A Single-center, Prospective, Non-comparative Clinical Trial of Ruxolitinib and Methylprednisolone as a First-line Treatment for Macrophage Activation Syndrome
试验速览
- 阶段
- 3 期
- 入组人数
- 40
- 主要终点
- Response rate
研究概览
简要总结
The present study was a single-center, prospective, non-comparative in which macrophage activation syndrome patients were selected as the main subjects to evaluate the effect and safety of Ruxolitinib and methylprednisolone regimens as the first-line therapy .
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
- 1 Year 至 75 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Meet hemophagocytic lymphohistiocytosis (HLH)-04 diagnostic criteria; patients were diagnosed with autoimmune disease associated HLH (Macrophage activation syndrome, MAS).
- •No HLH induction therapy was performed.
- •The expected survival time is more than 1 month.
- •Serum creatinine ≤ 1.5 times normal；Serum human immunodeficiency virus(HIV) antigen or antibody negative； Hepatitis C virus (HCV) antibody is negative, or HCV antibody is positive, but HCV RNA is negative；HBV copies less than 1E+03 copies/ml.
- •Total bilirubin ≤10 times the upper limit of normal; serum creatinine ≤1.5 times the normal value
- •The left ventricular ejection fraction (LVEF) was normal.
- •No uncontrollable infection.
- •Contraception for both male or female.
- •Informed consent obtained.
排除标准
- •Pregnancy or lactating Women；
- •Allergic to ruxolitinib；
- •Active bleeding of the internal organs；
- •uncontrollable infection；
- •Serious mental illness;
- •Non-melanoma skin cancer history;
- •Patients unable to comply during the trial and/or follow-up phase;
- •Participate in other clinical research at the same time.
研究组 & 干预措施
Ruxolitinib+methylprednisolone
Ruxolitinib and methylprednisolone administered as the first-line therapy
干预措施: Ruxolitinib (Drug)
Ruxolitinib+methylprednisolone
Ruxolitinib and methylprednisolone administered as the first-line therapy
干预措施: methylprednisolone (Drug)
结局指标
主要结局
Response rate
时间窗: Change from before and 2,4,6 and 8 weeks after initiating Ruxolitinib combined with methylprednisolone therapy
A complete response was defined as normalization of all of the quantifiable symptoms and laboratory markers of HLH, including levels of sCD25, ferritin, and triglyceride; hemoglobin; neutrophil counts; platelet counts; and alanine aminotransferase (ALT). A partial response was defined as at least a 25% improvement in 2 or more quantifiable symptoms and laboratory markers as follows: sCD25 response was\>1.5-fold decreased; ferritin and triglyceride decreased at least 25%; for patients with an initial neutrophil count of\<0.5 ×109/L, a response was defined as an increase by at least 100% to\>0.5× 109/L; for patients with a neutrophil count of 0.5 to 2.0 × 109/L, an increase by at least 100% to \>2.0 × 109/L was considered a response; and for patients with ALT \>400 U/L, response was defined as an ALT decrease of at least 50%.
Progression Free Survival
时间窗: 2 years
from date of inclusion to date of progression, relapse, or death from any cause
Adverse events
时间窗: 2 years
Adverse events including myelosuppression, infection, hemorrhage
次要结局
未报告次要终点
研究者
Zhao Wang
Clinical Professor
Beijing Friendship Hospital