在II期LIBRETTO-321试验中，接受塞尔帕替尼治疗的中国晚期融合阳性非小细胞肺癌、甲状腺癌和RET突变型甲状腺髓样癌患者的患者报告结局。

Patient-reported outcomes following selpercatinib treatment in Chinese patients with advanced fusion-positive non-small-cell lung cancer and thyroid cancer, and -mutant medullary thyroid cancer in the phase II LIBRETTO-321 trial.

作者信息

Lu Shun, Zheng Xiangqian, Sun Yuping, Huang Dingzhi, Wu Lin, Ji Qinghai, Zhou Chengzhi, Zhou Jianying, Guo Ye, Ge Minghua, Ding Ding, Shao Jingxin, Zhang Wanli, Gao Ming, Cheng Ying

机构信息

Department of Medical Oncology, Shanghai Chest Hospital, School of Medicine, Shanghai Jiao Tong University, Shanghai 200030, China.

Department of Thyroid and Neck Tumor, Tianjin Medical University Cancer Institute and Hospital, National Clinical Research Center for Cancer, Key Laboratory of Cancer Prevention and Therapy, Tianjin's Clinical Research Center for Cancer, Tianjin, China.

出版信息

Ther Adv Med Oncol. 2023 Aug 25;15:17588359231189429. doi: 10.1177/17588359231189429. eCollection 2023.

DOI:10.1177/17588359231189429

PMID:37655205

原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC10467255/

Abstract

BACKGROUND

Patient-reported outcomes (PROs) are increasingly becoming an important part of clinical trials as they are helpful in analyzing the safety and efficacy of treatment in chronic diseases like cancer.

OBJECTIVES

We report PROs and health-related quality of life (HRQoL) with selpercatinib treatment among Chinese patients with rearranged in transfection () fusion-positive non-small-cell lung cancer (NSCLC), fusion-positive thyroid cancer (TC), and -mutant medullary TC (MTC) as an exploratory analysis of the LIBRETTO-321 trial.

DESIGN

A total of 77 patients (47 fusion-positive NSCLC, 1 fusion-positive TC, and 29 -mutant MTC) were enrolled. Compliance for European Organization for Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire-Core 30 (QLQ-C30) was 100% at baseline and >90% at each time point.

METHODS

PROs were assessed using the EORTC QLQ-C30, and a bowel diary assessment for MTC patients with baseline diarrhea using the Systemic Therapy-Induced Diarrhea Assessment Tool. Data were collected at pre-dose; every 8 weeks from cycle 3; and every 12 weeks after cycle 13. A >10-point change from baseline was considered clinically meaningful. PRO changes were summarized through cycle 13.

RESULTS

Most patients with NSCLC or MTC showed improvement or remained stable on the global health status and functional subscales. For global health status, 47.4% of NSCLC and MTC patients showed definite improvement with only 19.7% showing definite worsening. For functional subscales, less than 30% of the patients showed definite worsening. For symptom subscales, more than 64% of the patients either improved or remained stable for the symptoms. For MTC patients with bowel diary assessment ( = 5), there was no severity or worsening from baseline in the diarrheal episodes observed during treatment with selpercatinib.

CONCLUSION

The study demonstrated favorable PROs in Chinese patients with fusion-positive NSCLC, TC, and -mutant MTC treated with selpercatinib. HRQoL was improved or stable as assessed by EORTC QLQ-30.

TRAIL REGISTRATION

This study was registered at ClinicalTrials.gov (https://clinicaltrials.gov/ct2/show/NCT04280081) ClinicalTrials.gov Identifier: NCT04280081.

摘要

背景

患者报告结局（PROs）日益成为临床试验的重要组成部分，因为它们有助于分析癌症等慢性病治疗的安全性和有效性。

目的

我们报告了接受塞尔帕替尼治疗的中国转染重排（RET）融合阳性非小细胞肺癌（NSCLC）、RET融合阳性甲状腺癌（TC）和RET突变型甲状腺髓样癌（MTC）患者的PROs及健康相关生活质量（HRQoL），作为LIBRETTO-321试验的探索性分析。

设计

共纳入77例患者（47例RET融合阳性NSCLC、1例RET融合阳性TC和29例RET突变型MTC）。欧洲癌症研究与治疗组织（EORTC）生活质量问卷核心30（QLQ-C30）的基线依从率为100%，各时间点均>90%。

方法

使用EORTC QLQ-C30评估PROs，对于基线时有腹泻的MTC患者，使用全身治疗引起的腹泻评估工具进行肠道日记评估。在给药前、第3周期开始每8周以及第13周期后每12周收集数据。与基线相比变化>10分被认为具有临床意义。总结了第13周期的PRO变化情况。

结果

大多数NSCLC或MTC患者在总体健康状况和功能子量表上显示改善或保持稳定。对于总体健康状况，47.4%的NSCLC和MTC患者显示明显改善，只有19.7%的患者显示明显恶化。对于功能子量表，不到30%的患者显示明显恶化。对于症状子量表，超过64%的患者症状改善或保持稳定。对于接受肠道日记评估的MTC患者（n = 5），在接受塞尔帕替尼治疗期间观察到的腹泻发作严重程度未超过基线水平或恶化。