Rare Disease Report

Jakafi May Be Even Better Than First Thought For Myelofibrosis

JUNE 04, 2013
Today, at the ASCO annual meeting, Hans Michael Kvasnicka, M.D., of the University of Frankfurt in Germany presented data from an ongoing, Incyte Corporation sponsored, Phase I/II single-arm, open-label clinical trial for Jakafi® (ruxolitinib) examining the effects of the drug on bone marrow fibrosis  in patients with Myelofibrosis (n=68).  Bone marrow fibrosis grade was determined at three time points (baseline, month 24, month 48)  by three expert hematopathologists using the World Health Organization scoring system and blinded to patient data and outcome.
The results of the study were quite remarkable. According to a press release, stabilization or improvement of bone marrow fibrosis was observed at both the 24 and 48 month points, and by month 48, bone marrow fibrosis was stabilized in 56% of Jakafi-treated patients and improved in 22%.
In the press release,  Dr. Kyansicka stated, “These results provide the first evidence of a drug therapy that may stabilize or improve bone marrow fibrosis and are encouraging because the only other proven option is bone marrow transplantation, which is a procedure with high risks. Future studies should improve our understanding of the significance of these findings.”
Srdan Verstovsek, M.D., Ph.D., Professor, Department of Leukemia, Division of Cancer Medicine, The University of Texas MD Anderson Cancer Center in Houston added, “Ruxolitinib has shown clinically meaningful results in symptom improvement and spleen reduction, and additional data has shown potential survival benefits. Now the findings from this exploratory analysis suggest that long-term therapy with ruxolitinib may positively impact another key underlying hallmark of this life-threatening and progressive disease.”
The results of the analysis were reported during an oral presentation, Exploratory Analysis of the Effect of Ruxolitinib on Bone Marrow Morphology in Patients with Myelofibrosis and slides from the presentation can be viewed at http://www.incyte.com/sites/default/files/ASCO%20BMF%20slides_Jun13.pdf
Myelofibrosis is a life-threatening blood cancer that belongs to a group of diseases referred to as myeloproliferative neoplasms (or MPNs). Myelofibrosis has a poor prognosis and limited treatment options.  Jakafi is the only orphan drug currently indicated to treat myelofibrosis.
Kvasnicka HM, Thiele J, Bueso-Ramos CE, et al.  Exploratory Analysis of the Effect of Ruxolitinib on Bone Marrow Morphology in Patients with Myelofibrosis. J Clin Oncol 31, 2013 (suppl; abstr 7030).

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