ARET2121: Testing whether adding a medicine called melphalan, given directly into the eye, can make early treatment more effective for treatment with retinoblastomaIntravitreal Melphalan for Intraocular Retinoblastoma

Inclusion

• Patient must be < 18 years of age at enrollment

• Patient must have newly diagnosed intraocular (localized) retinoblastoma and meet one of the following criteria:

  • Unilateral Group D retinoblastoma with vitreous seeding; OR
  • Bilateral retinoblastoma with worst eye Group D, with vitreous seeding present and the contralateral eye is Group A-C; OR
  • Bilateral retinoblastoma with one Group D eye with vitreous seeding and one Group E eye where the Group E eye has been enucleated prior to any therapy. Note exclusion for high-risk features
• Patients must have a performance status corresponding to Eastern Cooperative Oncology Group (ECOG) scores of 0, 1 or 2. Use Karnofsky for patients > 16 years of age and Lansky for patients =<16 years of age
• Peripheral absolute neutrophil count (ANC) >= 750/uL (must be performed within 7 days prior to enrollment unless otherwise indicated)
• Platelet count >= 75,000/uL (transfusion independent) (must be performed within 7 days prior to enrollment)

• A serum creatinine based on age/gender as follows (must be performed within 7 days prior to enrollment; must be repeated prior to the start of protocol therapy if > 7 days have elapsed from their most recent prior assessment):

  • 1 month to < 6 months = 0.4 (male and female)
  • 6 months to < 1 year = 0.5 (male and female)
  • 1 to < 2 years = 0.6 (male and female)
  • 2 to < 6 years = 0.8 (male and female)
  • 6 to < 10 years = 1.0 (male and female)
  • 10 to < 13 years = 1.2 (male and female)
  • 13 to < 16 years = 1.5 (male) and 1.4 (female)
  • >= 16 years = 1.7 (male) and 1.4 (female) OR - a 24-hour urine Creatinine clearance >= 70 mL/min/1.73 m^2 OR - a glomerular filtration rate (GFR) >= 70 mL/min/1.73 m^2. GFR must be performed using direct measurement with a nuclear blood sampling method OR direct small molecule clearance method (iothalamate or other molecule per institutional standard)

  • Note: Estimated GFR (eGFR) from serum creatinine, cystatin C or other estimates are not acceptable for determining eligibility

    • For patients < 1 month of age, serum creatinine levels must be < 1.5 x the treating institution's creatinine upper limit of normal (ULN) for patients < 1 month of age or the creatinine clearance or radioisotope GFR must be >= 70 mL/min/1.73 m^2
• Total bilirubin =< 1.5 x upper limit of normal (ULN) for age (must be performed within 7 days prior to enrollment; must be repeated prior to the start of protocol therapy if > 7 days have elapsed from their most recent prior assessment)

• Serum glutamate pyruvate transaminase (SGPT) (alanine aminotransferase [ALT]) =< 135 U/L (must be performed within 7 days prior to enrollment; must be repeated prior to the start of protocol therapy if > 7 days have elapsed from their most recent prior assessment)

  • Note: For the purpose of this study, the ULN for SGPT (ALT) has been set to the value of 45 U/L