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. (2024) Efficacy and Safety of the Melphalan/Hepatic Delivery System in Patients with Unresectable Metastatic Uveal Melanoma: Results from an Open-Label, Single-Arm, Multicenter Phase 3 Study. ANNALS OF SURGICAL ONCOLOGY, 31 (8). pp. 5340-5351. ISSN 1068-9265, 1534-4681
Full text not available from this repository. (Request a copy)Abstract
BackgroundUveal melanoma (UM) has a poor prognosis once liver metastases occur. The melphalan/Hepatic Delivery System (melphalan/HDS) is a drug/device combination used for liver-directed treatment of metastatic UM (mUM) patients. The purpose of the FOCUS study was to assess the efficacy and safety of melphalan/HDS in patients with unresectable mUM.MethodsEligible patients with mUM received treatment with melphalan (3.0 mg/kg ideal body weight) once every 6 to 8 weeks for a maximum of six cycles. The primary end point was the objective response rate (ORR). The secondary end points included duration of response (DOR), overall survival (OS), and progression-free survival (PFS).ResultsThe study enrolled 102 patients with mUM. Treatment was attempted in 95 patients, and 91 patients received treatment. In the treated population (n = 91), the ORR was 36.3 % (95 % confidence interval [CI], 26.44-47.01), including 7.7 % of patients with a complete response. Thus, the study met its primary end point because the lower bound of the 95 % CI for ORR exceeded the upper bound (8.3 %) from the benchmark meta-analysis. The median DOR was 14 months, and the median OS was 20.5 months, with an OS of 80 % at 1 year. The median PFS was 9 months, with a PFS of 65 % at 6 months. The most common serious treatment-emergent adverse events were thrombocytopenia (15.8 %) and neutropenia (10.5 %), treated mostly on an outpatient basis with observation. No treatment-related deaths were observed.ConclusionTreatment with melphalan/HDS provides a clinically meaningful response rate and demonstrates a favorable benefit-risk profile in patients with unresectable mUM (study funded by Delcath; ClinicalTrials.gov identifier: NCT02678572; EudraCT no. 2015-000417-44).
| Item Type: | Article |
|---|---|
| Uncontrolled Keywords: | PERCUTANEOUS HEPATIC PERFUSION; CRITERIA |
| Subjects: | 600 Technology > 610 Medical sciences Medicine |
| Divisions: | Medicine > Lehrstuhl für Dermatologie und Venerologie |
| Depositing User: | Dr. Gernot Deinzer |
| Date Deposited: | 16 Dec 2025 09:29 |
| Last Modified: | 16 Dec 2025 09:29 |
| URI: | https://pred.uni-regensburg.de/id/eprint/65474 |
Actions (login required)
 |
View Item |
