地夫可特治疗严重肝静脉阻塞性疾病的安全性和疗效。
Safety and efficacy of defibrotide for the treatment of severe hepatic veno-occlusive disease.
机构信息
Dana-Farber Cancer Institute, 450 Brookline Avenue, Boston, MA 02115, USA.
出版信息
Ther Adv Hematol. 2012 Aug;3(4):253-65. doi: 10.1177/2040620712441943.
Abstract
Hepatic veno-occlusive disease (VOD), also known as sinusoidal obstruction syndrome, is a potentially life-threatening complication of chemotherapeutic conditioning used in preparation for hematopoietic stem-cell transplantation (SCT). VOD may occur in up to 62% of patients undergoing SCT, with onset generally within the first month after SCT. In severe cases, 100-day mortality is in excess of 80%. Current management consists of best supportive care, with no agents to date approved for treatment in the USA or the EU. Defibrotide, a polydisperse oligonucleotide, has been shown in phase II and III trials to improve complete response and survival in patients undergoing SCT with severe VOD. This article reviews our current understanding of VOD, and examines recent clinical findings on defibrotide for the treatment and prophylaxis of VOD.
摘要
肝静脉闭塞病（VOD），也称窦状隙阻塞综合征，是造血干细胞移植（SCT）预处理中化疗药物的一种潜在致命并发症。VOD 可发生于多达 62%的 SCT 患者，通常在 SCT 后一个月内发病。在严重情况下，100 天死亡率超过 80%。目前的治疗方法包括最佳支持治疗，目前在美国或欧盟尚未批准任何药物用于治疗。多分散寡核苷酸地西他滨在 II 期和 III 期试验中显示可改善严重 VOD 患者 SCT 的完全缓解率和生存率。本文综述了我们目前对 VOD 的认识，并探讨了地西他滨治疗和预防 VOD 的最新临床研究结果。
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