Intra-arterial chemotherapy for the treatment of intraocular retinoblastoma
The purpose of this study is to show that chemotherapy delivered directly through the artery supplying the eye (ophthalmic artery) to patients with retinoblastoma is a safe and effective treatment alternative to conventional systemic chemotherapy, external beam radiation, and surgical removal of the eye. Delivering the chemotherapeutic agent in the arterial system through the ophthalmic artery transforms the treatment of retinoblastoma from systemic chemotherapy to local chemotherapy. Administration of the drug directly to the targeted site thus avoids the complications and adverse events associated with toxicity from systemic, rather than local, chemotherapy.
INCLUSION CRITERIA: 1) Patients newborn to 18 years old. 2) Patients with intraocular retinoblastoma, unilateral or bilateral, who would be treated either by systemic chemotherapy, EBR, or enucleation would be considered for this study. EXCLUSION CRITERIA: 1) Patients over the age of 18. 2) Patients with small, localized intraocular Rb amenable to focal therapy (laser or cryotherapy). 3) Patients with extraocular disease evident on MRI (extension into the optic nerve), massive choroidal/uveal invasion (grade IIC or IID per ARET0332) or disease outside the globe evident on MRI or physical examination. 4) Documented hypercoagulable disorders or vasculopathies. 5) Laboratory exclusion criteria: GFR less than 60 mL/min/1.73 m2
DRUG: Melphalan hydrochloride Drug administered intra-arterially (injection in the artery). STANDARD DOSE: 2.5mg (3-6 month old) 3.0 mg (6-12 month old) 4.0 mg (1-3 year old) 5.0 mg ( greater than 3 years old) DOSE MODIFICATION: Decrease standard dose by 25% if there are signs of toxicity. Increase the dose by 25% if there is inadequate tumor response. FREQUENCY: 2 treatment cycles at 3-4 week intervals, with 3rd treatment cycle if tumor requires it. Dose not to exceed 0.5mg/kg, per treatment cycle.
08/21/2014 04:02 AM