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SHR Neuro Krebs Kardio Lipid Stoffw Microb

Zager, JS; Orloff, M; Ferrucci, PF; Choi, J; Eschelman, DJ; Glazer, ES; Ejaz, A; Howard, JH; Richtig, E; Ochsenreither, S; Reddy, SA; Lowe, MC; Beasley, GM; Gesierich, A; Bender, A; Gschnell, M; Dummer, R; Rivoire, M; Arance, A; Fenwick, SW; Sacco, JJ; Haferkamp, S; Weishaupt, C; John, J; Wheater, M; Ottensmeier, CH.
An Open-label, Randomized Study of Melphalan/Hepatic Delivery System Versus Best Alternative Care in Patients with Unresectable Metastatic Uveal Melanoma.
Ann Surg Oncol. 2025; Doi: 10.1245/s10434-025-17231-x
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Abstract:
BACKGROUND: Metastatic uveal melanoma (mUM) has a poor prognosis, with liver metastases typically presenting a therapeutic challenge. Melphalan/Hepatic Delivery System (Melphalan/HDS) is a drug/medical device combination used for liver-directed treatment of unresectable mUM patients. This study assessed efficacy and safety of Melphalan/HDS versus best alternative care (BAC). METHODS: Eligible patients with unresectable mUM were randomized (1:1) to receive Melphalan/HDS (3 mg/kg ideal body weight) once every 6 to 8 weeks for a maximum of 6 cycles or BAC. Due to slow enrollment and patient reluctance to receive BAC treatment, the study design was amended to a single-arm Melphalan/HDS study, and all efficacy analyses of the randomized study were treated as exploratory. RESULTS: The study enrolled 85 patients. Eligible patients were randomized to receive Melphalan/HDS (n = 43) or BAC (n = 42), and 72 patients received study treatment (Melphalan/HDS [n = 40]; BAC [n = 32]). Exploratory analyses of efficacy endpoints showed numerical differences consistently favoring the Melphalan/HDS arm versus BAC (median overall survival: 18.5 vs. 14.5 months; median progression-free survival: 9.1 vs. 3.3 months; objective response rate: 27.5% vs. 9.4%; and disease control rate: 80.0% vs. 46.9%). Serious adverse events (SAEs) occurred in 51.2% of Melphalan/HDS and in 21.9% of BAC patients. The most common (>5%) SAEs included thrombocytopenia (19.5%), neutropenia (9.8%), leukopenia (9.8%) and febrile neutropenia (7.3%) in Melphalan/HDS patients and cholecystitis, nausea and vomiting (6.3% each) in BAC patients. No treatment-related deaths were observed. CONCLUSION: Treatment with Melphalan/HDS shows clinically meaningful efficacy and demonstrates a favorable benefit-risk profile in patients with unresectable mUM as compared to BAC.

Find related publications in this database (Keywords)
Metastatic uveal melanoma
Percutaneous hepatic perfusion
Melphalan/Hepatic Delivery System
Liver-directed therapy
Ocular melanoma
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