Induction Therapy plus Autologous Stem-Cell Transplantation Still Cost-Effective for Newly Diagnosed Multiple Myeloma

Chase Doyle

February 2017, Vol 8, No 1 - Value-Based Care

San Diego, CA—Although considered a standard of care for patients with newly diagnosed multiple myeloma, the benefit of autologous stem-cell transplantation (ASCT) has been recently questioned as a result of the rapid expansion of novel treatments.

A study comparing the cost-effectiveness of ASCT versus nontransplantation regimens with regard to progression-free survival (PFS) may put these questions to rest—for the moment, at least, reported Seema Niphadkar, DO, Hematology/Oncology, Drexel University College of Medicine, Philadelphia, at the 2016 American Society of Hematology meeting.

In this study, a regimen of lenalidomide (Revlimid) and dexamethasone plus ASCT, followed by lenalidomide maintenance therapy was shown to be effective for the treatment of patients with newly diagnosed multiple myeloma.

In addition, the cost associated with induction therapy plus ASCT represented only 22% of the total cost of treatment, whereas lenalidomide maintenance therapy accounted for 78% of the total cost of treatment. These findings underscore the need for alternative options for maintenance therapy, Dr Niphadkar suggested.

In Search of Optimal Therapy

“In an era of cost-consciousness, ASCT should continue to be an integral component of multiple myeloma treatment,” said Dr Niphadkar. “Considering secondary risks and the cost involved with lenalidomide maintenance, though, further investigation is warranted regarding the optimal duration of maintenance therapy and resultant PFS,” she added.

Numerous studies have shown an improvement in complete response and PFS with ASCT, but few have compared the cost-effectiveness of ASCT with novel therapeutic agents.

Using the Healthcare Cost and Utilization Project’s Nationwide Inpatient Sample, Dr Niphadkar and colleagues queried ICD-9 codes for multiple myeloma and ASCT between the years 2008 and 2013. All patients aged ≥18 years who underwent ASCT were included in the study.

The team examined the cost of ASCT admission and the length of stay. The cost of hospitalization was adjusted for inflation in reference to the year 2011 and the cost-to-charge ratio. Common treatment regimens, response rates, duration of treatment, and PFS were also reviewed in the literature.

A previous study by Gay and colleagues reported a median PFS of 43.4 months with 4 cycles of lenalidomide and dexamethasone, followed by ASCT and lenalidomide maintenance therapy until disease progression or unacceptable toxicity, for comparison with new drugs (Gay F, et al. Lancet Oncol. 2015;16:1617-1629).

The cost of novel regimens was estimated, based on a previous study by Roy and colleagues, using the 1-month commercial cost and adjunct cost for an equivalent duration of 43.4 months (Roy A, et al. Am Health Drug Benefits. 2015;8:204-215).

Cost of ASCT versus Drug Therapy

Between 2008 and 2013, a total of 9039 hospitalizations were reported for patients with multiple myeloma. The average length of stay related to admission for ASCT was 11.4 days, resulting in a cost of $109,856. There was a 16.89% decrease in the length of stay and a 1.99% increase in the cost of ASCT (P <.05).

Gay and colleagues reported a 43.4-month PFS with 4 cycles of lenalidomide and dexamethasone ($46,216), followed by ASCT ($109,856) and lenalidomide maintenance therapy (approximately 35.4 months; $564,453 adjusted for cost of office visits and laboratory work), for a total cost of $720,525.

“These data indicate that the majority of novel agents used in combination regimens are more expensive than ASCT,” said Dr Niphadkar. “Although most patients would not remain on one of these regimens for 43.4 months, they would presumably progress through multiple lines of therapy during the 43.4 months of PFS that ASCT provides,” she added.

Although bortezomib (Velcade) and ixazomib (Ninlaro) were shown to be the more cost-effective treatment options than the other regimens, studies to assess their efficacy in terms of PFS in the maintenance setting are still ongoing, said Dr Niphadkar.

Studies using novel agents for induction therapy before ASCT are also in progress and have yet to reach an optimal duration of therapy.

“It’s very interesting that the lenalidomide maintenance pill (10 mg) is more expensive than the 25-mg chemo version. There’s also the question of how long to continue maintenance given the risk of second malignancies. An optimal treatment duration has yet to be defined,” said Dr Niphadkar. “Right now, transplant is still a cost-effective option, but as newer, cheaper drugs come to market, who knows how long that will last,” she added.