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.
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
KPS≥60%
Adequate bone marrow and organ function
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.
Single group
Melphalan
DrugMelphalan is performed for intrathecal chemotherapy
progression-free survival
living subjects
overall survival at 2-year interval
living subjects
overall survival at 5-year interval
living subjects
response rate
tumor-free subjects
Number of Participants with Arachnoiditis
A disorder characterized by inflammation of the arachnoid membrane and adjacent subarachnoid space.
Number of Participants with Encephalopathy
A disorder characterized by a pathologic process involving the brain.
Number of Participants with Headache
A disorder characterized by a sensation of marked discomfort in various parts of the head, not confined to the area of distribution of any nerve.
Number of Participants with Encephalomyelitis infection
A disorder characterized by an infectious process involving the brain and spinal cord tissues.
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Eye & ENT Hospital of Fudan University
Lead sponsor
Zhongshan Ophthalmic Center, Sun Yat-sen University
Collaborator
Children's Hospital of Fudan University
Collaborator
