First-line autologous stem cell transplant (ASCT) is cost-effective in patients with newly diagnosed multiple myeloma (MM) but more research is needed to assess cost-effectiveness of other, newer antimyeloma regimens in different treatment-line settings, according to a systematic review of studies from 11 countries published over the past quarter of a century. The analysis was published in the journal PharmacoEconomics.

“For transplant-eligible MM patients, transplant is a cost-effective first-line treatment,” reported senior study author Professor David R. Lairson, PhD, co-director of the Center for Health Services Research at The University of Texas Houston Health Science Center School of Public Health in Houston, and coauthors.

However, the evidence for the relative cost effectiveness of other treatment regimens remains unclear and more research is needed, the researchers noted. Second-line bortezomib-based regimens, lenalidomide plus dexamethasone, and pomalidomide plus dexamethasone were each cost-effective compared with dexamethasone alone but which of these combinatorial regimens is the most cost-effective remains unclear.

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MM incidence rates have climbed in recent years, as baby boomers aged into late adulthood. MM treatment strategies have been changing rapidly over recent years, as well, thanks to newly approved agents and treatment regimens. Newer regimens are promising and benefit some patients; however, none are considered curative.

Previous systematic reviews have compared specific therapies. However, few comprehensive cost-effectiveness analyses have been undertaken that compare available therapies across treatment lines.

To compare the cost effectiveness of treatment regimes, the authors searched research literature databases and identified 24 such assessments, published between 1990 and 2018, summarizing incremental cost-effectiveness ratio (ICER), quality-adjusted life-year (QALY), and life-year gained (LYG) for different treatment regimens and treatment lines (first-line, second-line, and multiple-line treatments).

Unpublished literature, including some industry-sponsored studies and reports, or non-English reports, were not included, cautioned the researchers. In addition, 2 studies were not available in the databases searched and their exclusion from the analysis might have biased their results, the team acknowledged.

Four studies included in the review had compared transplant and no-transplant treatments’ cost-effectiveness. Among transplant-eligible patients, first-line transplant was “the most cost-effective option,” with an ICER of at least $4053 per QALY gained and between $3848 and $72,852 per LYG. Compared with conventional chemotherapy, first-line novel agents (bortezomib, thalidomide, and lenalidomide) had an ICER of $59,076 per QALY gained and $220,681 per LYG. ASCT in patients with new diagnoses was cost effective compared with melphalan, the researchers reported (ICER of $25,710 per LYG).

Second-line novel agent regimens had “inconsistent” ICERs, the researchers reported.

“More cost-effectiveness analyses comparing novel agents in the first-line treatment regimen are warranted to determine which agent or regimen is the most cost-effective,” the researchers concluded.

“[T]he papers included in this review were conducted from different perspectives, countries, and years, and using different cost-effectiveness criteria,” the researchers noted. “[R]eaders need to use caution when interpreting the conclusions in the context of their local jurisdiction and cost-effectiveness criteria.”

Multiple myeloma caused 12,590 deaths in 2017, representing 2% of all US cancer deaths.


  1. Fu S, Wu CF, Wang M, Lairson DR. Cost effectiveness of transplant, conventional chemotherapy, and novel agents in multiple myeloma: a systematic review [published online August 7, 2019]. PharmacoEconomics. doi:10.1007/s40273-019-00828-y

This article originally appeared on Oncology Nurse Advisor