Initial results from a large, randomized clinical trial for patients with multiple myeloma, a cancer of the blood and bone marrow, showed that patients who received the oral drug lenalidomide (Revlimid, also known as CC-5013) following a blood stem cell transplant had their cancer kept in check longer than patients who received a placebo.
The clinical trial, for patients ages 18 to 70, was sponsored by the National Cancer Institute (NCI), and conducted by a network of researchers led by the Cancer and Leukemia Group B (CALGB) in collaboration with the Eastern Cooperative Oncology Group (ECOG) and the Blood and Marrow Transplant Clinical Trials Network (BMT CTN). The BMT CTN is co-sponsored by NCI and the National Heart, Lung, and Blood Institute, both parts of the National Institutes of Health.
The independent data and safety monitoring committee overseeing the trial (known as CALGB-100104) found that the study demonstrated a longer time before the cancer progressed following autologous blood stem cell transplantation for those patients on the study drug than those on placebo and so the trial was stopped early.
Autologous blood stem cell transplantation is a procedure in which a patient's own blood stem cells are removed, the patient is then treated with high doses of chemotherapy and/or radiation therapy to kill the cancer, after which the blood stem cells are returned to the patient. It is a common procedure for patients with multiple myeloma.
A total of 568 patients with multiple myeloma, who had received no more than 12 months of prior therapy and no prior transplant, were enrolled between December 2004 and July 2009. All patients received autologous transplantation following a high dose of a drug called melphalan, which is commonly used to treat multiple myeloma.
Ultimately, 460 patients who had adequate organ function and no evidence of progressive disease, were randomized between 90 and 100 days after transplant to receive lenalidomide or placebo. Patients began lenalidomide or placebo between day 100 to 110 and continued until they had evidence of progressive disease.
Among the patients who received placebo, half had their myeloma progress (worsen) within an estimated 778 days. In contrast, for those patients taking lenalidomide, a median time to progression cannot be defined because fewer than half the patients had worsening of their myeloma. This represents a 58 percent reduction in the risk of disease progression for the group taking lenalidomide. This difference in time to progression was highly statistically significant.
This is the first randomized phase 3 trial (the final and most comprehensive aspect of a three-phase clinical trials process) to demonstrate a clinical benefit of lenalidomide following transplant for multiple myeloma. However, the trial has not yet shown evidence of an overall survival benefit.
The types of side effects observed in this trial were similar to those observed in other clinical trials with lenalidomide. Detailed results from this trial will be presented at a future scientific meeting, NCI informs.