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Efficacy, safety and cost of pomalidomide in relapsed and refractory multiple myeloma

European Journal of Haematology Feb 07, 2018

Gueneau P, et al. - In this study, a long-term favorable safety-efficacy profile of pomalidomide-dexamethasone regimen was reported in patients with relapsed and refractory multiple myeloma (RRMM), with substantial survival benefit seen among patients with stable disease.

Methods
  • This study included all patients (n=63) treated with pomalidomide-dexamethasone for RRMM in researchers' university hospital between August 2013 and October 2015.

Results
  • Early discontinuation of pomalidomide, due to progression (before the 4th cycle) in 17 (27%) patients, was reported.
  • Findings also showed that no case was discontinued for intolerance.
  • Time from diagnosis to pomalidomide initiation <3 years was identified as the only independent factor that predicted early pomalidomide discontinuation.
  • The reported overall response rate was 51%, including complete response in 8%, very good partial response in 25%, and partial response in 19% patients.
  • Stable disease was exhibited by 13 (33%) patients.
  • Researchers found that the median overall survival was 6.4 months in the 17 patients who discontinued pomalidomide early vs 26.8 months in the 14 patients with stable disease vs not achieved in the 32 responders (log-rank; p<10-3).
  • Neutropenia (14%) and infections (25%) were documented as the most common grade ≥3 adverse events.
  • In addition, the incremental cost-effectiveness ratio of pomalidomide-dexamethasone vs dexamethasone alone was estimated at €39,911 per life-year gained.
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