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New Drug May Help Fight Rare Bone Marrow Disorder
Date:3/1/2012

. National Library of Medicine.

Estimates of the prevalence of myelofibrosis in the United States vary, but one study from the Mayo Clinic calculates that about 30,000 Americans have the disorder.

There is no specific treatment approved to cure myelofibrosis, though bone marrow transplants may be successful, according to Dr. Alan Astrow, director of hematology and medical oncology at the Maimonides Medical Center in New York City.

But, for those who don't qualify for a bone marrow transplant, treatment is directed to relieving symptoms, such as blood transfusions to help relieve fatigue, excessive bleeding and shortness of breath.

Verstovsek's study included 299 people with myelofibrosis defined as intermediate-2 or high-risk myelofibrosis, a group that represents about 80 percent of people with myelofibrosis, according to the author. Half the group was randomly chosen to receive ruxolitinib twice daily, while the other half was given an inactive "placebo" twice daily.

Almost 42 percent of those on ruxolitinib had a significant reduction in the size of their spleens at 24 weeks of therapy, compared with less than 1 percent of the group receiving the placebo. About 46 percent receiving the drug reported a significant reduction in their symptoms versus about 5 percent of those on placebo, according to Verstovsek's study.

His team also found the risk of death during the study was decreased by 50 percent for those taking the medication.

Harrison's study included 219 people with the same level of myelofibrosis. Of those, 146 people were randomly chosen to receive ruxolitinib and 73 were given standard treatments to relieve symptoms.

After 48 weeks, 28 percent of those on ruxolitinib had a significant reduction in their spleen size, while no one in the standard treatment therapy achieved this goal. Those in the ruxolitinib group reported more symptom relief and better quality of life than those receiving usual care, a
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