# EXAM: cabozantinib in progressive medullary thyroid cancer

Source: https://onco.cc/key-papers/paper-exam-cabozantinib-mtc-elisei-jco-2013/  
OnCo record `paper-exam-cabozantinib-mtc-elisei-jco-2013` (Key paper). Data CC BY-NC 4.0, attribute "Data from OnCo (onco.cc)"; commercial use needs a licence.

## TL;DR

Cabozantinib, a kinase inhibitor blocking RET, MET and VEGF receptors, delayed progression by more than seven months compared with placebo in progressive medullary thyroid cancer, at the cost of considerable toxicity.

## Summary

Phase 3 placebo-controlled trial of 330 patients with progressive metastatic medullary thyroid cancer randomised 2:1 to cabozantinib 140 mg daily or placebo.

Median progression-free survival was 11.2 versus 4.0 months (hazard ratio 0.28) and response 28 versus 0 percent, with benefit regardless of RET status; diarrhoea, hand-foot syndrome, weight loss and fistula were notable toxicities.

## Fields

- Kind: Key paper
- Last checked: 2026-09-17
- Journal: Journal of Clinical Oncology
- Year: 2013
- DOI: 10.1200/JCO.2012.48.4659
- Authors: Elisei R, Schlumberger MJ, Müller SP, et al.
- Findings: Median progression-free survival 11.2 vs 4.0 months; hazard ratio 0.28.; Objective response 28 percent vs 0 percent.
- What it means: Cabozantinib is an option for progressive medullary thyroid cancer, now mainly for RET-negative disease or after selpercatinib.
- Caveats: No overall survival benefit overall, though RET M918T carriers may have benefited.; High dose with frequent reductions.

## Sources

- J Clin Oncol 2013: https://doi.org/10.1200/JCO.2012.48.4659
- PubMed: https://pubmed.ncbi.nlm.nih.gov/24002501/

## Connected records

- cancers: [Medullary thyroid cancer](https://onco.cc/cancers/medullary-thyroid-cancer/)
- drugs: [Cabozantinib](https://onco.cc/drugs/cabozantinib/)
- trials: [EXAM](https://onco.cc/trials/exam/)
- journals: [Journal of Clinical Oncology](https://onco.cc/journals/jco/)

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