What are treatment options for multiple myeloma (MM)?

Updated: May 11, 2021
  • Author: Dhaval Shah, MD; Chief Editor: Emmanuel C Besa, MD  more...
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Overall, the care of patients with MM is complex and should focus on treatment of the disease process and any associated complications. [3, 5] Although MM remains incurable, several drug therapies are valuable in the treatment of patients with MM, as are autologous stem cell transplantation, radiation, and surgical care in certain cases.

Several studies are evaluating the role of treatment in patients with high-risk smoldering multiple myeloma (SMM). Previous smaller studies evaluating thalidomide did not show a clear evidence of benefit with treatment in patients with SMM; however, these included patients with all risk levels of SMM.

In a phase III trial that was restricted to patients with high-risk SMM, the PETHEMA group found evidence of benefit from treatment with lenalidomide versus observation. After a median follow-up of 40 months, study patients who were randomized to lenalidomide and dexamethasone induction followed by lenalidomide maintenance demonstrated significantly prolonged median time to progression (median not reached vs 21 months) and higher 3-year survival rate (94% vs. 80%). [40]

Lenalidomide as single-agent therapy (without dexamethasone induction) may also slow progression of SMM to MM. A phase III trial in 182 patients found that after 3 years, SMM had not progressed to MM in 91% of patients receiving lenalidomide, compared with 66% of those who underwent observation only.  Many patients stopped taking lenalidomide early due to side effects (eg, fatigue, neutropenia); however, preliminary results suggest that even a short course of treatment may be beneficial. [41]

In addition, the success of three-drug combinations for MM has led to trials of their use in SMM. Triplets currently under study  for smoldering MM include carfilzomib, lenalidomide, and dexamethasone and daratumumab, lenalidomide, and dexamethasone.

Longer follow-up will be necessary, however. Concern for second primary malignancies (SPMs) with the use of lenalidomide is also a significant issue. Consequently, watchful observation and frequent monitoring remains the standard of care for patients with SMM. 

Patients with MM for whom therapy is indicated typically receive chemotherapy. Greater understanding of the cell biology of MM and the ability to identify prognostic factors has led to the increasing individualization of treatment for affected patients. Physicians treat many patients with high-dose therapy and peripheral blood or hematopoietic stem cell transplantation.

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