The prospective, multicenter, phase III EMN02/HO95 MM trial was designed to randomly compare (R1) (1:1 ratio; stratification by ISS) four 42-day cycles of standard dose bortezomib-melphalan-prednisone (VMP) vs either a single or two sequential courses of melphalan 200 mg/m2 followed by autologous stem cell transplantation (ASCT), as intensification therapy for newly diagnosed multiple myeloma (MM) patients. A second randomization (R2) to consolidation therapy with bortezomib-lenalidomide-dexamethasone vs no consolidation was performed after intensification, to be followed by lenalidomide maintenance until progression or toxicity in both arms. A primary study end point was progression-free survival (PFS) from R1. From February 2011 to April 2014, 1510 patients aged <0.001). In a multivariate Cox regression analysis, randomization to ASCT (HR=0.69; CI=0.55-0.86; P<0.001) and absence of high-risk cytogenetic abnormalities (0.69; CI=0.52-0.91; P=0.008) were the leading independent predictors of prolonged PFS. Overall survival was not yet mature and no difference between the two treatment groups was evident. It is concluded that upfront ASCT still continues to be the reference treatment choice for fit patients with newly diagnosed MM, even in the era of bortezomib-based therapies.
P., T., M., B., M., D., E., Z., F., D. R., F., G., … P., S. (2017). Intensification therapy with autologous stem cell transplantation versus bortezomib-melphalan-prednisone for newly diagnosed multiple myeloma patients: A multicenter, phase III study of the european myeloma network (EMN02/HO95 MM trial). Haematologica, 102(Supplement 3).