Masitinib plus gemcitabine may be effective in the treatment of specific subgroups of patients with advanced pancreatic ductal adenocarcinoma (PDAC), according to a recent study published in the Annals of Oncology.

Researchers led by Gaël Deplanque, MD, of the Saint Joseph Hospital in Paris looked at 353 patients with inoperable, chemotherapy-naïve PDAC who were randomized to either gemcitabine in combination with masitinib or placebo.

Primary endpoint was overall survival in a modified intent-to-treat population, with secondary analyses intended to define subgroups with poor survival when receiving single-agent gemcitabine with subsequent evaluation of masitinib.

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Two specific subgroups that were noted for having a significantly poor survival rate when receiving single-agent gemcitabine were those with an overexpression of acyl-CoA oxidase-1 (ACOX1) in the blood, and those with a baseline pain intensity threshold (VAS greater than 20 mm).

The researchers found that median overall survival was similar between the treatment arms for the overall population.

However, they found a significant treatment effect in the aforementioned subgroups treated with masitinib, with a median overall survival of 11.7 months in those with ACOX1 overexpression, and 8.0 months in the subgroup with a lower threshold for pain.

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While they noted higher rate of toxicities in patients treated with masitinib, they stated that these side effects were manageable.

“The present data warrant initiation of a confirmatory study that may support the use of masitinib plus gemcitabine for treatment of PDAC patients with overexpression of ACOX1 or baseline pain,” they concluded.


  1. Deplanque, G., et al. “A randomized, placebo-controlled phase II itrial of masitinib plus gemcitabine in the treatment of advanced pancreatic cancer.” Annals of Oncology. doi: 10.1093/annonc/mdv133. April 9, 2015.