Comparative Effectiveness Study Examined Treatment Modalities for Multiple Myeloma


In a recent study, researchers evaluated the role of autologous stem cell transplantation (ASCT) in patients with multiple myeloma in the context of novel agents, published in JAMA Oncology (online January 4, 2018; doi:10.1001/jamaoncol.2017.4600).

For the past 20 years, high-dose therapy with melphalan followed by ASCT (HDT/ASCT) has been considered the standard treatment for newly diagnosed patients with multiple myeloma. However, the role of HDT/ASCT in the context of novel agent induction continues to be debated.

Binod Dhakal, MD, MS, division of hematology/oncology, Medical College of Wisconsin, Milwaukee, and colleagues conducted a systematic review and meta-analysis of all phase III randomized clinical trials evaluating the role of HDT/ASCT. A systematic literature search was performed in Cochrane Central, MEDLINE, and Scopus from 2000 through 2017 to obtain four trials (2421 patients) for conventional meta-analysis and five trials (3171 patients) for network meta-analysis.

Criteria for trial selection included comparative design of HDT/ASCT with standard-dose therapy using novel agents, as well as comparative design with HDT/ASCT with bortezomib, lenalidomide, and dexamethasone consolidation and tandem transplantation.

Results of the analyses showed that the combined odds for complete response were 1.27 (95% CI, 0.97-1.65; P = .07) with HDT/ASCT when compared with standard-dose therapy. Additionally, the combined hazard ratio for progression-free survival (PFS) was 0.55 (95% CI, 0.41-0.74; P < .001) and 0.76 for overall survival (OS; 95% CI, 0.42-1.36; P = .20) in favor of HDT.

A meta-regression showed that longer follow-up was associated with superior PFS and OS. For the PFS section of the analysis, tandem HDT/ASCT had the most favorable hazard ratio (0.49; 95% CI, 0.37-0.65), followed by single HDT/ASCT with bortezomib, lenalidomide, and dexamethasone (HR, 0.53; 95% CI, 0.37-0.76) and single HDT/ASCT alone (HR, 0.68; 95% CI, 0.53-0.87) compared with standard-dose therapy.


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Researchers noted that for OS, none of the HDT/ASCT-based approaches had a significant effect on survival.

In their concluding remarks, researchers wrote that HDT/ASCT-based approaches were associated with superior PFS when compared with standard-dose therapy for multiple myeloma. “Longer follow-up may better delineate any OS benefit; however, is likely to be affected by effective post-relapse therapy,” they added.—Zachary Bessette