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Thalidomide-like Drug Benefits Some With Blood Disorder

Reduces need for transfusions for those with myelodysplastic syndromes, study says

WEDNESDAY, Feb. 9, 2005 (HealthDay News) -- A cousin of the drug thalidomide appears to have some benefit in certain individuals suffering from myelodysplastic syndromes, a group of disorders that can develop into acute myeloid leukemia.

The drug, called lenalidomide or Revlimid, may help some patients reduce their need for blood transfusions or avoid them altogether. And more than half the patients in the study went into remission, although the drug can produce serious side effects, the researchers said.

"This drug might help a subset of people who require transfusions to either lower their transfusion requirements or avoid them entirely," said Dr. Marshall Lichtman, executive vice president of research and medical programs at the Leukemia & Lymphoma Society.

Lichtman was not involved in the study, which appears in the Feb. 10 issue of the New England Journal of Medicine.

About 20,000 to 30,000 Americans are diagnosed with myelodysplastic syndromes (MDS) each year. In January, U.S. Representative Robert Matsui, a Democrat from California, died of complications from the disease.

The bone marrow in people suffering from myelodysplastic syndromes, sometimes also known as pre-leukemia or "smoldering leukemia," does not produce enough normal blood cells. The resultant anemia can be treated with recombinant erythropoietin, a hormone used to stimulate red blood production, and with transfusions.

Thalidomide, the drug that achieved notoriety in the 1960s for causing birth defects, has an effect on the disease but it can't be used for long periods of time or at high doses.

Revlimid is a more potent drug and doesn't have many of the side effects of thalidomide, the study authors said.

The new research involved 43 MDS patients who had had no response to erythropoietin or had high levels of erythropoietin in the body and so were unlikely to benefit from that therapy. All patients were given daily doses of Revlimid. The study was conducted and analyzed in consultation with Celgene, the company that makes Revlimid and also supplied the medication for the trial.

After four months, 24 patients (56 percent) had a response to the drug, including 20 individuals who no longer needed transfusions and three who had more than a 50 percent reduction in their need for transfusions.

Fifty-five percent of the participants went into a complete remission.

The responses were greatest among individuals with a certain genetic abnormality, the researchers add.

Unfortunately, there were also side effects. Sixty-five percent of the study participants developed neutropenia, an abnormally low number of certain white blood cells, while 74 percent developed thrombocytopenia or not enough platelets, which help the blood to clot. Treatment had to be interrupted or the dose changed for 25 (58 percent) of the patients.

"This is an incremental piece of information that helps define a subset of patients who might benefit from this thalidomide derivative and in that regard it's helpful," Lichtman said. "It's an inching forward."

The subset of patients includes those with "fairly severe anemia requiring transfusions who are not responsive to erythropoietin or are unlikely to respond," he added.

And the drug's side effects are not to be dismissed. "It's not without problems," Lichtman said. "The white blood cell count and the platelet count were lowered. That's something that a physician using this drug has to watch carefully. It could end up worsening other variables."

More research is needed on the drug, Lichtman said, including studies with longer results and more people.

More information

For more on MDS, visit the Aplastic Anemia & MDS International Foundation.

SOURCES: Marshall Lichtman, M.D., executive vice president of research and medical programs, Leukemia & Lymphoma Society, White Plains, N.Y.; Feb. 10, 2005, New England Journal of Medicine
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