DEP-ruxolitinib Regimen as a Salvage Therapy for Refractory/Relapsed Hemophagocytic Lymphohistiocytosis
试验速览
- 阶段
- 3 期
- 入组人数
- 80
- 试验地点
- 1
- 主要终点
- Evaluation of treatment response
研究概览
简要总结
This study aimed to investigate the efficacy and safety of ruxolitinib together with liposomal doxorubicin, etoposide and methylprednisolone (DEP-Ru) as a salvage therapy for refractory/relapsed hemophagocytic lymphohistiocytosis.
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 70 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •meet HLH-04 diagnostic criteria;
- •treated with HLH-94 no less than 2 weeks before enrollment and did not achieve at least PR; or relapsed patients after remission；
- •Life expectancy exceeds 1 month;
- •Age≥1 year old and ≤70 years old, gender is not limited；
- •Before the start of the study, total bilirubin ≤ 10 times the upper limit of normal; serum creatinine ≤ 1.5 times normal；
- •Serum HIV antigen or antibody negative；
- •HCV antibody is negative, or HCV antibody is positive, but HCV RNA is negative；
- •Both HBV surface antigen and HBV core antibody were negative. If any of the above is positive, peripheral blood hepatitis B virus DNA titer should be detected, and less than 1×103 copies/ml can enter the group；
- •Echocardiographic examination of LVEF ≥ 50%;
- •Informed consent.
排除标准
- •Heart function above grade II (NYHA)；
- •Accumulated dose of doxorubicin above 300mg/m2 or epirubicin above 450mg/m2；
- •Pregnancy or lactating Women；
- •Allergic to Pegaspargase, doxorubicin or etoposide；
- •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
DEP-Ru
doxorubicin (doxorubicin hydrochloride liposome injection) 25 mg/m2 day 1; etoposide 100 mg/m2 was administered day1; methylprednisolone 2 mg/kg days 1 to 5,then gradually reduce; ruxolitinib 0.3mg/kg/d。This regimen was repeated after 2 weeks.
干预措施: DEP-Ru (Drug)
结局指标
主要结局
Evaluation of treatment response
时间窗: Change from before and 2,4,6 and 8 weeks after initiating DEP-Ru salvage 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%.
次要结局
- Survival(from the time patients received DEP-Ru salvage therapy up to 24 months or June 2021)
- Adverse events that are related to treatment(through study completion, an average of 2 years)
研究者
Zhao Wang
Clinical Professor
Beijing Friendship Hospital