Initial results from a large, randomized clinical trial for patients with multiple myeloma 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. This clinical trial, for patients ages 18 to 70, was sponsored by the NCI and conducted by a network of researchers led by the Cancer and Leukemia Group B (CALGB) in collaboration with ECOG and the BMT CTN.
The independent data and safety monitoring committee overseeing the trial (known as CALGB-100104) found that the time to disease progression following autologous blood stem cell transplantation for those patients on lenalidomide was significantly longer than for those on placebo and so the trial was stopped early. 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 and was highly statistically significant. This is the first randomized phase 3 trial to demonstrate a clinical benefit of lenalidomide following transplant for multiple myeloma.