Thalidomide Continues to Show Benefits Against Myeloma

The once-notorious drug aiding certain patients with the blood cancer

WEDNESDAY, July 9, 2008 (HealthDay News) -- Researchers exploring the benefits of the once-banned drug thalidomide for patients with myeloma have learned a lesson that's as true to life as it is to science: Don't judge results too early.

Earlier findings showed that adding thalidomide into therapy regimens increased the odds of a complete remission, but not the length of the remission, and also slowed recurrences without benefiting overall survival. This was after a follow-up of about three years.

But now a letter published in the July 10 issue of the New England Journal of Medicine reports that, after a median follow-up of eight years, overall survival in people taking thalidomide was, in fact, extended, although most significantly in the one-third of patients with certain genetic abnormalities. The eight-year survival rate for patients with these cytogenetic abnormalities was 46 percent, versus 27 percent for patients in the control group.

But even that wasn't the end of the story. More recent results, current as of July 4, 2008, show that all patients taking thalidomide did better, although those with cytogenetic abnormalities showed the benefits earlier, said Dr. Bart Barlogie. He is lead author of the study and a professor of medicine and director of the Myeloma Institute for Research and Therapy at the University of Arkansas for Medical Sciences in Little Rock.

"The message is both for patients but, importantly, for myeloma physicians and investigators, to exercise constraint in interpretation of early data," Barlogie said.

Beyond that, the findings are obviously good news for people with this blood cancer, many of whom are 65 or older at the time of diagnosis.

"This trial is remarkable, because it shows that the addition of thalidomide now looks like it's making a difference in long-term survival in patients who had worse disease," said Dr. Bart Kamen, executive vice president and chief medical officer for the Leukemia & Lymphoma Society. "To my mind, it's a big deal, because we've added a good quality of life... About two-thirds of the patients stopped taking thalidomide after three-and-a-half to four years, so whatever good happened, it happened early."

According to the Leukemia & Lymphoma Society, more than 15,000 Americans are diagnosed each year with multiple myeloma. The disease affects blood cells called B-lymphocytes. While some myelomas are slow-moving and pose little immediate threat, others can be very aggressive.

Thalidomide was taken off the market in the 1960s, because women who took it during pregnancy had a much higher rate of severe birth defects. However, studies suggesting it might help against cancer led to its reintroduction -- with strict controls -- in 1998.

A second study in the same issue of the journal looked at a single African-American family with 5 cases of multiple myeloma; 3 cases of monoclonal gammopathy of undetermined significance (MGUS, the presence of an abnormal protein in the blood); and 5 cases of prostate cancer in just two generations.

The family should be studied further, stated the authors, from Creighton University in Omaha, to possibly shed light on the genetic underpinnings of myeloma.

Although the causes of myeloma are unknown, it is more common in blacks than in whites.

More information

Visit the Leukemia & Lymphoma Society for more on myeloma.

SOURCES: Bart Barlogie, M.D., professor of medicine and director, Myeloma Institute for Research and Therapy, University of Arkansas for Medical Sciences, Little Rock; Bart Kamen, M.D., Ph.D., executive vice president and chief medical officer, the Leukemia & Lymphoma Society, White Plains, N.Y.; July 10, 2008, New England Journal of Medicine
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