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结缔组织病相关间质性肺疾病患者临床试验的经验教训。

Learnings from clinical trials in patients with connective tissue disease-associated interstitial lung disease.

机构信息

Department of Internal Medicine, Section of Pulmonary, Critical Care and Sleep Medicine, Yale School of Medicine, New Haven, CT, 06520, USA.

Department of Internal Medicine, University of Connecticut School of Medicine, Farmington, CT, 06032, USA.

出版信息

Arthritis Res Ther. 2023 Jul 8;25(1):118. doi: 10.1186/s13075-023-03090-y.

DOI:10.1186/s13075-023-03090-y
PMID:37422652
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC10329300/
Abstract

Many clinical trial results are available to inform best practices in the treatment of patients with connective tissue disease-associated interstitial lung disease (CTD-ILD).Herein, we summarize the results of clinical trials, including patient-reported outcome instruments, for the treatment of patients with ILD associated with systemic sclerosis (SSc/scleroderma), rheumatoid arthritis, and idiopathic inflammatory myositis, the diseases with the most available data. For SSc-ILD, the US Food and Drug Administration approved nintedanib (a tyrosine kinase inhibitor) in 2020 and subcutaneous tocilizumab (an IL-6 receptor monoclonal antibody) in 2021. Rituximab was recently shown to have similar efficacy but better tolerability than intravenous cyclophosphamide (CYC) for CTD-ILD therapy. Scleroderma Lung Study II, conducted in patients with SSc-ILD, showed that oral CYC and mycophenolate mofetil (MMF) were comparable in their effects on lung function, but MMF was better tolerated. The increasing treatment armamentarium for patients with CTD-ILD offers physicians new opportunities to improve patient outcomes.

摘要

许多临床试验结果可用于为结缔组织病相关间质性肺疾病(CTD-ILD)患者的治疗提供最佳实践信息。在此,我们总结了与系统性硬化症(SSc/硬皮病)、类风湿关节炎和特发性炎性肌病相关的ILD 患者的临床试验结果,这些疾病的数据最为丰富,包括患者报告的结局指标。对于 SSc-ILD,美国食品和药物管理局于 2020 年批准尼达尼布(一种酪氨酸激酶抑制剂),2021 年批准皮下托珠单抗(一种 IL-6 受体单克隆抗体)。最近的研究表明,利妥昔单抗在治疗 CTD-ILD 方面的疗效与静脉注射环磷酰胺(CYC)相当,但耐受性更好。在 SSc-ILD 患者中进行的硬皮病肺研究 II 表明,口服 CYC 和霉酚酸酯(MMF)在肺功能方面的疗效相当,但 MMF 的耐受性更好。用于治疗 CTD-ILD 的治疗手段越来越多,为医生提供了改善患者预后的新机会。

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引用本文的文献

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Efficacy of rituximab versus cyclophosphamide in connective tissue disease‑related interstitial lung disease: a systematic review and meta-analysis.利妥昔单抗与环磷酰胺治疗结缔组织病相关间质性肺病的疗效:一项系统评价和荟萃分析。
Clin Rheumatol. 2025 Jun 13. doi: 10.1007/s10067-025-07533-6.
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Correction: Learnings from clinical trials in patients with connective tissue disease‑associated interstitial lung disease.更正:结缔组织病相关间质性肺病患者临床试验的经验教训。
Arthritis Res Ther. 2023 Jul 27;25(1):132. doi: 10.1186/s13075-023-03123-6.

本文引用的文献

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Safety and efficacy of rituximab in systemic sclerosis (DESIRES): a double-blind, investigator-initiated, randomised, placebo-controlled trial.利妥昔单抗治疗系统性硬化症的安全性和有效性(DESIRES):一项由研究者发起的双盲、随机、安慰剂对照试验。
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Breakthrough SARS-CoV-2 infections and prediction of moderate-to-severe outcomes during rituximab therapy in patients with rheumatic and musculoskeletal diseases in the UK: a single-centre cohort study.英国风湿和肌肉骨骼疾病患者在利妥昔单抗治疗期间的严重急性呼吸综合征冠状病毒2突破性感染及中重度结局预测:一项单中心队列研究
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Rituximab versus intravenous cyclophosphamide in patients with connective tissue disease-associated interstitial lung disease in the UK (RECITAL): a double-blind, double-dummy, randomised, controlled, phase 2b trial.利妥昔单抗与静脉注射环磷酰胺治疗英国结缔组织病相关间质性肺病患者(RECITAL):一项双盲、双模拟、随机、对照、2b 期试验。
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