Patients with multiple myeloma who received autologous hematopoietic cell transplantation (HCT) had longer progression-free survival compared with those who received only chemotherapy using novel agents, early findings that will be presented at the 2016 American Society of Clinical Oncology Meeting in Chicago, IL, have shown.1

Because the role of upfront autologous HCT for newly diagnosed multiple myeloma has been questioned in the era of novel agents, researchers sought to compare outcomes of patients who received intensification therapy with bortezomib, melphalan, and prednisone (VMP) vs high-dose melphalan (HDM) followed by single or double autologous HCT.

For the phase 3 study, researchers enrolled 1503 patients aged 65 years or younger with symptomatic newly diagnosed myeloma to receive induction therapy with bortezomib plus cyclophosphamide and dexamethasone followed by subsequent collection of peripheral blood stem cells. Of those, 1308 were randomly assigned to receive VMP, or HDM with HCT.

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Results showed that at a median follow-up of 24 months, progression-free survival was significantly longer in patients who received HDM compared with VMP (HR, 0.76; 95% CI, 0.61-0.94; P = .010). Subgroup analyses demonstrated a similar benefit across all subgroups, including those with revised ISS stage III (HR, 0.52; 95% CI, 0.32—0.84; P = .008) and those with high-risk cytogenetics (HR, 0.72; 95% CI, 0.54-0.97; P = .028). The study further demonstrated that randomization to HDM was an independent predictor or progression-free survival (HR, 0.61; 95% CI, 0.45-0.82; P = .001).

Researchers also found that significantly more patients achieved a very good partial response with HDM (84%) vs VMP (74%) (OR, 1.90; 95% CI, 1.42-2.54; P < .0001). Overall survival data was not yet mature; however, there appeared to be no difference between the 2 treatment arms.

“Our findings show that autologous stem cell transplant should remain the preferred treatment for patients with multiple myeloma age 65 and under,” said lead study author Michele Cavo, MD, Head of the Seràgnoli Institute of Hematology at the University of Bologna. “While transplant-free treatment with novel agents remains an intriguing prospect, the reality is that stem cell transplant remains a powerful and proven approach, and with novel agents playing a supporting role, it is more effective than ever.”

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Following intensification therapy, patients will be randomly assigned to consolidation therapy or no consolidation therapy, but interim analysis of that data is not yet complete. The study will also assess quality of life and toxicity relating to each therapeutic approach.

“Even in an age of novel therapies, proven approaches can retain their value,” said ASCO President Julie M. Vose, MD, MBA, FASCO. “This study demonstrated that combining the best of both worlds—initial therapy with a novel agent followed by stem cell transplant— resulted in the best patient outcomes.”                  


  1. Stem cell transplant remains important for multiple myeloma, even in novel agent era [news release]. Alexandria, VA: American Society of Clinical Oncology; May 18, 2016. Accessed May 18, 2016.