Multiple Myeloma: Key Insights on Diagnosis and Evolving Treatment Strategies

Multiple myeloma is a rare but serious hematologic malignancy characterized by abnormal plasma cell proliferation and excessive production of monoclonal (M) proteins. Although the lifetime risk is low, the disease carries significant morbidity, especially due to bone destruction, cytopenias, and immune suppression.¹ For pharmacists, understanding the evolving therapeutic landscape is essential—not only to optimize drug selection and dosing but also to counsel patients on complex regimens, manage adverse effects, and monitor for treatment-related toxicities. With recent advancements in induction therapies, stem cell transplantation strategies, and novel maintenance options, pharmacists play a critical role in improving outcomes and quality of life for patients with multiple myeloma.

Multiple myeloma | Image credit: jarun011 | stock.adobe.com

Epidemiology and Risk Factors

The American Cancer Society predicts that in 2025 there will be around 36,110 cases of multiple myeloma diagnosed in the United States, with a higher number of cases in men than women. Furthermore, there will be approximately 12,030 deaths expected for the year 2025 due to multiple myeloma.²

For the average person living in the United States, the lifetime risk of being affected by multiple myeloma is very low, as it is estimated to be less than 1%, with men being more affected by this disease than women.² The age group mostly affected by this disease is between the ages of 40 and 70 years old, although risk increases with age.¹

Multiple myeloma can affect various organs in the body, including the blood and bones, leading to serious health conditions. Because the abnormal plasma cells in multiple myeloma are less effective than normal plasma cells, this can result in blood disorders such as anemia, thrombocytopenia, and leukopenia. Furthermore, increased bone breakdown in multiple myeloma can lead to elevated calcium levels, resulting in hypercalcemia. This can cause symptoms such as thirst, urination, and dehydration.³

Diagnostic Workup and Disease Staging

Diagnosis of multiple myeloma typically requires multiple laboratory tests, including blood tests, urine tests, bone marrow tests, and imaging. Blood tests are significant because they may identify proteins made by myeloma cells, such as M protein or b-2-microglobulin. Urine tests may indicate that there are M proteins in the urine, and bone marrow biopsies can determine whether there are myeloma cells. Finally, imaging tests such as X-rays, MRIs, CT scans, or PET scans are significant because they can indicate bone damage associated with multiple myeloma.⁴

Multiple myeloma has 3 stages that indicate the rate at which myeloma is growing. Stage 1 indicates that the myeloma is growing slowly, whereas the higher the stage, the more rapid the growth. Multiple myeloma can also be assessed using a risk level, and it is imperative to use the stage and level in order to create an effective treatment plan that is individualized to each patient's specific needs.⁴

First-Line and Standard-of-Care Therapies

When treating multiple myeloma, treatment goals include lessening the amount of M protein in the body, ridding the bone marrow of myeloma cells, promoting longevity, reducing the risk of relapse, and increasing overall survival rates.⁵

For individuals with newly diagnosed multiple myeloma, the standard of care consists of induction therapy followed by an autologous stem cell transplant as well as maintenance therapy. Induction therapy is based on either triplet therapy or quadruplet therapy. These regimens last 3 to 4 weeks and are given for a duration of 3 to 4 cycles. The 3-drug regimen consists of an immunomodulatory drug, a proteasome inhibitor, and a steroid. The 4-drug regimen is inclusive of all the drugs in a 3-drug regimen, as well as an anti-CD38 monoclonal antibody.

With regard to autologous stem cell transplants, eligible candidates will receive induction therapy, then stem cell collection, high-dose melphalan chemotherapy, and the transplant. Subsequently, this is followed by consolidation and maintenance therapy. If the patient is ineligible for a transplant, they will proceed directly to consolidation or maintenance therapy after induction therapy. The typical options for maintenance therapy include lenalidomide (Revlimid; Bristol Myers Squibb), followed by bortezomib (Velcade; Takeda) and ixazomib (Ninlaro; Takeda) if lenalidomide is not tolerable.⁵

The standard pharmacotherapies for multiple myeloma include a variety of drugs, such as protease inhibitors, immunomodulatory drugs, antibodies, B-cell maturation antigen therapies, steroids, and chemotherapy. Proteasome inhibitors, in particular, are crucial drugs because they can be used at all stages of the disease. Currently, the 3 protease inhibitors approved to treat this disease are bortezomib, Kyprolis carfilzomib (Kyprolis; Amgen), and ixazomib. Although all 3 options are considered first-line agents, their differing routes of administration could affect the choice of therapy. Bortezomib is a subcutaneous injection and can be administered as a monotherapy, whereas carfilzomib is an intravenous medication that can be administered alone or in combination with another drug. Finally, ixazomib is an oral medication.⁵

Managing Adverse Effects and Patient Counseling Points

All of these treatments do have potential adverse effects. It is imperative to encourage patients to be in communication with their care team to address any concerns related to the treatment plan. Some adverse effects to educate patients about include gastrointestinal symptoms, such as constipation, diarrhea, nausea, and vomiting. Other adverse effects can include decreased blood cell counts, which should be monitored by the health care provider or the specialist. Because multiple myeloma results in a weakened immune system, patients should be informed that treatments can also increase the likelihood of developing an infection due to the low white blood cell count.⁵

Every patient with multiple myeloma should have a specific treatment plan catered to their specific needs, based on the staging and level of disease progression. Due to recent treatment advancements, there is a growing body of treatment options for this patient population, particularly with early detection.

REFERENCES

1. Multiple Myeloma. Cleveland Clinic. Updated August 18, 2025. Accessed August 27, 2025. https://my.clevelandclinic.org/health/diseases/6178-multiple-myeloma

2. Key Statistics About Multiple Myeloma. American Cancer Society. Updated February 28, 2025. Accessed August 27, 2025. https://www.cancer.org/cancer/types/multiple-myeloma/about/key-statistics.html

3. What Is Multiple Myeloma? American Cancer Society. Updated February 28, 2025. Accessed August 27, 2025. https://www.cancer.org/cancer/types/multiple-myeloma/about/what-is-multiple-myeloma.html

4. Multiple Myeloma. Mayo Clinic. December 20, 2024. Accessed August 27, 2025. https://www.mayoclinic.org/diseases-conditions/multiple-myeloma/diagnosis-treatment/drc-20353383

5. Treatments for Multiple Myeloma. Multiple Myeloma Research Foundation. Accessed August 27, 2025. https://themmrf.org/diagnosis-and-treatment/treatment-options/