Patient-Reported Tolerability of Selpercatinib Compared to Cabozantinib/Vandetanib: A Secondary Analysis of the LIBRETTO-531 Randomized-Controlled Trial in RET -Mutant Medullary Thyroid Cancer
Authors' conclusion
This study demonstrated superior PRT for selpercatinib compared with control in patients with RET-mutant MTC, further supporting selpercatinib use as the first-line treatment for patients with advanced RET-mutant MTC.
Study context
- Population
- Patients with advanced RET-mutant medullary thyroid cancer (MTC)tolerability evaluable population
- Participants
- 242 patients
- Comparison
- Selpercatinib vs vandetanib/cabozantinib
- Selpercatinibn = 161
- Controln = 81reference
- Primary endpoint
- Patient-reported tolerability: proportion of time on treatment (PTT) with "high" side-effect burden (FACT item GP5 score 3-4, weekly)
Methodological detailsphase 3, multiplicity-controlled endpoint, two-sided significance level 0.05, conditional on efficacy endpoints
- Phase 3
- Multiplicity-controlled endpoint
- Two-sided significance level 0.05, conditional on efficacy endpoints
Primary result
As reported in the abstract
PTT with high side-effect burden
P < 0.0001
Further findings
| Outcome | P value |
|---|---|
| PTT with HRQoL impairment (physical, role, cognitive, emotional, social function)Less with Selpercatinib | all p < 0.01 |
- Proportion of time on treatment (PTT) with health-related quality of life (HRQoL) impairment (physical, role, cognitive, emotional, social function) — Physical function, Selpercatinib vs Control
- 36% vs. 52%
- PTT with HRQoL impairment (physical, role, cognitive, emotional, social function) — Role function, Selpercatinib vs Control
- 2% vs. 11%
- PTT with HRQoL impairment (physical, role, cognitive, emotional, social function) — Cognitive function, Selpercatinib vs Control
- 4% vs. 8%
- PTT with HRQoL impairment (physical, role, cognitive, emotional, social function) — Emotional function, Selpercatinib vs Control
- 6% vs. 11%
- PTT with HRQoL impairment (physical, role, cognitive, emotional, social function) — Social function, Selpercatinib vs Control
- 2% vs. 8%
PRO questionnaire post-baseline compliance
- Compliance generally greater than 80% in both groups
Safety
| Outcome | P value |
|---|---|
| PTT with severe symptomatic AEs (patient-reported)Less with Selpercatinib | all p < 0.001 |
- PTT with severe symptomatic AEs (patient-reported) — Severe diarrhea, Selpercatinib vs Control
- 5% vs. 38%
- PTT with severe symptomatic AEs (patient-reported) — Fatigue, Selpercatinib vs Control
- 6% vs. 21%
- PTT with severe symptomatic AEs (patient-reported) — Taste change, Selpercatinib vs Control
- 3% vs. 15%
- PTT with severe symptomatic AEs (patient-reported) — Decreased appetite, Selpercatinib vs Control
- 2% vs. 15%
- PTT with severe symptomatic AEs (patient-reported) — Hand-foot syndrome, Selpercatinib vs Control
- 2% vs. 9%
Original publication
Patient-Reported Tolerability of Selpercatinib Compared to Cabozantinib/Vandetanib: A Secondary Analysis of the LIBRETTO-531 Randomized-Controlled Trial in RET -Mutant Medullary Thyroid Cancer. Thyroid. Published 18 August 2025. doi:10.
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