Related Pages Search for Clinical Trials 1 NCI's PDQ® Cancer Clinical Trials Registry. Multiple Myeloma/Other Plasma Cell Neoplasms 2 NCI's gateway for information about multiple myeloma and other plasma cell neoplasms.

Key Words

Multiple myeloma, thalidomide ³, melphalan, prednisone. (Definitions of many terms related to cancer can be found in the Cancer.gov Dictionary ⁴.)

Summary

Patients aged 75 and over with newly diagnosed multiple myeloma who received the drug thalidomide in addition to standard therapy (melphalan and prednisone) survived on average 14.9 months longer than patients who received standard therapy plus a placebo. The thalidomide group suffered worse side effects, however, and was more likely to discontinue treatment. Thalidomide is a treatment option for older patients who can tolerate the drug's side effects.

Source

Annual meeting of the American Society of Hematology ⁵, December 2007, Atlanta, Georgia. Updated results subsequently published online May 18, 2009, in the Journal of Clinical Oncology (see the journal abstract ⁶).

Background

Multiple myeloma occurs when a type of white blood cell called a plasma cell starts reproducing uncontrollably. The excess plasma cells crowd out healthy blood cells in the bone marrow (the spongy tissue inside large bones), causing pain and gradually destroying the bone.

An estimated 19,900 Americans were diagnosed with multiple myeloma in 2007 and about 11,000 died of the disease. Age is the most significant risk factor. Only one percent of cases of multiple myeloma are diagnosed in people under the age of 40. Two-thirds of those diagnosed are over 65; one in five is over 75.

The drug thalidomide, notorious in the 1960s because of its association with severe birth defects, has shown effectiveness as a treatment for multiple myeloma. Earlier trial results published in 2007 (see the journal abstract ⁷) established thalidomide, in combination with the drugs melphalan and prednisone, as standard treatment for patients aged 65 to 75 with newly diagnosed multiple myeloma.

Patients aged 75 and older often are excluded from clinical trials, so it has not been known whether such patients would benefit from the addition of thalidomide to standard treatment.

The Study

This study involved 229 patients with newly diagnosed multiple myeloma whose age ranged from 79 to 85 years. All of the patients received standard therapy (melphalan and prednisone). They were randomly assigned to receive either thalidomide or a placebo in addition to standard treatment. The patients were followed for a median of just under four years.

The principal investigator for this study was Cyrille Hulin, M.D., of University Hospital Center in Nancy, France.

Results

Patients treated with thalidomide plus standard therapy survived for a median of 44.0 months, compared with 29.1 months for those who received placebo plus standard therapy. Disease progression was delayed for a median of 24.1 months in the thalidomide group, compared with 18.5 months in the placebo group. Sixty-two percent of patients who received thalidomide, but only 31 percent of those given a placebo, had a partial or better response to treatment.

A higher incidence of adverse side effects was seen in patients treated with thalidomide. In that group, 42 percent of patients stopped treatment because of side effects, whereas only 13 percent of those in the placebo group did so. Tingling in the arms and legs, a drop in the white blood cell count, and depression all occurred more often in patients who received thalidomide.

Comments

In this study the addition of thalidomide to standard therapy statistically significantly improved survival in patients aged 75 and over with newly diagnosed multiple myeloma. However, because of the high incidence of adverse effects that were associated with thalidomide in this patient population, some doctors may be hesitant to recommend this regimen to their patients, says Michael Bishop, M.D., a multiple myeloma specialist with the National Cancer Institute's Center for Cancer Research.

Two new drugs, bortezomib ⁸ (Velcade®) and lenalidomide ⁹ (Revlimid®), have shown encouraging results in clinical trials in younger patients, adds Bishop, providing reason to believe that they may offer survival benefits with fewer side effects in older patients as well.

Glossary Terms

A type of research study that tests how well new medical approaches work in people. These studies test new methods of screening, prevention, diagnosis, or treatment of a disease. Also called clinical study.

Cancer that continues to grow or spread.

The number of new cases of a disease diagnosed each year.

A statistics term. The middle value in a set of measurements.

A drug that is used to treat multiple myeloma and ovarian epithelial cancer and is being studied in the treatment of other types of cancer. It belongs to the family of drugs called alkylating agents. Also called Alkeran.

An inactive substance or treatment that looks the same as, and is given the same way as, an active drug or treatment being tested. The effects of the active drug or treatment are compared to the effects of the placebo.

A type of immune cell that makes large amounts of a specific antibody. Plasma cells develop from B cells that have been activated. A plasma cell is a type of white blood cell. Also called plasmacyte.

A drug that lessens inflammation and suppresses immune responses. It may also kill leukemia and lymphoma cells. Prednisone is used to treat many conditions, including arthritis, certain skin diseases, allergies, low levels of some adrenal hormones, and anemia (a low level of red blood cells). It is also used to treat the symptoms of several types of leukemia and lymphoma. Prednisone is a type of therapeutic glucocorticoid.

Something that increases the chance of developing a disease. Some examples of risk factors for cancer are age, a family history of certain cancers, use of tobacco products, being exposed to radiation or certain chemicals, infection with certain viruses or bacteria, and certain genetic changes.

In medicine, treatment that experts agree is appropriate, accepted, and widely used. Health care providers are obligated to provide patients with standard therapy. Also called best practice and standard of care.