Division of Gastroenterology and Hepatology, Shanghai Jiao-Tong University, School of Medicine Renji Hospital, Shanghai Institute of Digestive Disease, Shanghai, China.
Gastroenterology. 2011 Nov;141(5):1629-37.e1-4. doi: 10.1053/j.gastro.2011.07.018. Epub 2011 Jul 22.
BACKGROUND & AIMS: Patients with recurrent bleeding from gastrointestinal vascular malformations are a challenge to treat. We investigated the long-term efficacy and safety of thalidomide for refractory bleeding from gastrointestinal vascular malformations in an open-label, randomized study.
Eligible patients were randomly assigned to groups that were given either 100 mg thalidomide (n = 28) or 400 mg iron (n = 27, controls), daily for 4 months; patients were followed for at least 1 year (mean, 39 months). Bleeding was defined by a positive result from an immunoassay fecal occult blood test. The primary end point was the effective response rate, defined as the proportion of patients in whom bleeding episodes had decreased by ≥ 50% in the first year of the follow-up period. The secondary end points included the rates of cessation of bleeding, blood transfusion, overall hospitalization, and hospitalization for bleeding. We also quantified yearly bleeding episodes, bleeding duration, levels of hemoglobin, and yearly requirements for transfusions of red cells, numbers of hospitalizations for bleeding, and hospital stays. Plasma levels of vascular endothelial growth factor were measured in the group given thalidomide.
Rates of response in the thalidomide and control groups were 71.4% and 3.7%, respectively (P < .001). All secondary end points differed significantly different between groups; thalidomide was more effective. No severe adverse effects were observed, although minor side effects were common among patients in the thalidomide group. Levels of vascular endothelial growth factor were significantly reduced by thalidomide (P < .001).
Thalidomide is an effective and relatively safe treatment for patients with refractory bleeding from gastrointestinal vascular malformations. Mechanisms of thalidomide activity might involve vascular endothelial growth factor.
胃肠道血管畸形再出血的患者治疗具有挑战性。我们在一项开放性、随机研究中调查了反应停治疗胃肠道血管畸形难治性出血的长期疗效和安全性。
合格患者被随机分配到每天接受 100 毫克反应停（n = 28）或 400 毫克铁（对照组，n = 27）治疗 4 个月的组中；患者随访至少 1 年（平均 39 个月）。出血定义为免疫测定粪便隐血试验阳性。主要终点是有效反应率，定义为在随访期第一年出血减少≥50%的患者比例。次要终点包括出血停止率、输血率、总住院率、出血住院率。我们还量化了每年的出血发作、出血持续时间、血红蛋白水平以及每年红细胞输血需求、因出血住院次数和住院时间。在接受反应停治疗的组中测量了血管内皮生长因子的血浆水平。
反应停组和对照组的反应率分别为 71.4%和 3.7%（P <.001）。各组之间的所有次要终点均有显著差异；反应停更有效。未观察到严重不良反应，尽管反应停组患者常有轻微副作用。血管内皮生长因子的水平明显被反应停降低（P <.001）。
反应停是治疗胃肠道血管畸形难治性出血患者的一种有效且相对安全的治疗方法。反应停的作用机制可能涉及血管内皮生长因子。
