An update on targeted therapies in systemic sclerosis based on a systematic review from the last 3 years.
An update on targeted therapies in systemic sclerosis based on a systematic review from the last 3 years.
复制标题
DOI：
10.1186/s13075-021-02536-5
复制
发表时间：
2021-06-01
影响因子：
4.9
通讯作者：
Allanore Y
中科院分区：
文献类型：
--
作者：
Campochiaro C;Allanore Y
最近出现了可以用特异性药物靶向治疗系统性硬化症（SSc）患者的新分子机制。在过去的3年中，一项大型3期试验的完成导致药物机构批准了第一种许可用于SSC相关间质性肺病的药物。鉴于SSc领域的这一激动人心的时刻，我们的目标是对关于SSc靶向治疗的I期、II期和III期临床试验以及大型观察性研究进行系统性文献综述。我们在MEDLINE/PubMed、EMBASE和ClinicalTrials.gov中检索了2016年以来的临床研究，这些研究将靶向治疗作为SSc患者皮肤或肺部受累的主要治疗，作为主要临床结局指标。审查了研究特征、使用的试验药物、试验药物作用的分子靶点、研究入选标准、治疗剂量、伴随免疫抑制的可能性、研究终点、研究持续时间和获得的结果的详细信息。在识别的973篇参考文献中，21篇（4篇会议摘要和17篇文章）被纳入系统综述。共分析了15项I/II期临床试验、2项III期临床试验和2项观察性研究。I期/II期研究中研究的药物包括：inebilizumab、达比加群、C-82、泊马度胺、利洛那塞、romilkimab、托珠单抗、托法替尼、吡非尼酮、来那巴、阿巴西普、贝利木单抗、利奥西呱、SAR 100842和拉尼非布诺。除3项研究外，所有研究均在具有不同入选标准的早期弥漫性SSc患者中进行，而3项研究在伴有间质性肺疾病（ILD）的SSc患者中进行。III期临床试验研究了尼达尼布和托珠单抗。在SSc-ILD患者中研究了Neptin，而托珠单抗则侧重于具有炎症特征的早期弥漫性SSc患者。还评估了两项观察性研究，包括> 50例使用利妥昔单抗作为靶向药物的患者。所有这些研究为SSc患者提供了真实的希望。未来的挑战将是定制患者特定的治疗方法，目标是为SSc开发精确的药物。
New molecular mechanisms that can be targeted with specific drugs have recently emerged for the treatment of systemic sclerosis (SSc) patients. Over the past 3 years, the achievement of one large phase 3 trial has led to the approval by drug agencies of the first drug licenced for SSc-related interstitial lung disease. Given this exciting time in the SSc field, we aimed to perform a systemic literature review of phase 1, phase 2 and phase 3 clinical trials and large observational studies about targeted therapies in SSc. We searched MEDLINE/PubMed, EMBASE, and ClinicalTrials.gov for clinical studies from 2016 with targeted therapies as the primary treatment in patients with SSc for skin or lung involvement as the primary clinical outcome measure. Details on the study characteristics, the trial drug used, the molecular target engaged by the trial drug, the inclusion criteria of the study, the treatment dose, the possibility of concomitant immunosuppression, the endpoints of the study, the duration of the study and the results obtained were reviewed. Of the 973 references identified, 21 (4 conference abstracts and 17 articles) were included in the systematic review. A total of 15 phase 1/phase 2 clinical trials, 2 phase 3 clinical trials and 2 observation studies were analysed. The drugs studied in phase 1/phase 2 studies included the following: inebilizumab, dabigatran, C-82, pomalidomide, rilonacept, romilkimab, tocilizumab, tofacitinib, pirfenidone, lenabasum, abatacept, belimumab, riociguat, SAR100842 and lanifibranor. All but 3 studies were performed in early diffuse SSc patients with different inclusion criteria, while 3 studies were performed in SSc patients with interstitial lung disease (ILD). Phase 3 clinical trials investigated nintedanib and tocilizumab. Nintedanib was investigated in SSc-ILD patients whereas tocilizumab focused on early diffuse SSc patients with inflammatory features. Two observational studies including > 50 patients with rituximab as the targeted drug were also evaluated. All these studies offer a real hope for SSc patients. The future challenges will be to customize patient-specific therapeutics with the goal to develop precision medicine for SSc.
登录
查看更多内容
影响因子：
27.4
作者：
Gonzalez, Estrella Garcia;Selvi, Enrico;Distler, Joerg H. W.
通讯作者：
Distler, Joerg H. W.
DOI：
10.1002/art.40358
发表时间：
2018-03
期刊：
Arthritis & rheumatology (Hoboken, N.J.)
影响因子：
--
作者：
Gordon JK;Martyanov V;Franks JM;Bernstein EJ;Szymonifka J;Magro C;Wildman HF;Wood TA;Whitfield ML;Spiera RF
通讯作者：
Spiera RF
影响因子：
11.4
作者：
Hecht, A;Vleminckx, K;Kemler, R
通讯作者：
Kemler, R
影响因子：
27.4
作者：
Khan, Korsa;Xu, Shiwen;Ong, Voon H.
通讯作者：
Ong, Voon H.
影响因子：
158.5
作者：
Distler, Oliver;Highland, Kristin B.;Maher, Toby M.
通讯作者：
Maher, Toby M.