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Summary: Multiple myeloma patients awaiting stem cell transplants may now have a better pre-transplant treatment option to choose. Writing in the journal Blood, researchers from Italy report that the drug combination of thalidomide and dexamethasone leads to more remissions than standard therapy with vincristine, doxorubicin, and dexamethasone, called VAD.

Why it's important: For many people with myeloma, especially those younger than 70, the standard treatment is an autologous stem cell transplant. These transplants are generally more successful if the number of myeloma cells in the body is first reduced with drug treatment — usually VAD. But this treatment is cumbersome because the vincristine and doxorubicin must be given continuously over four days through a special intravenous line. Also, the doxorubicin can damage the heart. Patients need an easier treatment that is still effective at killing myeloma cells in preparation for stem cell transplant.

What's already known: Recently it has been learned that thalidomide is effective in treating myeloma when combined with dexamethasone. Because both drugs are given as pills, the combination, called Thal-Dex, is much easier to take than intravenous VAD. But doctors questioned whether Thal-Dex was as good as VAD and whether, after the treatment, they could harvest enough stem cells for the transplant.

How this study was done: Two hundred people with myeloma were studied. Half received VAD and the other half got Thal-Dex. The researchers measured the response to each therapy (whether it fully or partially eliminated the myeloma), and when each therapy was completed, how many stem cells could be collected for transplant. They also looked at side effects.

What was found: People who were given Thal-Dex had a higher response rate than those given VAD, 76% versus 52%. The number of stem cells harvested from those in the Thal-Dex group was less than from the VAD patients, but still enough for transplantation, according to the researchers.

The side effects from the two treatments were different. Twelve percent of patients in the VAD group had serious problems with low blood counts, while blood clots were the most serious side effect for people who were given Thal-Dex, occurring in 15%. Other problems, like constipation, infections, and tingling in the arms and legs, occurred less often in both groups. Six people from each group died during the treatment. The deaths did not seem to be caused by the treatments.

Study strengths and limitations: Individuals on both treatment plans were closely matched by age and disease characteristics. However, they were studied after receiving the treatments, and they were not randomly assigned to Thal-Dex or VAD. A so-called randomized trial is considered a better comparison of different treatment options. Also, this trial included only 200 patients; larger studies give more definitive results.

The bottom line: Thal-Dex appears to be more effective and much easier to give than VAD as the first treatment in myeloma patients who will be undergoing stem cell transplantation. But the researchers say more studies are needed to find better ways to control the blood clots that Thal-Dex can cause and identify the people most at risk of developing them.

Citation: "Superiority of thalidomide and dexamethasone over vincristine-doxorubicin-dexamethasone (VAD) as primary therapy in preparation for autologous transplantation for multiple myeloma." Published online March 10, 2005, in Blood. First author: Michele Cavo, Institute of Hematology and Medical Oncology "Seragnoli," University of Bologna, Italy.