Pharmacokinetic Analysis of Melphalan after Superselective Ophthalmic Artery Infusion in Preclinical Models and Retinoblastoma Patients

Abstract

Potentially active levels of melphalan after SSOAI were achieved in the vitreous of animals. Low systemic exposure was found in animals and children. Doses greater than 0.48 mg/kg, given for bilateral tandem infusions, were associated with significantly higher plasma levels and increased risk of neutropenia. Synergistic in vitro cytotoxicity between melphalan and topotecan favors combination treatment.