Transplant Still Preferred Treatment Choice in Young Patients With Myeloma

Autologous stem cell transplantation (ASCT) is still the preferred treatment of choice for patients under the age of 65 years with newly diagnosed multiple myeloma (NDMM), according to early results from a phase 3 trial presented during a press briefing ahead of the 2016 American Society of Clinical Oncology Annual Meeting (June 3-7, 2016; Chicago, IL).

For the study, investigators led by Michele Cavo, head of the Seràgnoli Institute of Hematology at the University of Bologna, Italy, enrolled 1266 patients with NDMM, all of whom were 65 years old or younger. After induction therapy with bortezomib, cyclophosphamide, and dexamethasone, all patients were randomly assigned to receive either bortezomib, melphalan, and prednisone (MVP) or high-dose melphalan (HDM) followed by single ASCT.


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At a planned interim analysis performed in January 2016, progression-free survival—the study’s primary endpoint—was significantly improved (24%) in patients who were treated with HDM followed by single ASCT, a trend that was retained across predefined patient subgroups. In patients classified as having high-risk cytogenetics and those with revised International Staging System stage III disease, progression-free survival was improved by 28% and 48%, respectively, among those treated with HDM and ASCT.

In addition, transplant was also associated with an improved quality of response, with 84% of patients undergoing ASCT achieving a very good partial response compared with only 74% of patients treated with MVP.

Commenting on the study, ASCO President Julie M. Vose, MD, MBA, FASCO said: “I think this is a very important, very large study for those of us who take care of multiple myeloma patients, and we can now truly say that we have data to say that transplantation is still important even in the era of novel treatment.”

Thus, researchers concluded that upfront ASCT remains the preferred treatment for younger patients with NDMM. Results from the second stage of the study, where patients in both groups were randomly assigned to consolidation therapy with bortezomib, lenalidomide, and dexamethasone or no consolidation therapy with maintenance lenalidomide until progression, will be presented later once an interim analysis of those data are complete.