Real-world efficacy and safety of selpercatinib in RET -mutant medullary thyroid cancer at a Tertiary Referral Center
Authors' conclusion
In real-word settings, selpercatinib showed high efficacy and tolerability, particularly as first-line treatment. Our findings support its use as the preferred initial targeted treatment for RET-driven MTC beyond clinical trial populations.
Study context
- Population
- Patients with advanced RET-mutant medullary thyroid cancer (MTC) treated with selpercatinib, March 2021–May 2025, Essen Endocrine Tumor Center; 87% distant metastases, median age 54 years, 84% sporadic
- Participants
- 31 patients
- Exposure
- First-line vs later-line selpercatinib
- Outcome
- Median PFS and ORR
Methodological detailssingle-center
- Single-center
Main finding
As reported in the abstract
Median PFS and ORR First-line median not reached · Later-line median 18.3 months
ORR
One-year PFS rate
Observational data: associations, not effects.
Further findings
Objective response rate (ORR) by bone metastases
- ORR significantly higher in patients without bone metastases
- 90% vs 43%
ORR and progression-free survival (PFS) by age, sex, RET mutation subtype
- Age at treatment initiation, sex and RET mutation subtype did not impact ORR or PFS
Safety
- Hypertension
- 58.6%
- Erectile dysfunction
- 55.6%
- Grade ≥3 events (hypertension most frequent)
- 37.5%
- Treatment interruptions and dose reductions (mainly QTc prolongation, fatigue)
- 22.6%
- Permanent discontinuation due to adverse events (AEs)
- 3 (9.7%)
Original publication
Real-world efficacy and safety of selpercatinib in RET -mutant medullary thyroid cancer at a Tertiary Referral Center. Eur J Endocrinol. Published 22 December 2025. doi:10.
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