It is a treatment used in many cancer centers but "it has become a standard therapy without adequate data becoming available," says Dr. J. Anthony Child, a British hematology specialist. He is lead author of a report on the trial in the May 8 issue of the New England Journal of Medicine.
Multiple myeloma causes uncontrolled overproduction of the white blood cells, resulting in pain and destruction of bones. The American Cancer Society estimates there will be 14,600 new cases in the United States this year, with 10,900 deaths.
The treatment is far from a cure, Child says, but it does buy time -- an average of an extra year of life compared to lower-dose chemotherapy. "That may not sound like much, but it is very important to patients and their families," says Child, who is a clinical hematology professor at the General Infirmary at Leeds in Yorkshire.
The newly reported study helps clear up the picture painted by two smaller-scale French trials that produced conflicting results, Child adds. "This does give a much clearer picture," he says. "The British trial does give an answer in favor of high-dose therapy."
Support for that comes from Dr. Stephen D. Nimer, head of the division of hematologic oncology at Memorial Sloan-Kettering Cancer Center in New York City. "This paper supports what others and we are doing," Nimer says. "It is not the first trial to demonstrate that patients transplanted as part of initial high-dose therapy do better than those who are not, but it confirms it."
The trials included 407 patients, divided into low-dose and high-dose groups. Both groups were given a number of cancer drugs, but the drug regimen was different for the high-dose group and they were also given stem cells taken from their own bone marrow to fortify the body.
The initial response rate was almost the same for both groups -- the progression of the disease slowed or stopped in 40 percent of the standard therapy patients and 42 percent of those in the high-dose group. However, the long-term results were clearly different: average survival of 54.1 months for those getting intensive treatment, 44.9 months for those getting the lower doses.
Efforts to improve both survival time and quality of life are continuing, Child and Nimer say. The British physicians are beginning a new trial to see whether stem cells from a patient's close relative can give better results, while Nimer and his colleagues are trying two stem cell transplants instead of one and are trying thalidomide on patients whose disease comes back after a stem cell transplant.
"The median survival was 54 months, which is just four and a half years," Nimer says. "We've got to do better than that."
"A cure for this disease is what everyone is looking for," Child adds. "The next best is long-term control with very low-level symptoms. If you can achieve a low level of disease with therapy, it is perhaps a step toward therapy that can keep patients symptom-free in the very long term."