The drug appeared to be well tolerated in both studies, and both study authors agreed that the benefits of the medication outweighed any potential risks.
Maimonides' director Astrow said, "Ruxolitinib appears to be useful for patients who have symptoms related to large spleens, who aren't candidates for bone marrow transplant. This drug appears to be effective in significantly reducing the size of the spleen and in helping patients feel better. Their appetites improved, they had less pain and they had more energy. Overall, the results seem to show improved quality of life. But, it's not a cure," he added.
"We hope this is a first step toward improved treatment for people with this disease that currently has no curative therapy aside from allogeneic bone marrow transplants for some patients," Astrow said.
The wholesale cost of Jakafi in the United States is about $7,000 a month, according to Incyte, the drug's manufacturer. Astrow said that once someone is on this medication, they generally stay on it. He said if the drug is stopped, symptoms often return, sometimes "suddenly and severely."
Learn more about myelofibrosis from the U.S. National Library of Medicine.
SOURCES: Claire Harrison, D.M., consultant hematologist, Guy's Hospital, London; Srdan Verstovsek, M.D., Ph.D., associate professor, leukemia department, M.D. Anderson Cancer Center, Houston; Alan Astrow, M.D., director, hematology and medical oncology, Maimonides Medical Center, New York City; March 1, 2012, New England Journal of Medicine
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