attack frequency decreased while on anakinra treatment; in fact, no attacks were observed in 75 patients. Visual analogue scale score decreased from 7.49 (2.03) to 3.08 (2.82) (P = 0.001). Currently, 71 patients are still on anakinra treatment. Treatment of 34 patients was discontinued (32%). Insufficient response and side effects were the most common reasons for treatment discontinuation. All of the side effects observed were reversible and the patients alleviated after treatment cessation. In four patients, leukopenia was observed.

CONCLUSION

In patients who were refractory to colchicine, anti-IL-1 agent anakinra was shown to be effective and safe. The effectiveness of anakinra stems from preventing attacks and increasing the quality of life.

摘要

目的

阿那白滞素在临床试验中已被证明在秋水仙碱耐药患者的发作频率和亚临床炎症方面有效。本研究的目的是回顾在我们的单中心接受阿那白滞素治疗的 FMF 患者,这些患者因秋水仙碱反应不足。

方法

该研究在一家具有自身炎症性疾病经验的三级风湿病中心进行。患者至少接受了 1 个月的阿那白滞素治疗。不包括淀粉样变性和妊娠患者。在接受阿那白滞素治疗前后记录发作频率、患者疾病严重程度的整体评估量表和急性期反应物。治疗终止的标准为副作用、疾病缓解、反应不足、妊娠计划和不依从。

结果

106 名诊断为 FMF 的患者接受了阿那白滞素治疗;45.92%的患者存在纯合 M694V 突变;98 名接受 MEFV 检测的患者中有 83 名至少携带一份 M694V 拷贝。在接受阿那白滞素治疗期间,发作频率降低;事实上,75 名患者没有观察到发作。视觉模拟评分从 7.49(2.03)降至 3.08(2.82)(P=0.001)。目前,71 名患者仍在接受阿那白滞素治疗。有 34 名患者停止了治疗(32%)。反应不足和副作用是停止治疗最常见的原因。观察到的所有副作用都是可逆的,停药后患者症状缓解。在 4 名患者中观察到白细胞减少。

结论

在对秋水仙碱耐药的患者中,抗 IL-1 药物阿那白滞素被证明是有效和安全的。阿那白滞素的有效性源于预防发作和提高生活质量。

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