Gordon Jessica, Spiera Robert
Department of Rheumatology, Hospital for Special Surgery, 535 East 70th Street, New York, NY 10021, USA.
Int J Rheumatol. 2011;2011:842181. doi: 10.1155/2011/842181. Epub 2011 Oct 13.
Tyrosine kinase inhibitors (TKIs) have emerged as a targeted therapy of interest for the treatment of systemic sclerosis (SSc). Recently, several groups have performed pilot or "proof-of-concept" studies to determine the feasibility of this approach for the treatment of the cutaneous and pulmonary manifestations of this multisystem disease. The conclusions drawn by these different studies have been conflicting, and some controversy has arisen as to whether tyrosine kinase inhibition is a treatment approach worthy of continued study. This paper summarizes this research to date with emphasis on the challenges in interpreting proof-of-concept studies in this patient group.
酪氨酸激酶抑制剂（TKIs）已成为治疗系统性硬化症（SSc）的一种备受关注的靶向治疗方法。最近，几个研究小组开展了试点或“概念验证”研究，以确定这种方法治疗这种多系统疾病的皮肤和肺部表现的可行性。这些不同研究得出的结论相互矛盾，对于酪氨酸激酶抑制是否是一种值得继续研究的治疗方法也产生了一些争议。本文总结了迄今为止的这项研究，重点关注在解释该患者群体概念验证研究时面临的挑战。
