Real-world external control arm for the single-arm LIBRETTO-001 trial of selpercatinib in RET-mutation-positive medullary thyroid cancer: RECALIB-RET

J. Hadoux; J. Hernando; K.H. Wong; C. Do Cao; H. Lasolle; C. Buffet; D. Benisvy; F. Illouz; O. Schneegans; R. Varnier; D. Drui; S. Zanetta; S. Hescot; M. Müller; C. I. S. Nascimento; N. Roudaut; M. Khanal; S. Zheng; X. K. Li; R. Singh; W. H. Tian; A.S. Afonso; B. Houot; I. Borget; G. Segall

doi:10.1016/j.esmoop.2026.106083

Key Points

The study evaluates the effectiveness of selpercatinib in patients with RET-mutation-positive medullary thyroid cancer compared to traditional treatments.
Utilized retrospective real-world data to match with prospective trial data.
Focused on treatment-naïve patients in the first-line setting.
Assessed progression-free survival (PFS) outcomes against standard of care.
Selpercatinib showed significant improvement in progression-free survival for treatment-naïve patients.
Results were inconclusive for patients in the second-line treatment setting.
The approach demonstrated feasibility of external control arms for single-arm trials.

Implication

Real-world data analysis shows improved progression-free survival with selpercatinib in treatment-naïve patients, suggesting effective comparative evaluation methods.

Abstract

Selpercatinib conferred significant PFS benefit over SoC in treatment-naïve (1L) patients with RET-mutation-positive MTC, with inconclusive results in the ≥2L setting. Matching retrospective real-world data to prospective trial data is feasible. EC arms to single-arm trials may provide evidence supporting the evaluation of comparative effectiveness.

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Cite This Study

Hadoux et al. (2026) studied this question.

synapsesocial.com/papers/69abc0925af8044f7a4e9416 https://doi.org/10.1016/j.esmoop.2026.106083

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Real-world external control arm for the single-arm LIBRETTO-001 trial of selpercatinib in RET-mutation-positive medullary thyroid cancer: RECALIB-RET

Key Points

Implication

Abstract

Discussion

Cite This Study

Real-world external control arm for the single-arm LIBRETTO-001 trial of selpercatinib in RET-mutation-positive medullary thyroid cancer: RECALIB-RET

Key Points

Implication

Abstract

Discussion

Cite This Study

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