What is the role of maintenance therapy in the treatment of multiple myeloma (MM)?

Updated: May 11, 2021
  • Author: Dhaval Shah, MD; Chief Editor: Emmanuel C Besa, MD  more...
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In spite of advances in treatment, multiple myeloma remains an incurable disease. To improve overall survival (OS) in these patients, a number of trials have evaluated the role of maintenance therapy in both transplant-eligible and transplant-ineligible patients.

Five large phase III studies have looked at role of thalidomide maintenance after ASCT. Three initial studies showed an improvement in both PFS and OS. [76] However, two subsequent studies—including one large study with 1970 patients—did not show an improvement in OS with thalidomide maintenance. [77, 78] Long-term use of thalidomide is also associated with significant neuropathy, thus limiting its use in maintenance therapy.

Given its favorable toxicity profile and efficacy at low doses, lenalidomide has also been studied for maintenance therapy. Two large trials, CALGB 100104 and IFM 05-02, have evaluated the role of lenalidomide in maintenance therapy, using slightly different protocols and having somewhat different outcomes. [79, 80] Patients in both studies received induction treatment followed by ASCT. In the IFM 05-02 study, however, all patients received 2 months of consolidation treatment with lenalidomide before being randomized to lenalidomide or placebo.

Both studies showed a significant improvement in time to progression (46 vs 27 months in CALGB study and 41 vs 23 months in IFM study). However, CALGB 100104 study showed significant improvement in OS (85 % vs 77 %), whereas IFM 05-02 did not show an improvement in OS. Both studies showed an increased incidence of hematologic toxicity and second primary malignancies (SPMs), particularly acute myelogenous leukemia/myelodysplastic syndrome in the lenalidomide arm.

The reason for the difference in the two studies in terms of OS benefit is not very clear. Since all the patients in the IFM trial received 2 months of consolidation treatment with lenalidomide following ASCT, it is possible that only short period of maintenance therapy, rather than continuous maintenance therapy, is required to achieve all the OS benefit seen in the CALGB trial.

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