The myeloma community has been encouraged by a new study of Celgene Corp's Revlimid which shows its effectiveness in patients whose disease has not yet progressed.
The study, published in the New England Journal of Medicine, evaluated treatment with Revlimid (lenalidomide) in combination with dexamethasone followed by maintenance therapy with the Celgene drug, in patients with high-risk asymptomatic smouldering multiple myeloma. The latter refers to myeloma diagnosed so early there are no symptoms.
The Phase III trial involved 119 patients with high-risk smouldering myeloma split into an arm receiving treatment (57 patients) with the other group just being observed, which is the standard of care. An evaluation three years into the study shows that 77% of patients who were treated remained without symptoms but only 30% of patients in the 'watch-and-wait' group were progression free.
The study concludes that this delay in disease progression)translated into a significant overall survival benefit, noting that at three years, 94% of patients in the treatment arm were alive, compared to 80% in the just-observed group.
Brian Durie, chairman of the International Myeloma Foundation, said "the challenge now is to identify which patients stand to benefit from these findings”. He noted that the study used specific bone marrow characteristics protein secretions and other diagnostic criteria to identify high-risk patients and experts in the field .are looking "to further refine the criteria to determine which patients fit into this high-risk category that could be as much as 40% of asymptomatic patients.”
Susie Novis, IMF president, said these findings might change when myeloma patients are treated and also improve access to effective drugs. However, she noted that in Europe, where this study was conducted, this oral regimen is not approved as a first-line regimen for newly diagnosed patients, so when they progressed to symptomatic myeloma "and their role in the study ended they were not given this regimen. Our advocacy team is working to improve global access to effective therapies".
Revlimid is approved in combination with dexamethasone for multiple myeloma. It is also approved in the USA for mantle cell lymphoma and transfusion-dependent anaemia due to low- or intermediate-1–risk myelodysplastic syndromes (MDS).