Intrathecal Chemotherapy for Central Nervous System Metastasis in Retinoblastoma
Status:
Recruiting
Trial end date:
2030-05-01
Target enrollment:
Participant gender:
Summary
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.
Phase:
N/A
Details
Lead Sponsor:
Eye & ENT Hospital of Fudan University
Collaborators:
Children's Hospital of Fudan University Zhongshan Ophthalmic Center, Sun Yat-sen University