Study: Stem cell transplant should remain preferred therapy for multiple myeloma

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Patients with multiple myeloma who underwent autologous stem cell transplantation (ASCT) survived longer without disease progression than those who received only chemotherapy with novel agents, according to early findings from a phase 3 clinical trial. The findings, presented at the annual meeting of the American Society of Clinical Oncology, held June 3–7, 2016, in Chicago, Illinois, were from the largest study to date aimed at comparing ASCT with a bortezomib-based regimen alone in patients younger than 65 years of age. The proteasome inhibitor bortezomib was approved by the US Food and Drug Administration in 2008 for the upfront treatment of multiple myeloma. Since then, it has been incorporated into the standard treatment for patients with newly diagnosed multiple myeloma, whether or not they are able to undergo ASCT. The continued need for ASCT for patients younger than age 65 years has been debated in this era of novel agents such as bortezomib. Lead author Michele Cavo, MD, head of the Seràgnoli Institute of Hematology at the University of Bologna in Italy, notes that although transplant-free treatment with novel agents continues to be an “intriguing prospect,” stem cell transplantation still remains an important and proven approach, with novel agents playing a supporting role. The study included 1266 patients newly diagnosed with multiple myeloma. After induction therapy with bortezomib, cyclophosphamide, and dexamethasone, patients were randomly assigned to receive either bortezomib, melphalan, and prednisone (VMP) or high-dose melphalan followed by a single treatment with ASCT. In the study's second phase, patients in both groups were randomly assigned to either consolidation therapy with bortezomib, lenalidomide, and dexamethasone or no consolidation therapy. All patients received maintenance therapy with lenalidomide until disease progression or intolerable toxicity. The median follow-up after the first treatment randomization was 2 years. Data showed that patients who received stem cell transplants showed slower disease progression than those who received VMP therapy without transplantation. Among patients who had not yet experienced disease progression, those in the ASCT arm had a 24% lower risk of progressing at any future time point in comparison with patients who did not receive the transplant. In a further multivariate analysis, the benefit of transplantation was even greater among certain patients at high risk for early progression. Patients with advanced disease who were randomized to the ASCT arm had a 48% lower chance of having progressed by the next analysis in comparison with those not undergoing transplantation. Furthermore, among patients with certain high-risk genetic factors, ASCT was associated with a 28% lower chance of future progression in comparison with VMP therapy without transplantation. The study is ongoing and will include analyses of overall survival, toxicity, and quality of life.