Intra-arterial Chemotherapy (Melphalan) for the Treatment of Retinoblastoma

Inclusion Criteria

• Patients diagnosed with retinoblastoma
• Patients whose other treatment options would require systemic chemotherapy, radiotherapy, or enucleation
• Patient or parent/legal guardian must sign a written informed consent
• One of: * Patients who have bilateral Children's Oncology Group (COG) stage B, C, D, or E retinoblastoma who have undergone systemic chemotherapy without resolution (meaning either has not had complete response [CR] or has progressed despite systemic chemotherapy) and would have the following treatment options remaining: ** Intra-arterial chemotherapy (IAC) ** Enucleation of one eye ** Local radiation * Patients with non-germline retinoblastoma with unilateral disease who have COG A, B, C, or D tumors * Other patients may be considered on a case by case basis after discussion with pediatric ophthalmology, hematology/oncology, and interventional neuroradiology

Exclusion Criteria

• Opaque or hazy media which precluded visualization of the fundus
• New or recurrent retinoblastoma that can be controlled with other conservative measures such as cryotherapy, thermotherapy, or plaque radiotherapy
• Unilateral COG group E retinoblastoma with very poor visual prognosis as defined by pediatric ophthalmologist
• Patients who would benefit from systemic chemotherapy
• Patients with clinical or radiological evidence suggestive of retinoblastoma invasion of the optic nerve, choroid, sclera, orbit or metastatic sites
• Currently receiving any other investigational agents
• A history of allergic reactions attributed to compounds of similar chemical or biologic composition to melphalan or other agents used in the study
• Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, or cardiac arrhythmia
• Pregnant and/or breastfeeding. Participants of childbearing potential must have a negative pregnancy test within 14 days of study entry
• Patients with human immunodeficiency virus (HIV) are eligible unless their CD4+ T-cell counts are < 350 cells/mcL or they have a history of acquired immunodeficiency syndrome (AIDS)-defining opportunistic infection within the 12 months prior to registration. Concurrent treatment with effective antiretroviral therapy (ART) according to Department of Health and Human Services (DHHS) treatment guidelines is recommended