Sara Bindoli, Arianna De Matteis, Stéphane Mitrovic, Bruno Fautrel, Loreto Carmona, Fabrizio De Benedetti
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The intervention was any pharmacological treatment (approved or under evaluation) versus any comparator drug or placebo, and as outcomes, any relevant efficacy and safety event. The risk of bias (RoB) was assessed with the Cochrane RoB and AMSTAR-2 (Assessing the Methodological Quality of Systematic Reviews-2, version 2) for SRs.</p><p><strong>Results: </strong>128 full texts were included: 25 RCTs, 1 CCT, 11 SRs published after 2013 and 91 LOP/LOR studies. In Still's disease, interleukin (IL)-1 inhibitors (IL-1i) and IL-6R inhibitors (IL-6i) were the most studied drugs. Two meta-analyses on RCTs showed an OR, to achieve an ARC50 response rate, of 6.02 (95% CI 2.24 to 21.36) and 8.08 (95% CI 1.89 to 34.57) for IL-1i and IL-6Ri, respectively. Retrospective studies showed that early initiation of IL-1i or IL-6i was associated with high rates of clinically inactive disease. In MAS, GCs were employed in all patients, often associated with ciclosporin and/or anakinra. Rates of complete response were reported, with a range from 53% to 100%. Emapalumab was the only drug tested in a CCT, with a complete response of 93%.</p><p><strong>Conclusion: </strong>IL-1i and IL-6Ri show the highest level of efficacy in the treatment of Still's disease. For MAS, IL-1 and interferon-γ inhibition appear to be effective on a background of high-dose glucocorticoids.</p>","PeriodicalId":8087,"journal":{"name":"Annals of the Rheumatic Diseases","volume":" ","pages":"1731-1747"},"PeriodicalIF":20.3000,"publicationDate":"2024-11-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"Efficacy and safety of therapies for Still's disease and macrophage activation syndrome (MAS): a systematic review informing the EULAR/PReS guidelines for the management of Still's disease.\",\"authors\":\"Sara Bindoli, Arianna De Matteis, Stéphane Mitrovic, Bruno Fautrel, Loreto Carmona, Fabrizio De Benedetti\",\"doi\":\"10.1136/ard-2024-225854\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><strong>Objectives: </strong>To analyse the efficacy and safety of treatments for Still's disease and macrophage activation syndrome (MAS).</p><p><strong>Methods: </strong>Medline, Embase and Cochrane Library were searched for clinical trials (randomised, randomised controlled trial (RCT), controlled and clinical controlled trial (CCT)), observational studies (retrospective, longitudinal observational retrospective (LOR), prospective and longitudinal observational prospective (LOP)) and systematic reviews (SRs), in which the populations studied were patients with Still's disease and MAS. The intervention was any pharmacological treatment (approved or under evaluation) versus any comparator drug or placebo, and as outcomes, any relevant efficacy and safety event. The risk of bias (RoB) was assessed with the Cochrane RoB and AMSTAR-2 (Assessing the Methodological Quality of Systematic Reviews-2, version 2) for SRs.</p><p><strong>Results: </strong>128 full texts were included: 25 RCTs, 1 CCT, 11 SRs published after 2013 and 91 LOP/LOR studies. In Still's disease, interleukin (IL)-1 inhibitors (IL-1i) and IL-6R inhibitors (IL-6i) were the most studied drugs. Two meta-analyses on RCTs showed an OR, to achieve an ARC50 response rate, of 6.02 (95% CI 2.24 to 21.36) and 8.08 (95% CI 1.89 to 34.57) for IL-1i and IL-6Ri, respectively. Retrospective studies showed that early initiation of IL-1i or IL-6i was associated with high rates of clinically inactive disease. In MAS, GCs were employed in all patients, often associated with ciclosporin and/or anakinra. 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引用次数: 0
