Peter MacCallum Cancer Centre, Melbourne, Victoria, Australia.
Melanoma Institute Australia, North Sydney, New South Wales, Australia.
J Immunother Cancer. 2020 May;8(1). doi: 10.1136/jitc-2019-000440.
The efficacy of immune checkpoint inhibitors (ICI) in metastatic melanoma is well established. However, there are limited data regarding their efficacy in in-transit melanoma (ITM). This study assessed the efficacy of ICI in patients with ITM.
A retrospective review of patients with ITM commenced on an ICI between March 2013 and February 2018 at three tertiary centers in Australia. Patients were excluded if they had previous or synchronous distant metastases. Overall response rate (ORR), progression-free survival (PFS) and overall survival (OS) were based on a composite of radiological and clinical assessments.
Fifty-four patients were included: 27 (50%) female; median age 75 (range 26-94); 12 (22%) stage IIIB, 40 (74%) stage IIIC and 2 (4%) stage IIID; 10 (19%) BRAF mutant. Forty (74%) received single-agent anti-PD-1 (pembrolizumab or nivolumab), 8 (15%) single agent anti-CTLA-4 (ipilimumab), 5 (9%) combination anti-PD-1/anti-CTLA-4 (ipilimumab and nivolumab or pembrolizumab) and 1 (2%) combination anti-PD-L1 (atezolizumab) and MEK inhibitor (cobimetinib). The median follow-up was 15 months (2-46).ORR to ICI was 54%: 14 (26%) complete responses; 15 (28%) partial responses; 9 (17%) stable disease; 16 (30%) progressive disease. Thirteen (46%) responders had only one ITM lesion. ORR was 58% for single-agent anti-PD-1, 38% for single-agent anti-CTLA4 and 40% for anti-PD-1/anti-CTLA-4. The median PFS was 11.7 months (6.6-not reached). 1-year and 2-year PFS were 48% and 39%, respectively,. Fourteen progressed locoregionally and 11 progressed distantly. The median OS was not reached. 1-year and 2-year OS were 85% and 63%, respectively. No clinicopathological features were associated with ORR.
ICI produce objective responses in ITM and should be considered in patients with unresectable ITM or disease recurrence.
免疫检查点抑制剂（ICI）在转移性黑色素瘤中的疗效已得到充分证实。然而，关于其在转移黑色素瘤（ITM）中的疗效的数据有限。本研究评估了 ICI 在 ITM 患者中的疗效。
对 2013 年 3 月至 2018 年 2 月期间在澳大利亚的三个三级中心接受 ICI 治疗的 ITM 患者进行了回顾性研究。如果患者有先前或同步的远处转移，则将其排除在外。总缓解率（ORR）、无进展生存期（PFS）和总生存期（OS）是基于影像学和临床评估的综合结果。
共纳入 54 例患者：27 例（50%）为女性；中位年龄 75 岁（范围 26-94 岁）；12 例（22%）为 IIIB 期，40 例（74%）为 IIIC 期，2 例（4%）为 IIID 期；10 例（19%）为 BRAF 突变型。40 例（74%）接受了单药抗 PD-1（pembrolizumab 或 nivolumab）治疗，8 例（15%）接受了单药抗 CTLA-4（ipilimumab）治疗，5 例（9%）接受了抗 PD-1/抗 CTLA-4 联合治疗（ipilimumab 和 nivolumab 或 pembrolizumab），1 例（2%）接受了抗 PD-L1（atezolizumab）和 MEK 抑制剂（cobimetinib）联合治疗。中位随访时间为 15 个月（2-46 个月）。ICI 的总缓解率为 54%：14 例（26%）完全缓解；15 例（28%）部分缓解；9 例（17%）病情稳定；16 例（30%）疾病进展。13 例（46%）缓解者仅有一处 ITM 病变。单药抗 PD-1 的缓解率为 58%，单药抗 CTLA4 的缓解率为 38%，抗 PD-1/抗 CTLA-4 的缓解率为 40%。中位 PFS 为 11.7 个月（6.6-未达到）。1 年和 2 年 PFS 分别为 48%和 39%。14 例患者出现局部区域进展，11 例患者出现远处转移。中位 OS 未达到。1 年和 2 年 OS 分别为 85%和 63%。无临床病理特征与 ORR 相关。
ICI 在 ITM 中产生客观缓解，应考虑在不可切除的 ITM 或疾病复发的患者中使用。
