New Drug May Help Fight Rare Bone Marrow Disorder

WEDNESDAY, Feb. 29 (HealthDay News) — Two new studies confirm
that the new drug ruxolitinib can help people with the rare bone marrow
disorder called myelofibrosis.

While the drug, marketed in the United States under the brand name
Jakafi, won’t cure myelofibrosis, it can help ease symptoms, improve
quality of life, and possibly improve survival according to the studies.

“This is probably the most thrilling development I have seen in my
career. The drug delivers rapid, durable benefit which makes a great
difference to patients’ lives,” said the lead author of one of the
studies, Dr. Claire Harrison, a consultant hematologist at Guy’s Hospital
in London.

And, the lead author of the other study, Dr. Srdan Verstovsek, an
associate professor in the leukemia department at the M.D. Anderson Cancer
Center in Houston, said his team’s study “shows that ruxolitinib provides
a significant durable clinical benefit in terms of spleen-size reduction
and myelofibrosis-related symptom improvement that leads to an overall
survival benefit.”

Results of both studies are published in the March 1 issue of the
New England Journal of Medicine.

Verstovsek’s study was funded by Incyte, the manufacturer of Jakafi,
which was approved by the U.S. Food and Drug Administration in November
2011. Harrison’s study was funded by Novartis, the company that will
market the drug outside the United States. The drug is not currently
approved in Europe.

The drug is expensive, costing about $84,000 a year.

Myelofibrosis is a bone marrow disorder that causes scar tissue to
develop in the place of bone marrow. When the bone marrow becomes scarred,
it can’t produce enough blood cells any more. This causes a number of
problems including an enlarged spleen, a feeling of fullness, bone pain,
bruising, easy bleeding, tiredness and shortness of breath when
exercising, according to the U.S. 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,
according to the study. Harrison’s study didn’t find an effect on overall
survival.

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.”

More information

Learn more about myelofibrosis from the U.S. National Library of Medicine.

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