Multiple Myeloma Medication

Updated: Jan 11, 2018
  • Author: Dhaval Shah, MD; Chief Editor: Emmanuel C Besa, MD  more...
  • Print

Medication Summary

Multiple myeloma (MM) is treated with several categories of medications. Chemotherapeutic agents are used to reduce the disease burden, and bisphosphonates are used to promote bone healing and to provide secondary prophylaxis against skeletal-related events (eg, hypercalcemia, bone fracture, spinal cord compression, need for radiation, and need for surgery). In addition, erythropoietin is used to treat anemia, either alone or in conjunction with chemotherapy.


Chemotherapeutic Agents

Class Summary

The choice of chemotherapy depends on several factors, including the patient’s performance status, age, renal function, desire for inpatient or outpatient therapy, and likelihood of receiving future autologous stem cell transplantation.

In patients with renal failure or highly aggressive disease, therapy with vincristine, Adriamycin (doxorubicin), and dexamethasone (VAD) may be preferred. In elderly patients or patients in whom autologous transplantation is not possible in the future, melphalan and prednisone (MP) therapy is preferred because of its ease of administration and low toxicity. 

Cyclophosphamide (Cytoxan, Neosar)

Cyclophosphamide is chemically related to nitrogen mustards. It is an alkylating agent, and its mechanism of action of active metabolites may involve cross-linking of DNA, which may interfere with growth of normal and neoplastic cells.

Melphalan (Alkeran)

The most widely used regimen is MP. Melphalan is an alkylating agent and a derivative of mechlorethamine that inhibits mitosis by cross-linking DNA strands. It is indicated for the palliative treatment of multiple myeloma.

Doxorubicin (Adriamycin, Rubex)

Doxorubicin is part of VAD therapy. It inhibits topoisomerase II and produces free radicals, which may cause destruction of DNA; these 2 events, in turn, can inhibit growth of neoplastic cells.

Vincristine (Oncovin)

Vincristine inhibits cellular mitosis by inhibition of intracellular tubulin function, binding to microtubules, and synthesis of spindle proteins in the S phase. Vincristine is part of VAD therapy. Its mechanism of action is complex and includes depolymerization of microtubules.

Bortezomib (Velcade)

Bortezomib is the first drug approved in the group of anticancer agents known as proteasome inhibitors. The proteasome pathway is an enzyme complex existing in all cells, which degrades ubiquitinated proteins that control the cell cycle and cellular processes and maintains cellular homeostasis. Reversible proteasome inhibition disrupts pathways supporting cell growth, thus decreasing cancer cell survival. Bortezomib is indicated for patients with multiple myeloma. Development of peripheral neuropathy is a limiting factor. A decreased incidence of peripheral neuropathy has been observed with SC administration compared with the IV route.

Doxorubicin liposomal (Doxil)

Doxorubicin liposomal is a pegylated formulation that protects the liposomes and, thereby, increases blood circulation time. The drug inhibits topoisomerase II and produces free radicals, which may cause destruction of DNA; these 2 events can, in turn, inhibit growth of neoplastic cells.

Carfilzomib (Kyprolis)

Proteasome inhibitor; elicits antiproliferative and proapoptotic activities in vitro in solid and hematologic tumor cells. It is indicated as monotherapy, in combination with dexamethasone, or in combination with lenalidomide plus dexamethasone for relapsed or refractory multiple myeloma in patients who have received at least 1 prior line of therapy.  

Panobinostat (Farydak)

Panobinostat is a histone deacetylase (HDAc) inhibitor. HDAc catalyzes the removal of acetyl groups from the lysine residues of histones and some nonhistone proteins. Inhibition of HDAc activity results in increased acetylation of histone proteins and an epigenetic alteration that results in a relaxing of chromatin, leading to transcriptional activation. It is indicated in combination with bortezomib and dexamethasone for treatment of multiple myeloma in patients who have received at least 2 prior regimens, including bortezomib and an immunomodulatory agent.

Daratumumab (Darzalex)

Monoclonal antibody that binds with high affinity to the CD38 molecule, which is highly expressed on the surface of multiple myeloma cells. It is indicated for patients with multiple myeloma who have received at least 3 prior treatments, including a proteasome inhibitor (PI) and an immunomodulatory agent (IMiD), or who are double-refractory to a PI and IMiD.

Ixazomib (Ninlaro)

Reversible proteasome inhibitor. It preferentially binds and inhibits the chymotrypsinlike activity of the beta 5 subunit of the 20S proteasome. It is indicated in combination with lenalidomide and dexamethasone for patients with multiple myeloma who have received at least 1 prior therapy.