摘要
目的：分析治疗斯蒂尔病和巨噬细胞活化综合征（MAS）的疗效和安全性：分析治疗斯蒂尔病和巨噬细胞活化综合征（MAS）的有效性和安全性：在 Medline、Embase 和 Cochrane 图书馆检索了临床试验（随机试验、随机对照试验 (RCT)、对照试验和临床对照试验 (CCT)）、观察性研究（回顾性研究、纵向观察性回顾性研究 (LOR)、前瞻性研究和纵向观察性前瞻性研究 (LOP)）和系统综述 (SR)，研究人群为斯蒂尔病和巨噬细胞活化综合征患者。干预措施为任何药物治疗（已获批准或正在评估中）与任何对比药物或安慰剂的比较，结果为任何相关的疗效和安全性事件。偏倚风险（RoB）采用 Cochrane RoB 和 AMSTAR-2（Assessing the Methodological Quality of Systematic Reviews-2, version 2）进行评估：结果：共纳入 128 篇全文：结果：共纳入 128 篇全文：25 篇 RCT、1 篇 CCT、11 篇 2013 年后发表的 SR 和 91 篇 LOP/LOR 研究。在斯蒂尔病中，白细胞介素（IL）-1抑制剂（IL-1i）和IL-6R抑制剂（IL-6i）是研究最多的药物。两项 RCT 的荟萃分析表明，IL-1i 和 IL-6Ri 达到 ARC50 反应率的 OR 分别为 6.02（95% CI 2.24 至 21.36）和 8.08（95% CI 1.89 至 34.57）。回顾性研究显示，早期开始使用IL-1i或IL-6i与临床非活动性疾病的高发病率有关。在 MAS 中，所有患者都使用了 GCs，通常与环孢素和/或 Anakinra 联用。完全应答率从53%到100%不等。Emapalumab是唯一一种在CCT中进行测试的药物，完全应答率为93%：结论：IL-1i 和 IL-6Ri 在治疗斯蒂尔病方面显示出最高水平的疗效。对于 MAS，在大剂量糖皮质激素的背景下，抑制 IL-1 和干扰素-γ 似乎是有效的。
Efficacy and safety of therapies for Still's disease and macrophage activation syndrome (MAS): a systematic review informing the EULAR/PReS guidelines for the management of Still's disease.
Objectives: To analyse the efficacy and safety of treatments for Still's disease and macrophage activation syndrome (MAS).
Methods: Medline, Embase and Cochrane Library were searched for clinical trials (randomised, randomised controlled trial (RCT), controlled and clinical controlled trial (CCT)), observational studies (retrospective, longitudinal observational retrospective (LOR), prospective and longitudinal observational prospective (LOP)) and systematic reviews (SRs), in which the populations studied were patients with Still's disease and MAS. The intervention was any pharmacological treatment (approved or under evaluation) versus any comparator drug or placebo, and as outcomes, any relevant efficacy and safety event. The risk of bias (RoB) was assessed with the Cochrane RoB and AMSTAR-2 (Assessing the Methodological Quality of Systematic Reviews-2, version 2) for SRs.
Results: 128 full texts were included: 25 RCTs, 1 CCT, 11 SRs published after 2013 and 91 LOP/LOR studies. In Still's disease, interleukin (IL)-1 inhibitors (IL-1i) and IL-6R inhibitors (IL-6i) were the most studied drugs. Two meta-analyses on RCTs showed an OR, to achieve an ARC50 response rate, of 6.02 (95% CI 2.24 to 21.36) and 8.08 (95% CI 1.89 to 34.57) for IL-1i and IL-6Ri, respectively. Retrospective studies showed that early initiation of IL-1i or IL-6i was associated with high rates of clinically inactive disease. In MAS, GCs were employed in all patients, often associated with ciclosporin and/or anakinra. Rates of complete response were reported, with a range from 53% to 100%. Emapalumab was the only drug tested in a CCT, with a complete response of 93%.
Conclusion: IL-1i and IL-6Ri show the highest level of efficacy in the treatment of Still's disease. For MAS, IL-1 and interferon-γ inhibition appear to be effective on a background of high-dose glucocorticoids.
期刊介绍：
Annals of the Rheumatic Diseases (ARD) is an international peer-reviewed journal covering all aspects of rheumatology, which includes the full spectrum of musculoskeletal conditions, arthritic disease, and connective tissue disorders. ARD publishes basic, clinical, and translational scientific research, including the most important recommendations for the management of various conditions.
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