Intra-arterial Melphalan in Treating Younger Patients With Unilateral Retinoblastoma

This pilot clinical trial studies whether unilateral group D retinoblastoma, or retinoblastoma affecting one eye that has spread to the inner jelly like part of the eye, can be treated with a new technique for delivering chemotherapy directly into the blood vessel that supplies the affected eye. This new technique is called intra-arterial injection. Giving melphalan via intra-arterial injection may make it less likely that children will need surgery to remove the eye and may reduce the amount of treatment side effects.

PRIMARY OBJECTIVES:

I. To study the feasibility of delivering melphalan directly into the ophthalmic artery in children with newly diagnosed unilateral group D retinoblastoma, who would otherwise be considered for enucleation.

SECONDARY OBJECTIVES:

I. To estimate the ocular salvage rate after treatment with intra-arterial melphalan in children with newly diagnosed unilateral retinoblastoma with group D disease.

II. To evaluate the toxicities and adverse events associated with delivering multiple doses of intra-arterial chemotherapy.

III. To evaluate vision outcomes in children treated with intra-arterial chemotherapy.

IV. To monitor the rate of the development of metastatic disease while on protocol therapy.

TERTIARY OBJECTIVES:

I. To evaluate the effects of intra-arterial therapy on the histopathology of eyes enucleated for progression.

OUTLINE:

Patients receive melphalan intra-arterially (IA) on day 1. Treatment repeats every 28 days for up to 3 courses in the absence of disease progression or unacceptable toxicity.

After completion of study treatment, patients are followed up every 3 months for 1 year, every 4 months for 1 year, every 6 months for 1 year, and then periodically for 2 years.

Eligibility Criteria:
Inclusion Criteria:

* Newly diagnosed patients with unilateral group D retinoblastoma
* Magnetic resonance imaging (MRI) (or computed tomography \[CT\] if MRI is not available) of the brain must be performed within 14 days prior to study entry
* Diagnostic examination under anesthesia (EUA) must be performed within 14 days prior to study entry
* Rapid central review confirmation of group D disease based on RetCam images from diagnostic EUA must be obtained before starting treatment
* Patients must have a performance status corresponding to Eastern Cooperative Oncology Group (ECOG) scores of 0, 1 or 2
* Patients must have a life expectancy of \>= 8 weeks
* Patients must have adequate renal function, defined as:

  * Creatinine clearance or radioisotope glomerular filtration rate (GFR) \>= 70 mL/min/1.73 m\^2 or
  * A serum creatinine based on age/gender as follows:

    * 1 month to \< 6 months: 0.4 mg/dL
    * 6 months to \< 1 year: 0.5 mg/dL
    * 1 to \< 2 years: 0.6 mg/dL
    * 2 to \< 6 years: 0.8 mg/dL
    * 6 to \< 10 years: 1 mg/dL
    * 10 to \< 13 years: 1.2 mg/dL
    * 13 to \< 16 years: 1.5 mg/dL (male); 1.4 mg/dL (female)
    * \>= 16 years: 1.7 mg/dL (male); 1.4 mg/dL (female)
* Total bilirubin =\< 1.5 x upper limit of normal (ULN) for age
* Serum glutamate pyruvate transaminase (SGPT) (alanine aminotransferase \[ALT\]) \< 2.5 x upper limit of normal (ULN) for age

Exclusion Criteria:

* Patients with bilateral disease
* Unilateral retinoblastoma with group A, B, C, or E eyes
* Prior chemotherapy or radiation therapy for this disease (laser and cryotherapy are allowed and are not considered exclusion criteria)
* Clinical or neuroimaging evidence of extraocular disease or orbital optic nerve involvement