Autologous Hematopoietic Stem Cell Transplantation for Refractory Multiple Sclerosis
试验速览
- 阶段
- 不适用
- 状态
- 尚未招募
- 入组人数
- 20
- 主要终点
- 3 year MS activity free survival
研究概览
简要总结
Autologous hematopoietic stem cell transplantation (aHSCT) is the only treatment for refractory autoimmune diseases capable of inducing long-term, drug-free and asymptomatic remission. Over the past two decades, aHSCT has been used to treat inflammatory autoimmune disease of the CNS. Patients with relapsing-remitting multiple sclerosis benefit from aHSCT treatment. However, a certain percentage of patients still experience recurrence 3 or 5 years after transplantation. Therefore, exploration of conditioning regimens will drive therapeutic advances in aHSCT in autoimmune diseases of the CNS.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Basic Science
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 60 Years（Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Age 18-60 years;
- •Diagnosed multiple sclerosis with relapses or progression and sustained accumulated impairment by a neurologist expert in the field;
- •EDSS score of 3-6 (including 3 and 6);
- •EDSS cerebellar functional score ≥ 3 or EDSS pyramidal functional score ≥3；
- •Evidence of current disease activity;
- •If a patient has previously received a cytotoxic agent (mitoxantrone, cyclophosphamide etc.) they must have normal bone marrow morphology and cytogenetics before being considered eligible for this study ;
- •No evidence of hepatic inflammation or fibrosis;
排除标准
- •Patients with evidence of myelodysplasia or other non-autoimmune cytopenia;
- •Patients having received a cytotoxic agent within one month of enrolling in this study;
- •Patient with any active or chronic infection (herpes simplex virus, varicella-zoster virus, cytomegalovirus, EB virus, human immunodeficiency virus, hepatitis virus, syphilis, etc.);
- •Patients having received a cytotoxic agent within one month of enrolling in this study;
- •Patients with a malignant tumor currently or within the last 5 years;
- •Patients with cardiac, renal, pulmonary, hepatic or other organ impairment;
- •Patients whose life expectancy is severely limited by another conditions;
- •Pregnancy or risk of pregnancy;
- •Patients unable to give written informed consent in accordance with research ethics board guidelines.
结局指标
主要结局
3 year MS activity free survival
时间窗: 3 year follow-up post transplant
The events for the primary outcome are: clinical relapse, appearance of a new or Gd-enhancing lesion on MRI, or sustained progression of EDSS score.
次要结局
- Time to MS treatment failure(3 years)
- Transplant related morbidity(3 years)
- Transplant related mortality(3 years)
- Immune reconstitution following transplant(3 years)
- Hematopoietic reconstitution following transplant(3 years)
- Imaging changes associated with the disease activity(3 years)
研究者
Qiang Liu
Professor of Department of Neurology
Tianjin Medical University General Hospital