From OncoLog, May 2012, Vol. 57, No. 5

In Brief

Drug Provides Symptom Reduction and Survival Advantages for Myelofibrosis Patients

Ruxolitinib, which has been shown to alleviate debilitating symptoms and reduce the size of swollen spleens in patients with myelofibrosis, in November became the first drug to be approved by the U.S. Food and Drug Administration for the treatment of that disease.

About 3,000 new cases of myelofibrosis are diagnosed in the United States each year. Myelofibrosis is caused by an accumulation of abnormal bone marrow cells that triggers an inflammatory response, scarring the bone marrow and limiting its ability to produce blood. As the body tries to compensate for the lack of red blood cells produced by the bone marrow, the spleen doubles or even triples in size in about 80% of patients.

“The drug may also extend survival in a patient population that has lacked effective treatments.”
– Dr. Srdan Verstovsek

Patients with myelofibrosis have shortened survival due to progressive disease or transformation to acute leukemia. Srdan Verstovsek, M.D., Ph.D., an associate professor in the Department of Leukemia at The University of Texas MD Anderson Cancer Center, said, “Most of these patients die from body wasting, organ failure, and other disease complications within 5–7 years.”

About half of myelofibrosis patients have a mutated JAK2 gene. This gene is responsible for normal blood cell production, and the mutation causes aberrant blood cell production by the bone marrow. In patients without a mutation in JAK2, its abnormal function has other causes. Ruxolitinib targets the JAK2 enzyme regardless of whether the gene is mutated and so can treat patients with or without JAK2 mutations. All patients with myelofibrosis have the same chance to benefit from ruxolitinib.

In a phase III clinical trial at MD Anderson and other institutions, myelofibrosis patients treated with ruxolitinib not only had reduced symptoms but also had a higher survival rate than did those receiving a placebo. At a median follow-up of 51 weeks, the mortality rate was 8.4% among patients receiving ruxolitinib compared with 15.6% among patients receiving a placebo.

“The phase I/II clinical trial showed that ruxolitinib improves quality of life for many patients; this phase III study indicates that the drug may also extend survival in a patient population that has lacked effective treatments,” said Dr. Verstovsek, the principal investigator for both trials.

The report of the phase III trial was published in the March 1 issue of the New England Journal of Medicine.

For more information, talk to your physician, visit www.mdanderson.org, or call askMDAnderson at 877-632-6789.


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