Efficacy and Safety of Selective RET Inhibitors in Patients with Advanced Hereditary Medullary Thyroid Carcinoma
Authors' conclusion
In patients with advanced hMTC, selective RETis appear safe and effective with outcomes similar to clinical trial cohorts, which mostly comprised patients with sMTC.
Study context
- Population
- Patients with advanced hereditary medullary thyroid carcinoma (MTC) (MEN2A 15 [65%], MEN2B 8 [35%]) treated with a selective RETi; all with distant metastases; median age 51 years (range, 15–79); 52% prior systemic therapyevaluable
- Participants
- 23 patients
- Exposure
- Selpercatinib vs Pralsetinib
- Selpercatinibn = 13
- Pralsetinibn = 10
- Outcome
- Overall response rate (Response Evaluation Criteria in Solid Tumors (RECIST) v1.1)
Methodological detailssingle-centre, tertiary cancer center, 57% (13/23) treated within a clinical trial
- Single-centre
- Tertiary cancer center
- 57% (13/23) treated within a clinical trial
Main finding
As reported in the abstract
Best radiographical response
Partial response
18
78%
Stable disease
5
22%
Observational data: associations, not effects.
Secondary outcomes
Overall survival (OS) · median follow-up 49 months (range, 9-72)
- Median OS
- 51 months (CI, 40.5-61.3)
Progression-free survival (PFS)
- Median PFS
- not reached
Duration of RETi
- Median duration of RETi
- 25 months (range, 3-72)
- Remaining on drug at data cutoff due to ongoing response
- 11/23 (48%)
Safety
Most common adverse events (AEs)
- Increased ALT
- 48%
- Increased aspartate aminotransferase
- 26%
- Dry mouth
- 39%
- QT interval prolongation
- 39%
- Fatigue
- 35%
- Hypertension
- 26%
- AEs leading to dose reductions
- 8 (35%)
- Grade 5 treatment-related AEs
- 0
- Incidence of most AEs, other than grade 1-2 ALT elevation and QT prolongation, comparable to published clinical trials
Original publication
Efficacy and Safety of Selective RET Inhibitors in Patients with Advanced Hereditary Medullary Thyroid Carcinoma. Thyroid. Published 4 December 2024. doi:10.
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