Dana-Farber Cancer Institute, Medical Oncology, 450 Brookline Avenue, Mayer 232, Boston 02215, USA.
Expert Opin Drug Saf. 2013 Jan;12(1):123-36. doi: 10.1517/14740338.2012.749855. Epub 2012 Dec 10.
Hepatic veno-occlusive disease (VOD), also known as sinusoidal obstruction syndrome (SOS), is a potentially life-threatening complication of chemotherapeutic conditioning used in preparation for hematopoietic stem-cell transplantation (SCT). Defibrotide (DF) has been shown in Phase II and III trials to improve complete response in patients with severe VOD (sVOD). None of the articles, to date, provide a comprehensive review of the safety of DF in VOD and/or a range of other conditions.
This article reviews current clinical findings on DF, primarily in terms of safety for use in treatment and prophylaxis of VOD, and relevant safety data for its use in other diseases. The literature review was conducted using a PubMed search with the fixed term 'defibrotide' in combination with ≥ 1 of 'safety', 'veno-occlusive disease' (with and without 'treatment', 'prevention'), 'oncology', 'myeloma', 'microangiopathy', 'anti-thrombotic' and 'peripheral vascular disorder'. Related articles from the EBMT and ASH conference websites were also included.
DF was well tolerated in majority of the studies. The safety profile of DF is largely favourable with toxicities comparable to control populations in the setting of SCT complicated by sVOD.
肝静脉闭塞病（VOD），又称窦状隙阻塞综合征（SOS），是造血干细胞移植（SCT）前化疗预处理过程中潜在危及生命的并发症。在 II 期和 III 期试验中，已证明去纤维肽（DF）可改善严重 VOD（sVOD）患者的完全缓解率。迄今为止，尚无任何文章全面综述 DF 在 VOD 和/或其他多种疾病中的安全性。
本文主要综述了 DF 在 VOD 治疗和预防中的安全性方面的最新临床研究结果，以及该药在其他疾病中的使用的相关安全性数据。采用 PubMed 搜索，以固定术语“defibrotide”结合“safety”、“veno-occlusive disease”（with 和 without “treatment”、“prevention”）、“oncology”、“myeloma”、“microangiopathy”、“anti-thrombotic”和“peripheral vascular disorder”中的至少一个，对文献进行了综述。还纳入了来自 EBMT 和 ASH 会议网站的相关文章。
DF 在大多数研究中均具有良好的耐受性。DF 的安全性特征总体上较为有利，毒性与 SCT 并发 sVOD 患者的对照人群相当。
