Patient-reported outcomes with selpercatinib treatment in patients with RET-driven cancers in the phase I/II LIBRETTO-001 trial - Original Research

Sep 21, 2026

Uploaded by Eliana

Post-hoc retrospective analysis evaluates long-term patient-reported outcomes for RET-altered non-small-cell lung cancer, medullary thyroid cancer, non-medullary thyroid cancer, and tumor-agnostic patients from the phase I/II LIBRETTO-001 trial (data cut-off January 2023). EORTC QLQ-C30 and, for MTC, modified mSTIDAT quantify baseline status and changes after selpercatinib. Results summarize improvement, stability, or worsening and time-to-first improvement or worsening with duration metrics at 2 and 3 years.

LanguageEnglish

CategoryResearch & Report

FormatPDF

Pages9

Reading Time23 min

AccessFree to download

Background

Patients and methods

Results

Conclusions

Key words

INTRODUCTION

ORIGINAL RESEARCH Patient-reported outcomes with selpercatinib treatment in patients with RET-driven cancers in the phase I/II LIBRETTO-001 trial5 L. E. Raez1, H. Kang2, Y. Ohe3, M. Khanal4, Y. Han4, S. Szymczak5, S. S. Barker6 & A. M. Gilligan7 􀀁 1Memorial Cancer Institute/Florida Atlantic University (FAU), Thoracic Oncology, Boca Raton, Florida, USA; 2Department of Medicine, University of California, San Francisco, San Francisco, USA; 3National Cancer Center Hospital, Department of Thoracic Oncology, Tokyo, Japan; 4Real World and Access Analytics, Statistics, Eli Lilly and Company, Indianapolis, USA; 5Clinical Research, Oncology, Eli Lilly and Company Poland, Lilly Corporate Center, Indianapolis, USA; 6Medical Affairs, Eli Lilly and Company, Indianapolis, USA; 7Value Evidence Outcomes Research and Oncology, Eli Lilly and Company, Indianapolis, USA Available online xxx Background: 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 and methods: 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. Results: 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 . Conclusions: 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. Key words: rearranged during transfection (RET), selpercatinib, patient-reported outcomes (PROs), health-related quality of life (HRQoL), non-small-cell lung cancer (NSCLC), medullary thyroid cancer (MTC), tumor agnostic (TA) INTRODUCTION Oncogenic alterations in REarranged during Transfection (RET), a receptor tyrosine kinase, manifest in several cancers including non-small-cell lung cancer (NSCLC), medullary thyroid cancer (MTC), papillary thyroid cancer (TC), and tumor-agnostic non-lung, non-thyroid solid cancers (TA) .1 *Correspondence to: Dr Adrienne M. Gilligan, Eli Lilly and Company, Indianapolis, IN, USA E-mail: [gilligan_adrienne@lilly.com](gilligan_adrienne@lilly.com) (A. M. Gilligan). 5Note: Previous presentation: A portion of these results were presented atthe European Society for Medical Oncology (ESMO) Congress 2023 in Madrid, Spain (Poster \# 669P; October 20-24, 2023). 2059-7029/© 2024 The Authors. Published by Elsevier Ltd on behalf of European Society for Medical Onco

FAQs

What patient-reported outcome measures were used in the study?

The study used the EORTC QLQ-C30 questionnaire for patients. Patients with medullary thyroid cancer also completed a modified Systemic Therapy-Induced Diarrhea Assessment Tool (mSTIDAT).

How were changes in patient status after baseline reported?

The study reports the proportion of patients whose status improved, remained stable, or worsened after baseline, with PRO summaries at 2 years or 3 years depending on cohort.

What did the study conclude about quality of life on long-term selpercatinib?

Most patients with RET-driven cancers improved or remained stable across most QLQ-C30 domains, indicating favorable health-related quality of life during long-term selpercatinib treatment.

Sep 21, 2026

Uploaded by Eliana