A multi-institutional study led by Moffitt Cancer Center has demonstrated that percutaneous hepatic perfusion using a melphalan hepatic delivery system offers significant benefits for patients with metastatic uveal melanoma, a rare and aggressive eye cancer that has spread to the liver. Unlike traditional chemotherapy, this innovative approach delivers high doses of the chemotherapy drug melphalan directly to the liver via a series of catheters and balloons, isolating the organ and filtering the drug before it enters the rest of the body. This targeted delivery minimizes systemic exposure and associated side effects. The treatment received FDA approval in August 2023 following promising results from the phase 3 FOCUS trial .
In the FOCUS trial, patients treated with the melphalan hepatic delivery system experienced a median progression-free survival of 9.1 months, compared to 3.3 months for those receiving standard care. The objective response rate was 27.5%, nearly three times higher than the 9.4% observed in the control group. Additionally, the disease control rate increased from 46.9% to 80.0%, and median overall survival improved to 18.5 months versus 14.5 months with conventional treatments. While some patients experienced side effects related to blood cell counts, these were generally manageable with standard outpatient care. Lead author Dr. Jonathan Zager emphasized that this treatment provides a new option for patients, offering improved outcomes without compromising quality of life .
