NCT00334672Unknown3 期
Hemophagocytic Lymphohistiocytosis
Children's Cancer and Leukaemia Group25 个研究点 分布在 1 个国家目标入组 288 人开始时间: 2006年3月1日最近更新:
适应症
相关药物
试验速览
- 阶段
- 3 期
- 发起方
- 入组人数
- 288
- 试验地点
- 25
- 主要终点
- Survival
研究概览
简要总结
RATIONALE: Drugs used in chemotherapy work in different ways to stop the growth of hemophagocytic lymphohistiocytosis cells, either by killing the cells or by stopping them from dividing. Giving more than one drug (combination chemotherapy) may kill more hemophagocytic lymphohistiocytosis cells. A donor stem cell transplant may be able to replace blood-forming cells that were destroyed by chemotherapy. Sometimes the transplanted cells from a donor can make an immune response against the body's normal cells. Cyclosporine and methotrexate may stop this from happening.
PURPOSE: This phase III trial is studying how well combination chemotherapy followed by a donor stem cell transplant works in treating patients with hemophagocytic lymphohistiocytosis.
详细描述
OBJECTIVES:
Primary
- Provide and evaluate revised induction and maintenance therapy comprising etoposide, dexamethasone, and cyclosporine, in terms of achieving and maintaining an acceptable clinical condition in order to perform a curative allogeneic hematopoietic stem cell transplantation (AHSCT), in patients with primary inherited or severe and persistent secondary hemophagocytic lymphohistiocytosis (HLH).
- Evaluate and improve the outcome of AHSCT with various types of donors.
- Determine the prognostic importance of the state of remission at the time of AHSCT.
- Evaluate the neurological complications, in terms of early neurological alterations and cerebrospinal fluid (CSF) findings, in patients treated with this regimen.
Secondary
- Improve the understanding of the pathophysiology of HLH by conducting biological studies of genetics and cytotoxicity in these patients, including genotype-phenotype studies and the prognostic value of natural killer (NK) cell activity subtyping.
研究设计
- 研究类型
- Interventional
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- — 至 17 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •DISEASE CHARACTERISTICS:
- •Newly diagnosed hemophagocytic lymphohistiocytosis (HLH) meeting 1 of the following criteria*:
- •Diagnosis by molecular/genetic methods
- •Diagnosis by meeting 5 out of 8 of the following criteria:
- •Clinical criteria:
- •Splenomegaly
- •Laboratory criteria:
- •Cytopenias affecting ≥ 2 of 3 lineages in the peripheral blood, including the following:
- •Hemoglobin < 9.0 g/dL (< 10.0 g/dL in infants < 4 weeks of age)
- •Platelet count < 100,000/mm^3
- •Neutrophil count < 1,000/mm^3
- •Hypertriglyceridemia and/or hypofibrinogenemia:
- •Fasting triglycerides ≥ 3.0 mmol/L (i.e., ≥ 265 mg/dL)
- •Fibrinogen ≤ 1.5 g/L
- •Histopathologic criteria:
- •Hemophagocytosis in bone marrow, spleen, or lymph nodes
- •No evidence of malignancy
- •New diagnostic criteria:
- •Low or absent natural killer (NK) cell activity
- •Ferritin ≥ 500 mcg/L
- •Soluble CD25 (i.e., soluble interleukin-2 receptor) ≥ 2,400 U/mL NOTE: *Patients who do not meet the diagnostic criteria for HLH but who have a strong clinical suspicion of HLH may be eligible at the discretion of the investigator
- •Primary HLH (i.e., familial hemophagocytic lymphohistiocytosis [FLH]) OR secondary HLH (i.e., severe acquired form of HLH)
- •Acceptable donor meeting 1 of the following criteria:
- •HLA-identical related donor
- •Matched unrelated donor
- •Mismatched unrelated donor
- •Familial haploidentical donor
- •PATIENT CHARACTERISTICS:
- •Not specified
- •PRIOR CONCURRENT THERAPY:
- •No prior cytotoxic treatment for HLH
- •No prior cyclosporine treatment for HLH
排除标准
- 未提供
结局指标
主要结局
Survival
次要结局
未报告次要终点
研究者
研究点 (25)
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