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Purpose <p>To assess efficacy and safety in subgroups of patients treated with Melphalan/Hepatic Delivery System (melphalan/HDS), a drug/device combination for liver-directed treatment of metastatic UM (mUM) patients. Previously reported FOCUS study results indicated melphalan/HDS treatment provides a clinically meaningful response rate and favorable benefit-risk ratio in patients with unresectable mUM.</p> Methods <p>Patients with mUM received treatment with melphalan (3.0&#xa0;mg/kg ideal body weight) every 6–8&#xa0;weeks for up to 6 cycles. Post hoc analyses of efficacy and safety were conducted for patient subgroups based on demographic and baseline disease characteristics.</p> Results <p>102 patients with mUM were enrolled; treatment was attempted in 95 patients; 91 patients received treatment. Subgroup analyses showed consistent tumor response regardless of age, sex, geographic region, presence/absence of extrahepatic lesions, and prior therapy. Patients with lower tumor burden had better objective response rate (ORR), progression-free survival (PFS), and overall survival (OS) than those with higher tumor burden (ORR: 51.1 vs. 22.2%, <i>p</i> = 0.008; mPFS: 11.3 vs. 5.8&#xa0;months, <i>p</i> = 0.007; mOS: 26.7 vs. 15.4&#xa0;months, <i>p</i> = 0.008). Patients with 1–25% liver involvement had higher mOS than those with 26–50% liver involvement (22.4 vs. 16.9&#xa0;months; <i>p</i> = 0.030); patients with low or normal lactate dehydrogenase (LDH) had higher mOS than those with elevated LDH (23.5 vs. 15.3&#xa0;months; <i>p</i> = 0.019). The overall safety profile was similar across subgroups without evidence of cumulative toxicity with successive treatment cycles.</p> Conclusion <p>Results demonstrate a favorable benefit-risk profile for melphalan/HDS across clinically relevant subgroups. However, early treatment in patients with low tumor burden may offer best results.</p>

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Subgroup analyses of the phase 3 FOCUS study of melphalan/hepatic delivery system in patients with unresectable metastatic uveal melanoma

  • 期刊论文

  • 出版模式：

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  • 发表日期：

    2025年12月29日

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  • Jonathan S. Zager,
  • Marlana Orloff,
  • Pier Francesco Ferrucci,
  • Junsung Choi,
  • David J. Eschelman,
  • Evan S. Glazer,
  • Aslam Ejaz,
  • Erika Richtig,
  • Sebastian Ochsenreither,
  • Sunil A. Reddy,
  • Michael C. Lowe,
  • Georgia M. Beasley,
  • Anja Gesierich,
  • Martin Gschnell,
  • Reinhard Dummer,
  • Ana Arance,
  • Stephen William Fenwick,
  • Joseph J. Sacco,
  • Johnny John,
  • Matthew Wheater,
  • Christian H. Ottensmeier

摘要

Purpose

To assess efficacy and safety in subgroups of patients treated with Melphalan/Hepatic Delivery System (melphalan/HDS), a drug/device combination for liver-directed treatment of metastatic UM (mUM) patients. Previously reported FOCUS study results indicated melphalan/HDS treatment provides a clinically meaningful response rate and favorable benefit-risk ratio in patients with unresectable mUM.

Methods

Patients with mUM received treatment with melphalan (3.0 mg/kg ideal body weight) every 6–8 weeks for up to 6 cycles. Post hoc analyses of efficacy and safety were conducted for patient subgroups based on demographic and baseline disease characteristics.

Results

102 patients with mUM were enrolled; treatment was attempted in 95 patients; 91 patients received treatment. Subgroup analyses showed consistent tumor response regardless of age, sex, geographic region, presence/absence of extrahepatic lesions, and prior therapy. Patients with lower tumor burden had better objective response rate (ORR), progression-free survival (PFS), and overall survival (OS) than those with higher tumor burden (ORR: 51.1 vs. 22.2%, p = 0.008; mPFS: 11.3 vs. 5.8 months, p = 0.007; mOS: 26.7 vs. 15.4 months, p = 0.008). Patients with 1–25% liver involvement had higher mOS than those with 26–50% liver involvement (22.4 vs. 16.9 months; p = 0.030); patients with low or normal lactate dehydrogenase (LDH) had higher mOS than those with elevated LDH (23.5 vs. 15.3 months; p = 0.019). The overall safety profile was similar across subgroups without evidence of cumulative toxicity with successive treatment cycles.

Conclusion

Results demonstrate a favorable benefit-risk profile for melphalan/HDS across clinically relevant subgroups. However, early treatment in patients with low tumor burden may offer best results.