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Spontaneous Murine Model of Anaplastic Thyroid Cancer
05:39

Spontaneous Murine Model of Anaplastic Thyroid Cancer

Published on: February 3, 2023

Phase II clinical trial of sorafenib in metastatic medullary thyroid cancer

Elaine T Lam1, Matthew D Ringel, Richard T Kloos

  • 1Departments of Internal Medicine, Center for Biostatistics, The Ohio State University, Columbus, OH, USA. manisha.shah@osumc.edu

Journal of Clinical Oncology : Official Journal of the American Society of Clinical Oncology
|April 7, 2010
PubMed
Abstract

Insights

Sorafenib showed antitumor activity in advanced medullary thyroid cancer (MTC). This multikinase inhibitor demonstrated clinical benefit in sporadic MTC, though rare fatal toxicities warrant caution.

Area of Science:

  • Oncology
  • Molecular Biology
  • Pharmacology

Background:

  • Medullary thyroid cancer (MTC) pathogenesis involves RET proto-oncogene mutations and VEGFR activity.
  • Sorafenib, a multikinase inhibitor, targets Ret and VEGFR, showing preclinical promise in MTC.

Purpose of the Study:

  • To evaluate the efficacy and safety of sorafenib in patients with advanced MTC.
  • To assess objective response rates, toxicity, and correlation with tumor markers, imaging, and RET mutations.

Main Methods:

  • Phase II trial with a two-stage design enrolling patients with hereditary (Arm A) and sporadic (Arm B) MTC.
  • Patients received sorafenib 400 mg orally twice daily.
  • Primary endpoint was objective response; secondary endpoints included toxicity and biomarker correlations.

Main Results:

  • In sporadic MTC (Arm B), one partial response (6.3%) and 14 stable disease (87.5%) were observed.
  • Median progression-free survival was 17.9 months; tumor markers decreased in most patients.
  • RET mutations were detected in 10 of 12 sporadic MTCs; Arm A was terminated early due to slow accrual.

Conclusions:

  • Sorafenib appears well-tolerated and offers potential clinical benefit for sporadic MTC patients.
  • Rare but fatal toxicities associated with sorafenib necessitate careful patient monitoring.
  • Further investigation into sorafenib's role in MTC management is warranted.

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