In patients with myelofibrosis treated with ruxolitinib, harboring three or more mutations was inversely associated with spleen response and time to treatment discontinuation, a new study published online ahead of print in the journal Blood has shown.

For the study, researchers from The University of Texas MD Anderson Cancer Center in Houston, Texas, sought to identify genes that may predict response to ruxolitinib, a drug approved by the U.S. Food and Drug Administration (FDA) for the treatment of intermediate or high-risk myelofibrosis.

Researchers performed gene sequencing of a panel of 28 genes recurrently mutated in patients with hematologic malignancies in a group of patients with myelofibrosis who participated in a phase 1/2 study evaluating treatment with ruxolitinib.

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Results showed that 98% of patients had at least one gene mutation. Researchers found patients with two or fewer mutations had 9-fold higher odds of achieving a spleen response compared with those who had three or more mutations.

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The study also demonstrated that patients with three or more mutations had a shorter time to treatment discontinuation, as well as shorter overall survival, than those with two or fewer mutations.

The findings suggest that multigene profiling may be useful for therapeutic planning for myelofibrosis.


  1. Patel KP, Newberry KJ, Luthra R, et al. Correlation of mutation profile and response in patients with myelofibrosis treated with ruxolitinib. Blood. 2015. [epub ahead of print]. doi: 10.1182/blood-2015-03-633404.