Phase 2
N=55
Long-term Effects of Thalidomide for Recurrent Gastrointestinal Bleeding Due to Vascular Malformation
Obscure Gastrointestinal Bleeding · Angiodysplasia · Gastric Antral Vascular Ectasia · Thalidomide
Bottom Line
View on ClinicalTrials.gov: NCT00964496 ↗Enrolled (actual)
55
Serious AEs
0.0%
Results posted
Apr 2010
Primary outcome: Primary: Participants Whose Rebleeds Decreased From Baseline by ≥ 50% at 12 Months — 20; 1 participants — p=0.00000013
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Thalidomide (Drug); Iron (Drug)
- Age
- Adult, Older Adult · 40+ yrs
- Sex
- All
- Sponsor
- Shanghai Jiao Tong University School of Medicine
- Primary completion
- Nov 2007
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Participants Whose Rebleeds Decreased From Baseline by ≥ 50% at 12 Months |
20; 1 | 0.00000013 sig |
| PRIMARY Cessation of Bleeding |
13; 0 | 0.00003962 sig |
| SECONDARY Change From Baseline in Hemoglobin (Hb) Level at 12 Months |
3.06; -0.01 | <0.001 sig |
| SECONDARY Change From Baseline in Bleeding Episodes at 12 Months |
-9.36; 1.41 | <0.01 sig |
| SECONDARY Change From Baseline in Bleeding Duration at 12 Months |
5.2; 0.8 | 0.00024740 sig |
| SECONDARY Participants Dependent on Blood Transfusions |
3; 13 | 0.00298881 sig |
| SECONDARY Change From Baseline in Total Transfused Red Cell Requirements at 12 Months |
-1585.71; -28.57 | <0.01 sig |
Summary
Background: Repeated episodes of bleeding from gastrointestinal vascular malformations refractory to endoscopic or surgical therapy often pose a major therapeutic challenge.
Methods: The investigators performed a randomized, parallel controlled study of thalidomide as a therapy for recurrent gastrointestinal bleeding due to vascular malformation. Patients with at least six episodes of bleeding in the prior year due to vascular malformation were randomly grouped, prescribed a four-month regimen of either 25 mg of thalidomide or 100 mg of iron orally four times daily, and monitored for at least one year. The primary end point was defined as the patients whose rebleeds decreased from baseline by ≥ 50% at 12 months and the cessation of bleeding. Rebleeding was defined based on a positive fecal occult blood test (FOBT) (monoclonal colloidal gold color technology) at any visit after treatment. Secondary outcomes included the participants dependent on blood transfusions and changes from baseline in transfused packed red cell units, bleeding episodes, bleeding durations, and hemoglobin levels at 12 months. Statistical significance was defined at P < 0.05.
Eligibility Criteria
Inclusion Criteria
- Age between 40-85 years; women were post-menopausal, post-tubal ligation, or on some form of birth control like long-term laying up contraceptive ring or using condom;
- History of at least six documented gastrointestinal bleeding episodes in the year prior to randomization, which were refractory or inaccessible to endoscopic therapy or surgical ectomy;
- Confirmed diagnosis of vascular malformation by esophagogastroduodenoscopy (EGD), capsule endoscope (CE), double-balloon endoscope (DBE), or colonoscopy, but no obvious infectious, neoplastic, or other specific diagnosis;
- Angiodysplasia at endoscopy characterized by focal or diffused venous/capillary lesions presenting as bright red ectatic vessels or pulsatile red protrusions, with surrounding venous dilatation or patchy erythema with or without oozing;
- Endoscopic appearance of GAVE (also known as watermelon stomach), indicated by longitudinal antral folds converging on the pylorus, containing visible columns of tortuous red ecstatic vessels.
Exclusion Criteria
- Patients were excluded if their bleeding history were less than 1 year;
- if they had cirrhotic or portal hypertension gastropathy; severe co-morbidities of cardiac, pulmonary, renal, liver, hematological, rheumatologic disorders, or uncontrollable diabetes mellitus or hypertension;
- if they had a history of severe bilateral peripheral neuropathy or seizure activity, thromboembolic disease, known thalidomide or iron allergy, or prior treatment of gastrointestinal bleeding with thalidomide;
- if they had a history of treatment with any dose of systemic or oral topical corticosteroids or aspirin, NSAIDs, anti-platelet drugs, anticoagulants, or Chinese medications (with salicylates), gingko, or Echinacea, or other putative immunomodulators or anti-angiogenic agents;
- Currently pregnant or lactating or currently undergoing systemic cancer chemotherapy or receiving radiation
- if they were undergoing systemic cancer chemotherapy or receiving radiation.
Data sourced from ClinicalTrials.gov (NCT00964496). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.