Role of new treatment approaches in defining treatment goals in multiple myeloma – the ultimate goal is extended survival

SummaryMultiple myeloma is a chronic disease for which there is currently no cure, thus, the overall goal of treatment is to improve survival. Few patients achieved a complete response (CR) with conventional chemotherapy regimens; however, this has changed with the use of high-dose therapy followed by autologous stem cell transplantation and the advent of novel therapies, such as thalidomide, lenalidomide, and bortezomib. With more patients achieving CR and studies reporting that achievement of CR correlates with survival, the role of CR as an endpoint in myeloma therapy has gained prominence. However, there is evidence that the benefit of CR is not the same with all treatment regimens, and that CR is not associated with improved survival in all patients, but mainly in poor-risk patients with more aggressive myeloma. In addition, recent evidence suggests that continued therapy with novel agents may improve the depth of response and/or prolong CR. Finally, despite improvements in response rates, time to progression, and survival achieved with novel therapies in both relapsed/refractory and newly diagnosed myeloma patients, none of the available drugs have been shown to be curative. Therefore, at this time, extended survival should remain the goal of myeloma therapy.