Maintenance Treatment with Oral Lenalidomide after Stem Cell Transplant Reduces Risk of Disease Progression for Multiple Myeloma Patients

Sunday, June 6, 2010

BUFFALO, NY - Updated data from a National Cancer Institute-sponsored clinical trial conducted by the Cancer and Leukemia Group B (CALGB) was presented June 6th at the 46th annual meeting of the American Society of Clinical Oncology in Chicago. The study evaluated the benefits of maintenance treatment with lenalidomide (Revlimid®) administered orally following an autologous stem cell transplant for patients with newly diagnosed multiple myeloma, compared to placebo. Maintenance therapy is an ongoing treatment given after patients achieve remission with initial therapy to try to prolong remission.

Results of the Phase III, randomized, double-blind study involving 418 patients illustrated that more than twice as many of those in the placebo arm saw their disease progress. After 12 months, 13.8% of patients treated with lenalidomide had disease progression, compared with 27.8% of patients who received a placebo. Patients treated with lenalidomide had a 58% reduction in the risk of their disease progressing. The most common side effects experienced by patients receiving lenalidomide were low white blood cell counts; 13% of lenalidomide patients and 2% of the placebo group stopped treatment due to side effects.

“This study answers important questions for multiple myeloma patients who undergo a stem cell transplant and want to know if they should get other therapy,” said Principal Investigator Philip McCarthy Jr., MD, Associate Professor and Director of the Blood & Marrow Transplant Program at Roswell Park Comprehensive Cancer Center. “We now know that prolonged maintenance therapy with lenalidomide will delay disease progression compared to placebo. That's important and welcome news, because so many patients have relapse or progressive disease even after a stem cell transplant.”

Lenalidomide is an oral drug that is already used to treat myeloma that recurs or persists despite prior therapy. In this trial, patients were randomized 100-110 days after an autologous stem cell transplant to receive either low-dose lenalidomide, 10 mg initially escalated to 15 mg after three months, or placebo until disease progression. Multiple myeloma occurs when plasma cells, a type of immune cell, become too numerous and crowd out healthy blood cells in the bone marrow, causing pain, and gradually damaging the bones and other body organs.

Multiple myeloma is treated with high-dose chemotherapy and autologous stem cell transplantation — a procedure in which some of a patient's stem cells are removed before therapy and returned after treatment to rebuild the patient's immune system. More than 90 percent of patients, however, eventually experience a cancer relapse. An estimated 20,580 people will be diagnosed with multiple myeloma in the United States in 2010.

After a planned interim analysis in September 2009, the independent Data and Safety Monitoring Committee announced that the study had met its primary endpoint, recommending that the trial be halted early; the study was un-blinded in December 2009.

The study, CALGB 100104, was sponsored by the National Cancer Institute (NCI), part of the National Institutes of Health, and is led by one of its Cooperative Clinical Trials Groups — the Cancer and Leukemia Group B.

About Cancer and Leukemia Group B (CALGB)
The Cancer and Leukemia Group B (CALGB) is a national clinical research group sponsored by the National Cancer Institute. The Chair’s Office is located at Brigham and Women’s Hospital in Boston, MA; Central Office at the University of Chicago and Statistical Center at Duke University in Raleigh, NC. Founded in 1956, the CALGB brings together clinical oncologists and laboratory investigators to develop better treatments for cancer. The Group has a national network of 26 university medical centers, over 225 community hospitals and more than 3000 oncology specialists who collaborate in clinical research studies aimed at reducing the morbidity and mortality from cancer, relating the biological characteristics of cancer to clinical outcomes and developing new strategies for the early detection and prevention of cancer. For more information, visit the CALGB Web site at

About Roswell Park Comprehensive Cancer Center
The mission of Roswell Park Comprehensive Cancer Center is to understand, prevent and cure cancer. Roswell Park, founded in 1898, was one of the first cancer centers in the country to be named a National Cancer Institute-designated comprehensive cancer center and remains the only facility with this designation in Upstate New York. The Institute is a member of the prestigious National Comprehensive Cancer Network, an alliance of the nation’s leading cancer centers; maintains affiliate sites; and is a partner in national and international collaborative programs. For more information, visit Roswell Park’s website at, call 1-800-ROSWELL (1-800-767-9355) or email

Editor’s note: Dr. McCarthy presented the findings of this study, “Phase III intergroup study of lenalidomide versus placebo maintenance therapy following single autologous stem cell transplant for multiple myeloma," at the ASCO meeting Sunday, June 6 at 11am in McCormick Place Convention Center, Room E354B, as part of the Lymphoma and Plasma Cell Disorders oral abstract session.


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Annie Deck-Miller, Senior Media Relations Manager