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Current and Future Outlook on Disease Modification and Defining Low Disease Activity in Systemic Sclerosis.
Arthritis & Rheumatology ( IF 11.4 ) Pub Date : 2020-03-05 , DOI: 10.1002/art.41246
Vivek Nagaraja 1 , Marco Matucci-Cerinic 2 , Daniel E Furst 3 , Masataka Kuwana 4 , Yannick Allanore 5 , Christopher P Denton 6 , Ganesh Raghu 7 , Vallerie Mclaughlin 1 , Panduranga S Rao 1 , James R Seibold 8 , John D Pauling 9 , Michael L Whitfield 10 , Dinesh Khanna 1
Affiliation  

Systemic sclerosis (SS c) is an autoimmune rheumatic disease with heterogeneous clinical manifestations and a variable course in which the severity of the pathology dictates the disease prognosis and course. Among autoimmune rheumatic diseases, SS c has the highest mortality rate among all rheumatic diseases, though there are exciting new therapeutic targets that appear to halt the progression of SS c manifestations such as skin or lung fibrosis. In selected patients, high‐intensity regimens with autologous stem cell transplantation can favorably modify the course. In what was once thought to be an untreatable disease, targeted therapies have now changed the outlook of SS c to a treatable disorder. Herein, we discuss the targeted therapies modifying the outlook on selected organ involvement and creating opportunities for future treatment. We also present a framework for defining low disease activity in SS c.

中文翻译:

当前和未来的疾病改变和定义系统性硬化症的低疾病活动的前景。

系统性硬化症(SS c)是一种自身免疫性风湿性疾病,具有异质的临床表现和可变的病程,其中病理的严重程度决定了疾病的预后和病程。在自体免疫性风湿性疾病中,SS c在所有风湿性疾病中具有最高的死亡率,尽管有一些激动人心的新治疗靶标似乎阻止了诸如皮肤或肺纤维化等SS c表现的发展。在某些患者中,高强度自体干细胞移植疗法可以很好地改变病程。在曾经被认为是无法治愈的疾病中,靶向疗法现已将SS c的前景改变为可治疗的疾病。在本文中,我们讨论了靶向疗法,该疗法改变了对选定器官参与的看法,并为将来的治疗创造了机会。
更新日期:2020-03-05
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