Systemic Sclerosis-Associated Interstitial Lung Disease: How to Incorporate Two Food and Drug Administration-Approved Therapies in Clinical Practice.
Systemic Sclerosis-Associated Interstitial Lung Disease: How to Incorporate Two Food and Drug Administration-Approved Therapies in Clinical Practice.
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DOI：
10.1002/art.41933
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发表时间：
2022-01
期刊：
影响因子：
--
通讯作者：
Denton CP
中科院分区：
文献类型：
--
作者：
Khanna D;Lescoat A;Roofeh D;Bernstein EJ;Kazerooni EA;Roth MD;Martinez F;Flaherty KR;Denton CP
在所有风湿性疾病中，系统性硬化症（SSc；硬皮病）的个人死亡率最高，而间质性肺疾病（ILD）是导致SSc相关死亡的主要原因之一。两种药物现已获得美国食品和药物管理局（FDA）批准，用于减缓SSc- ild患者肺功能下降的速度：尼达尼布（一种酪氨酸激酶抑制剂）和托珠单抗（第一种靶向SSc中白细胞介素-6途径的生物制剂）。此外，两种具有细胞毒性和免疫调节活性的仿制药霉酚酸酯和环磷酰胺在II期试验中显示出相当的疗效，但尚未获得fda批准用于SSc-ILD。鉴于该疾病的异质性，SSc-ILD患者的最佳治疗策略仍有待确定。本综述的目的有两个：(1)综述集中在SSc-ILD诊断和治疗方面的研究；(2)提出临床诊断、分层、管理和治疗决策的实用方法。本综述根据疾病严重程度（亚临床与临床ILD）和相关进展风险（低风险与高风险）对SSc患者进行了实用分类。鉴于tocilizumab最近被批准用于SSc-ILD，我们讨论了作为一线和二线治疗的药物和非药物选择，以及潜在的联合治疗方法。
Systemic sclerosis (SSc; scleroderma) has the highest individual mortality of all rheumatic diseases and interstitial lung disease (ILD) is among the leading causes of SSc-related death. Two drugs are now approved by the Food & Drug Administration (FDA) and indicated for slowing the rate of decline in pulmonary function in patients with SSc-ILD: nintedanib (a tyrosine kinase inhibitor) and tocilizumab (the first biologic agent targeting the interleukin-6 pathway in SSc). In addition, two generic drugs with cytotoxic and immunoregulatory activity, mycophenolate mofetil and cyclophosphamide, have shown comparable efficacy in a Phase II trial but are not FDA-approved for SSc-ILD. In light of the heterogeneity of the disease, the optimal therapeutic strategy in the management of patients with SSc-ILD is still to be determined. The objectives of this review are two-fold: (1) review the body of research focused on the diagnosis and treatment of SSc-ILD; and (2) propose a practical approach for diagnosis, stratification, management, and therapeutic decision-making in this clinical context. This review presents a practical classification of SSc patients in terms of disease severity (subclinical vs. clinical ILD) and associated risk of progression (low vs. high risk). The pharmacological and non-pharmacological options as first and second-line therapy, as well as potential combination approaches, are discussed in light of the recent approval of tocilizumab for SSc-ILD.
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DOI：
10.1016/j.xcrm.2020.100140
发表时间：
2020-11-17
期刊：
Cell reports. Medicine
影响因子：
--
作者：
Gao X;Jia G;Guttman A;DePianto DJ;Morshead KB;Sun KH;Ramamoorthi N;Vander Heiden JA;Modrusan Z;Wolters PJ;Jahreis A;Arron JR;Khanna D;Ramalingam TR
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Ramalingam TR
影响因子：
158.5
作者：
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影响因子：
7.3
作者：
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通讯作者：
Andrade LE
影响因子：
5.5
作者：
Dougherty, Dane H.;Kwakkenbos, Linda;Mayes, Maureen D.
通讯作者：
Mayes, Maureen D.
DOI：
10.1164/rccm.2106012
发表时间：
2002-06-15
影响因子：
24.7
作者：
Bouros, D;Wells, AU;du Bois, RM
通讯作者：
du Bois, RM