Intrathecal Chemotherapy for Central Nervous System Metastasis in Retinoblastoma (A Multicenter Prospective Single Arm Trial)
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 18
- 试验地点
- 1
- 主要终点
- progression-free survival
研究概览
简要总结
Retinoblastoma is the most common intraocular malignancy in infancy and childhood. The overall survival rate of retinoblastoma was reported to exceed 95% when children were early diagnosed with localized intraocular phase. The advanced retinoblastoma cases with central nervous system metastasis (CNS)is associated with exceedingly poor prognosis. CNS metastasis is the main cause of death in retinoblastoma. Meningeal involvement combined with spinal cord membrane involvement might be due to the meninges invasion and/or the spreading of CSF.
As the most sensitive drug to retinoblastoma tumor cells in vitro, melphalan is the most important drug in the local treatment of retinoblastoma, and it is also an irreplaceable drug in the current eye preservation treatment, which greatly improves the success rate of eye preservation for advanced retinoblastoma. According to the research and the practice of vitreous injection (melphalan), the drug concentration in vitreous can effectively kill retinoblastoma tumor cells.
It is reasonable to speculate that the tumor cells in CSF can be effectively killed by melphalan. Therefore, based on the above background, this study will explore the effectiveness and feasibility of intrathecal injection of melphalan in patients with CNS metastasis of retinoblastoma through a multicenter prospective study.
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
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •The pathological diagnosis was retinoblastoma. According to the IRSs (International retinoblastoma staging system) staging system, the stage was IVB
- •CSF tumor cells on cytology, or radiographic evidence of NM on MRI scans and histologic diagnosis of systemic malignancy needed
- •Adequate bone marrow and organ function
- •The parents signed the informed consent and were willing to accept the treatment and follow-up.
排除标准
- •1.Eye diseases other than retinoblastoma
- •The eyes were infected within 30 days before screening
- •There was a history of surgery and / or unhealed wound within 1 month before enrollment .
- •Those who have allergic reaction or allergic history to chemotherapeutic drugs.
- •Infectious diseases requiring oral, intramuscular or intravenous administration.
- •Patients with systemic immune diseases.
- •Active disseminated intravascular coagulation.
- •Abnormal coagulation function.
- •Abnormal bone marrow and organ function.
- •Uncontrollable clinical problems.
- •The researchers believe that those who need to be excluded.
研究组 & 干预措施
intrathecal chemotherapy in patients with central metastases
Systemic chemotherapy and intrathecal chemotherapy are performed every 3 weeks. After three treatment cycles, the treatment response is comprehensively evaluated including cerebrospinal fluid, intracranial and orbital tumors. If necessary, local radiotherapy and arterial interventional chemotherapy are performed for local solid tumors. Cerebrospinal fluid is detected in each treatment cycle. If RB tumor cells still exist in cerebrospinal fluid, chemotherapy and intrathecal chemotherapy are continued until the end event. If cerebrospinal fluid was negative, intrathecal chemotherapy is supplemented for another 2 cycles with a total of 6 cycles of systemic chemotherapy. The patients are followed up after treatment.
干预措施: Melphalan (Drug)
结局指标
主要结局
progression-free survival
时间窗: 1 year
living subjects
disease-free survival at 5-year interval
时间窗: 5 years
tumor-free subjects
overall survival at 2-year interval
时间窗: 2 years
living subjects
disease-free survival at 2-year interval
时间窗: 2 years
tumor-free subjects
overall survival at 5-year interval
时间窗: 5 years
living subjects
response rate
时间窗: 1 year
tumor-free subjects
次要结局
- Number of Participants with Headache(2 years)
- Ophthalmic Cancer-Related Quality of life(10 years)
- Number of Participants with Encephalopathy(5 years)
- Number of Participants with Encephalomyelitis infection(1 year)
- Number of Participants with Arachnoiditis(2 years)
- Health-Related Quality of life(10 years)
研究者
Jiang Qian
Professor
Eye & ENT Hospital of Fudan University