FOCUS

FOCUS was an open-label phase 3 trial of melphalan delivered by percutaneous hepatic perfusion with the Hepatic Delivery System in patients with unresectable metastatic uveal melanoma and liver-dominant disease. Of 102 patients enrolled, treatment was attempted in 95 and 91 received it. Melphalan 3 mg per kg ideal body weight was given once every 6 to 8 weeks for up to six cycles. The primary endpoint was objective response rate by independent central review, judged against the upper confidence bound of a benchmark meta-analysis. The registry's outcome definitions date events from the eligibility date for PHP-OCM-301A patients and from the randomisation date for PHP-OCM-301 patients, the trace of an earlier randomised phase of the same study.

In the 91 treated patients the objective response rate was 36.3 percent (95% CI 26.44 to 47.01), including complete responses in 7.7 percent. The lower confidence bound exceeded the 8.3 percent benchmark, so the study met its primary endpoint. Median duration of response was 14 months, median overall survival 20.5 months (80 percent alive at 1 year) and median progression-free survival 9 months (65 percent progression-free at 6 months). The most common serious treatment-emergent adverse events were thrombocytopenia (15.8 percent) and neutropenia (10.5 percent), and there were no treatment-related deaths (Annals of Surgical Oncology 2024). The registry results section adds a disease control rate of 73.6 percent and gives the duration-of-response median as 14.00 months (95% CI 8.31 to 17.74).