Efficacy of Thalidomide for Refractory Gastrointestinal Bleeding From Vascular Malformation
Ge ZZ et al. – 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.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.
- 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.
- Secondary end points included the rates of cessation of bleeding, blood transfusion, overall hospitalization, and hospitalization for bleeding.
- This study 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).