Zager, Jonathan S. and Orloff, Marlana and Ferrucci, Pier Francesco and Choi, Junsung and Eschelman, David J. and Glazer, Evan S. and Ejaz, Aslam and Howard, J. Harrison and Richtig, Erika and Ochsenreither, Sebastian and Reddy, Sunil A. and Lowe, Michael C. and Beasley, Georgia M. and Gesierich, Anja and Bender, Armin and Gschnell, Martin and Dummer, Reinhard and Rivoire, Michel and Arance, Ana and Fenwick, Stephen William and Sacco, Joseph J. and Haferkamp, Sebastian and Weishaupt, Carsten and John, Johnny and Wheater, Matthew and Ottensmeier, Christian H. (2025) An Open-label, Randomized Study of Melphalan/Hepatic Delivery System Versus Best Alternative Care in Patients with Unresectable Metastatic Uveal Melanoma. ANNALS OF SURGICAL ONCOLOGY, 32 (7). pp. 4976-4988. ISSN 1068-9265, 1534-4681
Full text not available from this repository. (Request a copy)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.
| Item Type: | Article |
|---|---|
| Uncontrolled Keywords: | PERCUTANEOUS HEPATIC PERFUSION; PHASE-II; CRITERIA; Metastatic uveal melanoma; Percutaneous hepatic perfusion; Melphalan/Hepatic Delivery System; Liver-directed therapy; Ocular melanoma |
| Subjects: | 600 Technology > 610 Medical sciences Medicine |
| Divisions: | Medicine > Lehrstuhl für Dermatologie und Venerologie |
| Depositing User: | Dr. Gernot Deinzer |
| Date Deposited: | 31 Mar 2026 09:43 |
| Last Modified: | 31 Mar 2026 09:43 |
| URI: | https://pred.uni-regensburg.de/id/eprint/67838 |
Actions (login required)
 |
View Item |
