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Summaries of Newsworthy Clinical Trial Results

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    Posted: 03/15/2006    Updated: 09/16/2008
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Thalidomide Effective in Multiple Myeloma

Reprinted from the NCI Cancer Bulletin, vol. 3/no. 11, March 14, 2006 (see the current issue).

Current treatment for multiple myeloma entails a long and intricate chemotherapy regimen that includes one or two transplants with a patient's own stem cells accompanied by the drug melphalan. The anti-angiogenesis agent thalidomide has proven to be an effective component of therapy, but questions remain about where in the course of treatment it can best be used. In the March 9, 2006, issue of the New England Journal of Medicine (see the journal abstract), researchers report superior event-free and complete response rates when the drug was used before and during primary therapy and also thereafter for maintenance. Yet the controls who took no thalidomide lived just as long. [See note, below.]

After 4.5 years, Dr. Bart Barlogie and colleagues from the University of Arkansas found that 56 percent of patients taking thalidomide had no adverse events - disease progression, relapse, or death from any cause - compared with 44 percent of controls following the same treatment regimen without the drug; and an even greater difference was seen in complete responses, 62 to 43 percent. But each group contributed comparably to the 190 patients who died during this time, "owing in part to significantly shorter survival after relapse in the thalidomide group," wrote the authors, 1.1 compared with 2.7 years. Further compromising the advantage for thalidomide were more serious adverse events, such as deep-vein thrombosis and severe peripheral neuropathy.

In an editorial, Drs. Michele Cavo and Michele Baccarani, of the University of Bologna in Italy, suggest one interpretation of the data was to reserve treatments such as thalidomide "for the sequential treatment of relapses as a means of controlling the growth or regrowth of tumor."

[Note: Longer-term follow-up data from this trial were subsequently published online May 20, 2008, in the journal Blood, showing that there was a survival advantage for those in the thalidomide group after all, but only among those with a high-risk type of myeloma; see the journal abstract.]

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