乳腺癌相关淋巴水肿（BCRL）是一种缺乏治疗的常见并发症。 Lymfactin® 是一种基于 5 型腺病毒的基因治疗和促淋巴管生成生长因子载体，可诱导血管内皮生长因子 C (VEGF-C) 表达。我们的目的是评估 Lymfactin® 联合血管化淋巴结转移 (VLNT) 的治疗效果。这项 II 期、双盲、安慰剂对照、随机多中心研究评估了 Lymfactin® 联合 VLNT 的疗效和安全性。主要终点是水肿体积、生活质量 (LyQoLI) 和淋巴闪烁扫描。记录所有不良事件。进行了协方差重复测量分析的混合模型。这项研究是之前 I 期 Lymfactin® 研究的延续。2018 年 6 月至 2019 年 12 月期间招募了 39 名 BCRL 患者，并随机接受 Lymfactin® (n = 20) 或安慰剂 (n = 19) 治疗。主要终点显示，与基线相比，两组 VLNT 均具有积极作用，但 12 个月时组间无统计学差异。此外，与安慰剂组相比，Lymfactin® 组测量皮肤间质液水平的组织介电常数比有更大的改善 (p = 0.020)。各组之间没有检测到不良事件的差异。这项研究是为数不多的在前瞻性临床多中心环境中客观显示 VLNT 积极作用的研究之一。这也是有史以来第一项随机前瞻性临床研究，显示药物治疗对淋巴水肿的水肿具有定量的积极作用，尽管未能显示主要结果指标各组之间的差异。版权所有 © 2024 美国整形外科医生协会。

Breast cancer-related lymphedema (BCRL) is a common complication lacking medical treatment. Lymfactin® is an adenovirus type 5-based gene therapy and prolymphangiogenic growth factor vector that induces vascular endothelial growth factor C (VEGF-C) expression. Our aim was to evaluate the therapeutic effect of Lymfactin® with vascularized lymph node transfer (VLNT).This Phase II, double-blind, placebo-controlled, randomized multicenter study evaluated the efficacy and safety of Lymfactin® in combination with VLNT. The primary endpoints were edema volume, quality of life (LyQoLI), and lymphoscintigraphy. All adverse events were recorded. A mixed model of repeated measures analysis of covariance was performed. This study was a continuation of a previous Phase I Lymfactin® study.Thirty-nine patients with BCRL were recruited between June 2018 and December 2019 and randomized to receive either Lymfactin® (n = 20) or placebo (n = 19). The primary endpoints showed a positive effect of VLNT in both groups compared to the baseline, but without statistical differences between groups at 12 months. Additionally, greater improvements were observed in the tissue dielectric constant ratios measuring skin interstitial fluid levels in the Lymfactin® group compared to the placebo group (p = 0.020). No differences in adverse events were detected between the groups.This study was one of the few studies to objectively show a positive effect of VLNT in a prospective clinical multicenter setting. It was also the first-ever randomized prospective clinical study showing a quantitatively positive effect of a medical therapy on the edema of lymphedema although failing to show differences between groups in primary outcome measures.Copyright © 2024 by the American Society of Plastic Surgeons.