Thyroid Cancer Coverage from Every Angle

ESMO 2020: Nintedanib in Progressive Medullary Thyroid Cancer

By: Sarah Campen, PharmD
Posted: Monday, September 28, 2020

Slow patient accrual led to the closure of a phase II study of treatment with the kinase inhibitor nintedanib in patients with progressive advanced medullary thyroid cancer, reported Kate Newbold, MD, of the Institute of Cancer Research and Royal Marsden NHS Foundation Trust, Sutton, United Kingdom, and colleagues at the European Society for Medical Oncology (ESMO) Virtual Congress 2020 (Abstract 1919P). Although nintedanib appears safe, “no conclusion on efficacy could be drawn due to the low power of the study,” stated the authors. Nintedanib is an oral medication used for the treatment of idiopathic pulmonary fibrosis and along with other medications for some types of non–small cell lung cancer.

The double-blind study enrolled 31 patients with advanced medullary thyroid cancer with documented disease progression after treatment with vandetanib and/or cabozantinib. Participants were randomly assigned to receive the triple angiogenesis inhibitor (n = 22) or placebo (n = 9).

The study was not able to reach the targeted statistical power, falling short of enrolling the 67 patients required. Of the 20 patients enrolled in the per-protocol population—15 in the nintedanib arm and 5 in the placebo arm—the median progression-free survival was 7.0 and 3.9 months, respectively. The best overall response was stable disease in nine patients in the nintedanib arm and four patients in the placebo arm. The median overall survival was 12.3 months with nintedanib and 16.4 months with the placebo.

As for safety, at least one grade 3 or 4 adverse event occurred in 59% of patients receiving nintedanib and 33% of those on the placebo arm. The most common adverse events observed with nintedanib treatment were diarrhea (18 %), nausea (9%), increase in gamma-glutamyl transferase (18%), and lymphopenia (18%).

Disclosure: For full disclosure of the study authors, visit


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