Short Course of Bortezomib in Anemic Patients With Refractory Cold Agglutinin Disease
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 21
- 试验地点
- 8
- 主要终点
- Number of patients who become transfusion-free after Bortezomib therapy.
研究概览
简要总结
This multicenter, phase II, open label study will enroll patients with chronic cold agglutinin disorder. A single course of Bortezomib will be given at the dose of 1,3 mg/sqm iv on days 1, 4, 8, 11.
详细描述
Based on its activity in MM, single agent Bortezomib was tested in phase II trials in lymphoplasmacytic lymphoma, the disorder most frequently associated with CAD, and achieved 40-80% responses. These striking clinical responses indicate that proteasome activity is critical for the survival of immunoglobulin-secreting cells. The resolution of transfusion requirement in two patients with refractory CAD associated with IgMk monoclonal gammopathy has been reported after treatment with a short course of Bortezomib. It may be interesting to test the efficacy of Bortezomib in a larger series of patients with refractory CAD, idiopathic or associated with an otherwise asymptomatic B cell clonal disorder, and to evaluate the duration of clinical benefit.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
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
- •Patients with chronic cold agglutinin disorder requiring or with a hemoglobin concentration below 10g/L determined at least monthly during the two months before entering the trial;
- •Failure of at least one previous treatment attempt;
- •Hemoglobin level assessment;
- •Presence of serum cold agglutinin (CA). If an overnight incubation is used for CA detection, a titer at 4°C of 64 or higher is required;
- •Written informed consent.
排除标准
- •Presence of a concomitant lymphoproliferative disorder requiring specific treatment for reasons other than cold agglutinin related hemolytic anemia;
- •Preexisting peripheral neuropathy;
- •Known hypersensitivity to Bortezomib;
- •Non-cooperative behaviour or non-compliance;
- •Psychiatric diseases or conditions that might impair the ability to give informed consent;
- •Patients who are pregnant (women of childbearing potential must have a negative serum pregnancy test). Post-menopausal women must be amenorrhoic for at least 24 months to be considered of non-childbearing potential. Male and female patients must agree to employ an effective barrier method of birth control throughout the study and for up to 3 months following discontinuation of study drug.
研究组 & 干预措施
Bortezomib therapy
A single course of Bortezomib will be given at the dose of 1,3 mg/sqm iv on days 1, 4, 8, 11. Prophylaxis of HZ reactivation will be given with oral acyclovir at the dosage of 400 mg twice daily for one month after the end of Bortezomib.
干预措施: Bortezomib (Drug)
结局指标
主要结局
Number of patients who become transfusion-free after Bortezomib therapy.
时间窗: After 3 months from study entry.
Cumulative proportion of patients transfusion-free at three months after Bortezomib therapy.
Number of patients who have never been transfused with a >2g hemoglobin rise compared to baseline.
时间窗: After 3 months from study entry.
次要结局
- Number of CTC grade 3 and 4 adverse events.(After 12 months from study entry.)
- Duration in months of transfusion independence.(At 12 months from study entry.)
- Effect of treatment on the underlying clonal B cell disorder.(At 3 months from study entry.)