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

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

doi:10.1177/17588359231189429

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

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

Authors

Shun Lu¹, Xiangqian Zheng², Yuping Sun³, Dingzhi Huang⁴, Lin Wu⁵, Qinghai Ji⁶, Chengzhi Zhou⁷, Jianying Zhou⁸, Ye Guo⁹, Minghua Ge¹⁰, Ding Ding¹¹, Jingxin Shao¹¹, Wanli Zhang¹¹, Ming Gao^{12

13

14}, Ying Cheng¹⁵

Affiliations

¹ Department of Medical Oncology, Shanghai Chest Hospital, School of Medicine, Shanghai Jiao Tong University, Shanghai 200030, China.
² 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.
³ Department of Oncology, Jinan Central Hospital Affiliated to Shandong University, Jinan, China.
⁴ Department of Thoracic Oncology, Tianjin Medical University Cancer Institute and Hospital, Tianjin, China.
⁵ Department of Thoracic Medical Oncology, Hunan Cancer Hospital, The Affiliated Cancer Hospital of Xiangya School of Medicine, Central South University, Changsha, China.
⁶ Department of Head and Neck Surgery, Fudan University Shanghai Cancer Center, Shanghai, China.
⁷ Pulmonary and Critical Care Medicine, National Center for Respiratory Medicine, National Clinical Research Center for Respiratory Disease, State Key Laboratory of Respiratory Disease, Guangzhou Institute of Respiratory Health, The First Affiliated Hospital of Guangzhou Medical University, Guangzhou, China.
⁸ Department of Respiratory Diseases, First Affiliated Hospital, College of Medicine, Zhejiang University, Hangzhou, China.
⁹ Department of Medical Oncology, Shanghai East Hospital, Tongji University School of Medicine, Shanghai, China.
¹⁰ Department of Head, Neck and Thyroid Surgery, Zhejiang Provincial People's Hospital, People's Hospital of Hangzhou Medical College, Hangzhou, China.
¹¹ Eli Lilly and Company, Shanghai, China.
¹² 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 300040, China.
¹³ Department of Thyroid and Breast Surgery, Tianjin Union Medical Center, Tianjin 300121, China.
¹⁴ Tianjin Key Laboratory of General Surgery Inconstruction, Tianjin Union Medical Center, Tianjin 300121, China.
¹⁵ Department of Thoracic Oncology, Jilin Cancer Hospital, Changchun 130012, Jilin, China.

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 (RET) fusion-positive non-small-cell lung cancer (NSCLC), RET fusion-positive thyroid cancer (TC), and RET-mutant medullary TC (MTC) as an exploratory analysis of the LIBRETTO-321 trial.

Design: A total of 77 patients (47 RET fusion-positive NSCLC, 1 RET fusion-positive TC, and 29 RET-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 (n = 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 RET fusion-positive NSCLC, TC, and RET-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.

Keywords: HRQoL; LIBRETTO-321; PROs; RET fusion-positive NSCLC; RET fusion-positive thyroid cancer; RET-mutant MTC; Selpercatinib.

Associated data

ClinicalTrials.gov/NCT04280081