Elotuzumab (Empliciti)

Humanized IgG1 monoclonal antibody that specifically targets the SLAMF7 (signaling lymphocytic activation molecule family member 7) protein. SLAMF7 is expressed on myeloma cells and natural killer cells and plasma cells. Facilitates the interaction with natural killer cells to mediate the killing of myeloma cells through antibody-dependent cellular cytotoxicity. It is indicated in combination with lenalidomide and dexamethasone for multiple myeloma in patients who have received 1-3 prior therapies.



Class Summary

Corticosteroids have anti-inflammatory properties and cause profound and varied metabolic effects. They modify the body’s immune response to diverse stimuli. 

Prednisone (Deltasone, Orasone, Meticorten)

The most widely used regimen is MP. Prednisone stabilizes lysosomal membranes and suppresses lymphocyte and antibody production.

Dexamethasone (Decadron)

Dexamethasone is part of VAD therapy. Many believe that the high-dose steroid component of VAD accounts for much of its efficacy. In some patients, high-dose dexamethasone alone may produce significant clinical responses. Dexamethasone stabilizes lysosomal membranes and suppresses lymphocyte and antibody production.



Class Summary

Interferons are naturally produced proteins with antiviral, antitumor, and immunomodulatory actions. Alfa-, beta-, and gamma-interferons may be administered topically, systemically, and intralesionally. 

Interferon alfa-2B (Intron A)

Interferon alfa-2B is a protein product manufactured by recombinant DNA technology. The mechanism of antitumor activity is not clearly understood; however, direct antiproliferative effects against malignant cells and modulation of host immune response may play important roles.



Class Summary

Bisphosphonates inhibit bone resorption via action on osteoclasts or osteoclast precursors.

Pamidronate (Aredia)

Pamidronate inhibits normal and abnormal bone resorption. It appears to inhibit bone resorption without inhibiting bone formation and mineralization. The optimal timing and duration of therapy are being studied. Pamidronate is administered intravenously (IV) over 2 hours. Newer drugs similar in structure and function are being studied and may have improved efficacy and greater convenience.

Zoledronic acid (Zometa)

Zoledronic acid inhibits bone resorption, possibly by acting on osteoclasts or osteoclast precursors. It is effective in treating the hypercalcemia of malignancy.


Monoclonal Antibodies

Class Summary

Monoclonal antibodies that target RANKL are used to prevent skeletal-related events (SREs).

Denosumab (Xgeva)

Monoclonal antibody that specifically targets RANKL. It binds to RANKL and inhibits its binding to RANK receptor, thereby preventing osteoclast formation. This results in decreased bone resorption and increases bone mass in osteoporosis. RANKL inhibition decreases tumor-induced bone destruction and SREs. Denosumab is indicated for prevention of SREs in patients with multiple myeloma.


Colony-stimulating Factors

Class Summary

Colony-stimulating factors induce erythropoiesis.

Epoetin alfa, erythropoietin (Epogen, Procrit)

Erythropoietin stimulates the division and differentiation of committed erythroid progenitor cells and induces the release of reticulocytes from bone marrow into the blood stream.

Erythropoietin is a naturally occurring hormone produced by the kidneys to stimulate bone marrow production of red blood cells. In patients with MM, administration of exogenous erythropoietin may correct anemia, leading to a significant improvement in performance status and quality of life.


Immunosuppressant Agents

Class Summary

Immunosuppressant agents inhibit key factors in the immune system that are responsible for immune reactions.

Thalidomide (Thalomid)

Thalidomide, when used in combination with dexamethasone, is indicated for the treatment of patients with newly diagnosed multiple myeloma. Thalidomide is an immunomodulatory agent that may suppress excessive production of tumor necrosis factor (TNF)-alpha and may down-regulate selected cell-surface adhesion molecules involved in leukocyte migration. Because of concerns regarding teratogenicity, thalidomide can only be prescribed by registered physicians and is dispensed by registered pharmacists. Patients must participate in ongoing surveys to receive therapy, and only a 28-d supply can be prescribed at a time.

Lenalidomide (Revlimid)

Lenalidomide is indicated in combination with dexamethasone for multiple myeloma. It is structurally similar to thalidomide. Lenalidomide elicits immunomodulatory and antiangiogenic properties. It inhibits proinflammatory cytokine secretion and increases anti-inflammatory cytokines from peripheral blood mononuclear cells.