Intrathecal Melphalan Chemotherapy for Retinoblastoma with Central Nervous System Metastasis A Multicenter Prospective Single Group Study

What this Trial Is About

Retinoblastoma is the most common eye cancer in infants and children. While early diagnosis of localized retinoblastoma has a survival rate exceeding 95%, cases with central nervous system CNS metastasis have a very poor prognosis, with CNS involvement being the main cause of death. This study investigates the use of intrathecal injection of melphalan, a drug sensitive to retinoblastoma tumor cells, to treat CNS metastasis in retinoblastoma through a multicenter prospective approach. Participants will receive systemic chemotherapy combined with intrathecal chemotherapy every three weeks. After three cycles, treatment response is evaluated by examining cerebrospinal fluid, brain, and eye tumors. If needed, local radiotherapy and arterial interventional chemotherapy may be applied for solid tumors. Chemotherapy continues until no tumor cells are found in cerebrospinal fluid, with a total of six cycles planned including additional intrathecal chemotherapy. Patients will be followed up after completing treatment. During the study, researchers monitor cerebrospinal fluid samples each cycle and assess tumor response. They will measure progression-free survival, overall survival, response rates, and disease-free survival at various time points up to five years. Secondary outcomes include monitoring side effects such as arachnoiditis, encephalopathy, headaches, infections, and quality of life related to health and eye cancer over up to ten years. The total duration of follow-up varies with long-term assessments included.

Intrathecal Chemotherapy for Central Nervous System Metastasis in Retinoblastoma