Division of New Drugs and Early Drug Development, European Institute of Oncology, IRCCS, Milan, Italy.
Department of Oncology and Hemato-Oncology, University of Milan, Milan, Italy.
Oncologist. 2024 Jan 5;29(1):75-83. doi: 10.1093/oncolo/oyad212.
Baseline tumor size (BTS) has been associated with outcomes in patients with cancer treated with immunotherapy. However, the prognostic impact of BTS on patients receiving targeted therapies (TTs) remains undetermined.
We reviewed data of patients with advanced solid tumors consecutively treated within early-phase clinical trials at our institution from 01/2014 to 04/2021. Treatments were categorized as immunotherapy-based or TT-based (biomarker-matched or not). BTS was calculated as the sum of RECIST1.1 baseline target lesions.
A total of 444 patients were eligible; the median BTS was 69 mm (IQR 40-100). OS was significantly longer for patients with BTS lower versus higher than the median (16.6 vs. 8.2 months, P < .001), including among those receiving immunotherapy (12 vs. 7.5 months, P = .005). Among patients receiving TT, lower BTS was associated with longer PFS (4.7 vs. 3.1 months, P = .002) and OS (20.5 vs. 9.9 months, P < .001) as compared to high BTS. However, such association was only significant among patients receiving biomarker-matched TT, with longer PFS (6.2 vs. 3.3 months, P < .001) and OS (21.2 vs. 6.7 months, P < .001) in the low-BTS subgroup, despite a similar ORR (28% vs. 22%, P = .57). BTS was not prognostic among patients receiving unmatched TT, with similar PFS (3.7 vs. 4.4 months, P = .30), OS (19.3 vs. 11.8 months, P = .20), and ORR (33% vs. 28%, P = .78) in the 2 BTS groups. Multivariate analysis confirmed that BTS was independently associated with PFS (P = .03) and OS (P < .001) but not with ORR (P = .11).
Higher BTS is associated with worse survival outcomes among patients receiving biomarker-matched, but not biomarker-unmatched TT.
基线肿瘤大小（BTS）与接受免疫疗法治疗的癌症患者的预后相关。然而，BTS 对接受靶向治疗（TTs）的患者的预后影响仍未确定。
我们回顾了 2014 年 1 月至 2021 年 4 月期间在我们机构接受早期临床试验连续治疗的晚期实体瘤患者的数据。治疗分为基于免疫疗法或基于 TT（有或无生物标志物匹配）。BTS 计算为 RECIST1.1 基线靶病灶的总和。
共有 444 名患者符合条件；中位 BTS 为 69mm（IQR 40-100）。BTS 低于或高于中位数的患者 OS 显著更长（16.6 与 8.2 个月，P<.001），包括接受免疫治疗的患者（12 与 7.5 个月，P=.005）。在接受 TT 的患者中，BTS 较低与 PFS（4.7 与 3.1 个月，P=.002）和 OS（20.5 与 9.9 个月，P<.001）较长相关，与 BTS 较高相比。然而，这种关联仅在接受生物标志物匹配 TT 的患者中具有显著性，低 BTS 亚组的 PFS（6.2 与 3.3 个月，P<.001）和 OS（21.2 与 6.7 个月，P<.001）较长，尽管 ORR 相似（28%与 22%，P=.57）。在接受不匹配 TT 的患者中，BTS 无预后意义，两组间 PFS（3.7 与 4.4 个月，P=.30）、OS（19.3 与 11.8 个月，P=.20）和 ORR（33%与 28%，P=.78）相似。多变量分析证实，BTS 与 PFS（P=.03）和 OS（P<.001）独立相关，但与 ORR 无关（P=.11）。
在接受生物标志物匹配而非生物标志物不匹配 TT 的患者中，较高的 BTS 与较差的生存结果相关。
