Intrathecal Chemotherapy for Central Nervous System Metastasis in Retinoblastoma

  • End date
    May 1, 2030
  • participants needed
  • sponsor
    Eye & ENT Hospital of Fudan University
Updated on 9 September 2021


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.

Condition Retinoblastoma, Extraocular, Neoplasm Metastases of Retinoblastoma
Treatment melphalan
Clinical Study IdentifierNCT04903678
SponsorEye & ENT Hospital of Fudan University
Last Modified on9 September 2021


Yes No Not Sure

Inclusion Criteria

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

Exclusion Criteria

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
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