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Ruxolitinib, an inhibitor of Janus kinase (JAK)1/2, decreases spleen volume and improves survival in some but not all patients with myelofibrosis (NEJM JW Oncol Hematol Oct 2012). Although a few studies suggested that the prognosis of myelofibrosis was related to genetic subtype (NEJM JW Oncol Hematol Oct 2014), others reported that ruxolitinib improved survival independent of mutation profile (Blood 2014; 123:2157).
To resolve this controversy, investigators sequenced 28 genes known to be mutated in hematologic malignancies in a cohort of 95 myelofibrosis patients treated with ruxolitinib in a previous phase I/II clinical trial (N Engl J Med 2010; 363:1117). They made the following observations:
At least 1 mutation occurred in 53.7% of patie…