Pomalidomide plus dexamethasone is a safe and effective treatment option leading to positive long-term outcomes among patients with relapsed/refractory (R/R) multiple myeloma (MM), according to a study published in the European Journal of Haematology.

Multiple myeloma is associated with very poor prognosis, making the cost of treatment an important factor in determining management strategy. Investigators assessed the safety, efficacy, and cost effectiveness of pomalidomide in the treatment of R/R MM.

For this retrospective study, researchers assessed the outcomes of 63 patients with R/R MM who were treated with oral pomalidomide 4 mg daily plus dexamethasone. To be eligible for the study, patients must have received 2 or more prior antimyeloma therapies, including 2 or more cycles of lenalidomide and bortezomib.

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The 3-month overall response rate was 51%, of which 8% of patients had a complete response, 25% had a very good partial response, and 19% had partial response. Thirty-three percent of patients achieved stable disease.

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Median overall survival (OS) was 6.4 months in 17 patients who discontinued pomalidomide early vs 26.8 months in 15 patients with stable disease; median OS was not evaluable among the 34 responders to therapy.

The most frequently reported grade 3 or worse adverse events included neutropenia and infections.

For the 63 patients with a follow-up of 28.0 months, the total cost of treatment was €4,702,011; the incremental cost-effectiveness ratio of pomalidomide plus dexamethasone vs dexamethasone alone was approximately €39,911 per life-year gained.

The authors concluded that the long-term safety-efficacy profile of pomalidomide-dexamethasone regimen for patients with R/R MM is favorable. “The survival benefit of this treatment is substantial even in patients with stable disease. Further economic analyses are required to determine the best cost-effective strategy in R/R MM.”


Gueneau P, Chretien ML, Cansac-Miet A, et al. Efficacy, safety, and cost of pomalidomide in relapsed and refractory multiple myeloma [published online February 2, 2018]. Eur J Haematol. doi: 10.1111/ejh.13039