A Multicenter, Randomized, Open-label Clinical Trial Assessing the Efficacy of Octreotide in Decreasing Blood and Iron Requirements in Patients With Refractory Anemia Due to Angiodysplasias
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 62
- 试验地点
- 12
- 主要终点
- Difference in blood and parenteral iron requirements (transfusion units)
研究概览
简要总结
The purpose of this study is to determine whether 40 mg octreotide long-acting release intramuscular every 28 days is effective in the treatment of patients with refractory anemia due to gastrointestinal angiodysplasias. We hypothesize that octreotide is effective in reducing the transfusion requirements (consisting of red blood cell transfusions and intravenous iron infusions) of patients with angiodysplasia-related anemia.
详细描述
Rationale: Gastrointestinal angiodysplasias are a common source of intractable small bowel bleeding, especially in older patients. Endoscopic ablation of angiodysplasias has limited efficacy and rebleeding rates are substantial. Recurrent bleeding results in refractory anemia which is managed with blood transfusions and/or iron infusions. Transfusion dependency reduces quality of life and is associated with substantial cardiovascular morbidity and mortality. Small cohort studies suggest a beneficial effect of octreotide in bleeding angiodysplasias, but evidence from rigorous, well-controlled studies are lacking.
Objective: To assess the efficacy of octreotide in reducing the transfusion requirements (consisting of blood transfusions and iron infusions) of patients with refractory anemia due to gastrointestinal angiodysplasias despite endoscopic intervention.
Study design: Multicenter, randomized, open-label intervention study.
Study population: Patients aged 18 years or older with transfusion-dependent anemia due to endoscopically confirmed angiodysplasias. Transfusion units consist of iron infusions (of 500 milligrams [mg]) and red blood cell (RBC) transfusions. At least one endoscopic attempt to treat the angiodysplasias needs to be recorded unless contra-indications are present. Patients with liver cirrhosis Child-Pugh C or liver failure, uncontrolled diabetes mellitus (defined by a glycated hemoglobin >64 mmol/mL), symptomatic cholecystolithiasis, and pregnant or nursing women, are regarded as ineligible because of the pharmacological profile of octreotide. Patients with hereditary hemorrhagic diseases or hematological disorders on active treatment, other alternative causes of gastrointestinal bleeding, presence of left ventricular assist devices, as well as patients with cancer under active treatment, and those with a life expectancy <1 year are excluded from enrolment
Intervention: Patients will be randomized (1:1) into two groups. The intervention group receives 40 mg octreotide long-acting release (Sandostatin LAR) every 28 days for a total period of 52 weeks as an adjunct to standard of care. The control group receives standard of care along. The last follow-up visit is in week 60.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Single (Outcomes Assessor)
盲法说明
The trial was blinded for the outcome adjudication committee but not for patients or attending physicians.
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- 未提供
排除标准
- •Liver cirrhosis Child-Pugh C, liver failure or diagnosed portal hypertension
- •Previous treatment with octreotide for the same indication (refractory anemia due to angiodysplasias)
- •Current thalidomide treatment which is effective (no transfusion dependency)
- •Life expectancy < 1 year
- •Left ventricular assist devices (LVAD's)
- •Hereditary hemorrhagic diseases or hematological disorders with active treatment
- •Pregnancy or nursing women
- •Uncontrolled diabetes as defined by HbA1C >64 mmol/ml, despite adequate therapy
- •Known hypersensitivity to somatostatin analogs
- •Symptomatic cholecystolithiasis
- •Systemic cancer under active treatment (chemotherapy or radiation therapy)
研究组 & 干预措施
Octreotide
Drug: Sandostatin LAR Sandostatin LAR 40 mg will be administered once every 4 weeks as a intramuscular injection
干预措施: Octreotide (Drug)
结局指标
主要结局
Difference in blood and parenteral iron requirements (transfusion units)
时间窗: Study year (52 weeks)
The mean difference in blood (RBC transfusions per 500 ml or packed cells) and parenteral iron (IV iron infusions per 500 mg) requirements between the intervention and standard of care arm, corrected for baseline transfusion requirements and follow-up time.
次要结局
- Difference in ferritin levels(Study year (52 weeks))
- Use of concomitant care(Study year (52 weeks))
- Difference in endoscopic procedures(Study year (52 weeks))
- Proportion with a good treatment response(During the study year (52 weeks) compared to the year (52 weeks) before randomization)
- Difference in hemoglobin levels(Study year (52 weeks))
- Difference in quality of life(Study year (52 weeks))
- Difference in serious adverse events(Study year (52 weeks))
- Difference in mortality(Study year (52 weeks))
- Difference in bleeding episodes(Study year (52 weeks))
- Difference in healthcare utilization(Study year (52 weeks))
- Difference in fatigue levels(Study year (52 weeks))
- Difference in adverse events(Study year (52 weeks))