Thalidomide for multiple myeloma patients may lengthen survival

ROCHESTER, Minn. — Nearly one-third of patients with advanced multiple myeloma who had failed current standard therapy of chemotherapy or stem cell transplantation responded to thalidomide for a median duration of nearly one year in a Mayo Clinic study of the effects of thalidomide on myeloma. The findings are reported in the January issue of Mayo Clinic Proceedings.
Many studies in the last three years have determined that thalidomide is effective in the treatment of multiple myeloma, following the initial report by researchers at the University of Arkansas. However, information is limited on how long thalidomide therapy works and on survival rates with such therapy. The Mayo Clinic researchers report on the results of a study that looked at 32 patients with relapsed multiple myeloma.

“Thalidomide is useful in the treatment of patients with relapsed multiple myeloma,” said Vincent Rajkumar, M.D., a hematologist at Mayo Clinic and an author of the study. “Our study confirms an earlier report from the University of Arkansas that among patients who respond to therapy, the benefits are not transient, but last approximately one year on average.” Studies are now addressing thalidomide’s role in combination with other treatments and in earlier stages of the disease.

The researchers note that an estimated 14,600 new patients were diagnosed with myeloma in the United States during 2002 and an estimated 10,800 deaths will be due to myeloma in the same period. The average survival from diagnosis among patients treated with conventional chemotherapy is three to four years. Multiple myeloma, cancer of the bone marrow, remains an incurable cancer despite advances in high-dose chemotherapy and stem cell transplantation therapy. Thalidomide is not currently approved by the U.S. Food and Drug Administration for the treatment of myeloma.

Researchers are looking to thalidomide, as well as other treatments or combinations, to find ways to lengthen the survival of patients with multiple myeloma. Another study in the same issue of the journal reviews the records of all patients in whom multiple myeloma was initially diagnosed at Mayo Clinic Rochester from Jan. 1, 1985, to Dec. 31, 1998, and found the median duration of survival among the 1,027 patients was 33 months and did not improve during this period.

The article reviews its findings of the features of the disease to aid physicians in recognizing and diagnosing it. They found that bone pain and fatigue related to anemia were common.

Despite the lack of improved survival over 13 years, Mayo Clinic researchers say there is reason to believe survival rates in the future will improve significantly because high-dose therapy with stem cell support and new agents for treatment are being introduced.

First, the use of stem cell transplantation has been shown to prolong survival significantly compared with standard-dose chemotherapy. Second, thalidomide has recently shown significant activity in relapsed myeloma, with a median response duration of approximately one year. Third, promising new drugs have shown impressive activity in patients with advanced myeloma. Supportive care has improved for patients with bony lesions, and efforts to develop oral maintenance drug regimens are ongoing.

“These advances, coupled with remarkable strides in the understanding of the biology of the disease, provide considerable hope and optimism for both patients and myeloma researchers,” said Robert Kyle, M.D., of Mayo Clinic and an author of the study.

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The study was supported by a grant from the National Cancer Institute.

In an editorial in the same issue, Kenneth Anderson, M.D., of the Dana-Farber Cancer Institute in Boston, Mass., notes the progress in the research of multiple myeloma and lauds the research of Dr. Kyle, whose work over more than 25 years has contributed to the understanding of the history and symptoms of multiple myeloma.

The study by Dr. Rajkumar and others was supported in part by grants from the National Cancer Institute in Bethesda, Md., and by the Celgene Corporation of Warren, N.J. Drs. Raphael Fonseca, a hematologist at Mayo Clinic and a researcher on the study, and Rajkumar received Leukemia and Lymphoma Society of America Translational Research Awards, and Dr. Rajkumar is supported by the Goldman Philanthropic Partnerships of Lake Forest, Ill., and the Multiple Myeloma Research Foundation.