PILOT INVESTIGATION OF THE SAFETY AND EFFICACY OF THALIDOMIDE IN SCLERODERMA

硬皮病是一种病因不明的结缔组织病。 以皮肤和内脏器官过度纤维化为特征。 硬皮病患者对传统免疫抑制药物没有反应 和抗炎疗法，而这些患者中的大多数 经历进行性疾病，有明显的发病率和死亡率。 骨髓移植中发生的慢性移植物抗宿主病 硬皮病患者有许多共同特征。这种药 沙利度胺，先前因其早期的致畸作用而闻名 1960年的S，自那以后被发现具有抗炎和免疫- 在许多免疫介导性疾病中的调节作用，包括 移植物抗宿主病。此外，初步研究表明， 移植物抗宿主病的常规治疗方法是环孢素A， 可能对治疗硬皮病有效。沙利度胺的使用还没有 在硬皮病患者中有报道。这项初步研究将获得 沙利度胺治疗硬皮病的安全性和有效性的初步数据 患者通过建立基线的临床和血清学资料 患者，然后在每天暴露于 沙利度胺的疗程为12周。

Scleroderma is a connective tissue disease of unknown etiology characterized by excessive fibrosis of the skin and visceral organs. Scleroderma patients have not responded to traditional immunosuppressive and anti-inflammatory regimens, and the majority of these patients experience progressive disease with marked morbidity and mortality. Chronic graft versus host disease that occurs in bone marrow transplant patients shares many characteristics with scleroderma. The drug thalidomide, previously known for its teratogenic effects in the early 1960's, has since been found to have anti-inflammatory and immune- modulating effects in a number of immune mediated diseases, including graft-versus-host disease. Furthermore, preliminary studies suggest that the conventional treatment of graft-versus-host disease, cyclosporin A, may be effective in treating scleroderma. Thalidomide use has not been reported in scleroderma patients. This pilot study will obtain preliminary data on the safety and efficacy of thalidomide in scleroderma patients by establishing baseline clinical and serological profiles of patients and then follow those parameters during daily exposure to thalidomide over the course of 12 weeks.