Background: Mucosal angiodysplasias, either inherited or acquired, can cause gastrointestinal bleeding, sometimes refractory to treatment. From earlier case reports, thalidomide has been described to possess some benefits in this disease, but its benefits and risks nevertheless remain unclear.
Objectives: This pilot study assesses the efficacy, safety, and side-effect of thalidomide in the treatment of patients with chronic gastrointestinal bleeding from angiodysplasias.
Methods: Patients with chronic angiodysplasia bleeding and requiring ongoing transfusion were eligible for this open nonrandomized study. Thalidomide was started with 50 mg/day and then increased incrementally by 50 mg every week up to 200 mg/day, if tolerated, and continued for 6 months. Adverse events, hemoglobin, blood chemistry, and blood transfusion were monitored during the treatment and for 6-months posttreatment.
Results: Seven patients were recruited in this study. Four patients discontinued thalidomide within 3-8 weeks, because of fatigue (two patients), peripheral neuropathy (one patient), and skin rash (one patient). All side-effects resolved when thalidomide was discontinued. These four patients required the same volume of blood transfusions per month as pre-study. In contrast, the three patients who continued 100-200 mg/day of thalidomide for 6 months did not require any transfusions during the 6 months of medication. During 6-months posttreatment of these three patients, one maintained response without any transfusion for 2 months, then required 1 U of blood every 4 weeks, one patient required 2 U of blood every 3-4 weeks, and one patient died from diabetes complications.
Conclusion: Thalidomide should be considered as a therapeutic option in patients who are resistant to conventional therapy, but it has a high discontinuation rate because of its side-effects.