Memorial Cancer Institute/Florida Atlantic University (FAU), Thoracic Oncology, Boca Raton, Florida, USA.
Department of Medicine, University of California, San Francisco, San Francisco, USA.
ESMO Open. 2024 May;9(5):103444. doi: 10.1016/j.esmoop.2024.103444. Epub 2024 May 14.
This post-hoc retrospective study describes long-term patient-reported outcomes (PROs) for REarranged during Transfection (RET)-altered non-small-cell lung cancer (NSCLC), medullary thyroid cancer (MTC), non-MTC thyroid cancer (TC), and tumor agnostic (TA) patients (Data cut-off: January 2023) from the LIBRETTO-001 trial.
Patients completed the European Organization for Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire-Core 30 (QLQ-C30). Patients with MTC also completed a modified version of the Systemic Therapy-Induced Diarrhea Assessment Tool (mSTIDAT). The proportion of patients with improved, stable, or worsened status after baseline was reported. PROs were summarized at 3 years (cycle 37) post-baseline for the NSCLC and MTC cohorts, and at 2 years (cycle 25) post-baseline for the TC and TA cohorts. Time-to-event outcomes (time to first improvement or worsening and duration of improvement) were reported.
The baseline assessment was completed by 200 (63.3%), 209 (70.8%), 50 (76.9%), and 38 (73.1%) patients in the NSCLC, MTC, TC, and TA cohorts, respectively. The total compliance rate was 80%, 82%, 70%, and 85%, respectively. Approximately 75% (NSCLC), 81% (MTC), 75% (TC), and 40% (TA) of patients across all cohorts reported improved or stable QLQ-C30 scores at year 3 (NSCLC and MTC) or year 2 (TC and TA) with continuous selpercatinib use. Across cohorts, the median time to first improvement ranged from 2.0 to 19.4 months, the median duration of improvement ranged from 1.9 to 28.2 months, and the median time to first worsening ranged from 5.6 to 44.2 months. The total compliance rate for the mSTIDAT was 83.7% and the proportion of patients with MTC who reported diarrhea on the mSTIDAT was reduced from 80.8% at baseline to 35.6% at year 3.
A majority of patients with RET-driven cancers improved or remained stable on most QLQ-C30 domains, demonstrating favorable health-related quality of life as measured by the QLQ-C30 during long-term treatment with selpercatinib.
本回顾性研究描述了 LIBRETTO-001 试验中 RET 改变的非小细胞肺癌（NSCLC）、甲状腺髓样癌（MTC）、非 MTC 甲状腺癌（TC）和肿瘤不定型（TA）患者的长期患者报告结局（PROs）（数据截止日期：2023 年 1 月）。
患者完成欧洲癌症研究与治疗组织（EORTC）核心 30 项生活质量问卷（QLQ-C30）。MTC 患者还完成了改良版系统治疗诱导性腹泻评估工具（mSTIDAT）。报告了基线后改善、稳定或恶化的患者比例。NSCLC 和 MTC 队列在基线后 3 年（第 37 个周期），TC 和 TA 队列在基线后 2 年（第 25 个周期）汇总 PROs。报告了首次改善或恶化的时间和改善持续时间等时间事件结局。
NSCLC、MTC、TC 和 TA 队列的基线评估分别完成了 200（63.3%）、209（70.8%）、50（76.9%）和 38（73.1%）例患者。总依从率分别为 80%、82%、70%和 85%。大约 75%（NSCLC）、81%（MTC）、75%（TC）和 40%（TA）的患者在所有队列中，在继续使用塞普替尼治疗的情况下，在第 3 年（NSCLC 和 MTC）或第 2 年（TC 和 TA）时报告了 QLQ-C30 评分的改善或稳定。在各队列中，首次改善的中位时间范围为 2.0 至 19.4 个月，改善的中位持续时间范围为 1.9 至 28.2 个月，首次恶化的中位时间范围为 5.6 至 44.2 个月。mSTIDAT 的总依从率为 83.7%，基线时报告腹泻的 MTC 患者比例为 80.8%，第 3 年时为 35.6%。
在接受塞普替尼长期治疗期间，大多数 RET 驱动的癌症患者在大多数 QLQ-C30 领域得到改善或保持稳定，表明生活质量良好，这可通过 QLQ-C30 进行测量。
