Department of Radiology, Leiden University Medical Center, Leiden, The Netherlands.
Department of Surgery, Leiden University Medical Center, Leiden, The Netherlands.
Ann Surg Oncol. 2021 Feb;28(2):1130-1141. doi: 10.1245/s10434-020-08741-x. Epub 2020 Aug 5.
Ocular melanoma is the most common primary intraocular malignancy and has a very poor prognosis once liver metastases occur. The aim of this study was to prospectively assess the efficacy and safety of percutaneous hepatic perfusion with melphalan (M-PHP) using the new second-generation (GEN 2) hemofiltration system in patients with ocular melanoma metastases confined to the liver.
Prospective, single-center, single-arm, phase II study including patients with unresectable ocular melanoma metastases confined to the liver. Treatment consisted of two M-PHP procedures at 6-8 weeks interval. Procedures were performed using the CHEMOSAT (GEN 2) system with 3 mg/kg melphalan. Primary endpoints were overall response rate (ORR) and best overall response (BOR). Secondary endpoints included overall survival (OS), progression-free survival (PFS), hepatic PFS (hPFS), and safety.
Sixty-four M-PHP procedures were performed in 35 patients between February 2014 and June 2017. The ORR was 72%. BOR was as follows: complete response in 3%, partial response in 69%, stable disease in 13%, and progressive disease in 16%. There was no treatment-related mortality. Fourteen serious adverse events occurred. At a median follow-up of 19.1 months (range 5.6-69.5), median OS was 19.1 months and was significantly longer in responders than in nonresponders (27.5 vs. 11.9 months, p < 0.001). The 1- and 2-year OS was 77% and 43%, respectively. PFS and hPFS were 7.6 and 11.2 months, respectively.
M-PHP using the GEN 2 filter can achieve a high ORR and prolonged survival in patients with liver-only ocular melanoma metastases.
眼部黑色素瘤是最常见的原发性眼内恶性肿瘤，一旦发生肝转移，预后极差。本研究旨在前瞻性评估使用第二代（GEN 2）血液滤过系统行肝内灌注丝裂霉素（M-PHP）治疗局限于肝脏的眼部黑色素瘤转移患者的疗效和安全性。
本研究为前瞻性、单中心、单臂、Ⅱ期研究，纳入无法切除的局限于肝脏的眼部黑色素瘤转移患者。治疗包括 6-8 周时行两次 M-PHP 治疗。采用 CHEMOSAT（GEN 2）系统，以 3mg/kg 丝裂霉素行 M-PHP 治疗。主要终点为总缓解率（ORR）和最佳总缓解（BOR）。次要终点包括总生存期（OS）、无进展生存期（PFS）、肝脏无进展生存期（hPFS）和安全性。
2014 年 2 月至 2017 年 6 月，共纳入 35 例患者，行 64 次 M-PHP 治疗。ORR 为 72%。BOR 为完全缓解 3%，部分缓解 69%，稳定 13%，进展 16%。无治疗相关死亡。共发生 14 例严重不良事件。中位随访时间 19.1 个月（5.6-69.5 个月），中位 OS 为 19.1 个月，缓解者的 OS 明显长于未缓解者（27.5 个月 vs. 11.9 个月，P<0.001）。1 年和 2 年 OS 分别为 77%和 43%。PFS 和 hPFS 分别为 7.6 个月和 11.2 个月。
采用 GEN 2 过滤器行 M-PHP 治疗局限于肝脏的眼部黑色素瘤转移患者，可获得较高的 ORR 和延长的生存。
