Thalidomide and its derivatives have been used in the treatment of myelodysplastic syndrome (MDS) because of their anti-angiogenic and immunomodulatory effects. In recent years, some studies have found that thalidomide and its derivatives not only showed significant efficacy in lower-risk MDS patients with del (5q), but also showed advantages in non-del (5q) MDS patients. In addition, the discovery of its molecular targets and new substrates makes it possible to develop a new generation of immunomodulatory drugs (IMiDs) and to design IMiDs-based proteolysis-targeting chimeras. In this review, the new progress in mechanism and clinical application of thalidomide and its derivatives were summarized briefly, so as to provide a more scientific, reasonable and effective scheme to the treatment of MDS.
题目: 沙利度胺及其衍生物治疗骨髓增生异常综合征的研究进展.
摘要: .沙利度胺及其衍生物因具有抗血管生成和免疫调节作用而用于骨髓增生异常综合征（MDS）的治疗，近年来，有研究发现其不仅在伴del（5q）的较低危MDS患者中疗效显著，在非del（5q）MDS的治疗中也显示出优势。另外，其分子靶点及新底物的发现使得新一代免疫调节药（IMiDs）的开发及基于IMiDs的蛋白水解靶向嵌合体的药物设计成为可能。本文就沙利度胺及其衍生物的作用机制及临床应用新进展作一综述，为MDS的治疗提供更加科学、合理、有效的方案。.