title: Efficacy of thalidomide for refractory gastrointestinal bleeding from vascular malformation.
abstract: OBJECTIVE
  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.
  
  
  METHODS
  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.
  
  
  RESULTS
  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).
  
  
  CONCLUSIONS
  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.
authors.affiliation: Division of Gastroenterology and Hepatology, Shanghai Jiao-Tong University, School of Medicine Renji Hospital, Shanghai Institute of Digestive Disease, Shanghai, China. zhizhengge@yahoo.com.cn
authors.forename: Zhi-Zheng
authors.initials: ZZ
authors.lastname: Ge
authors.forename: Hui-Min
authors.initials: HM
authors.lastname: Chen
authors.forename: Yun-Jie
authors.initials: YJ
authors.lastname: Gao
authors.forename: Wen-Zhong
authors.initials: WZ
authors.lastname: Liu
authors.forename: Chun-Hong
authors.initials: CH
authors.lastname: Xu
authors.forename: Hong-Hong
authors.initials: HH
authors.lastname: Tan
authors.forename: Hai-Ying
authors.initials: HY
authors.lastname: Chen
authors.forename: Wei
authors.initials: W
authors.lastname: Wei
authors.forename: Jing-Yuan
authors.initials: JY
authors.lastname: Fang
authors.forename: Shu-Dong
authors.initials: SD
authors.lastname: Xiao
mesh_terms: D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D020533:Angiogenesis Inhibitors / Q000009:adverse effects / Q000627:therapeutic use*; D004305:Dose-Response Relationship, Drug; D005260:Female; D006471:Gastrointestinal Hemorrhage / Q000188:drug therapy* / Q000209:etiology* / Q000517:prevention & control; D006801:Humans; D007501:Iron / Q000627:therapeutic use; D008297:Male; D008875:Middle Aged; D055502:Secondary Prevention; D013792:Thalidomide / Q000009:adverse effects / Q000627:therapeutic use*; D016896:Treatment Outcome; D042461:Vascular Endothelial Growth Factor A / Q000097:blood; D054079:Vascular Malformations / Q000150:complications*
chemical_list: D020533:Angiogenesis Inhibitors; D042461:Vascular Endothelial Growth Factor A; D013792:Thalidomide; D007501:Iron