Multiple Myeloma: Treatment Options

Approved by the Cancer.Net Editorial Board, 10/2016

ON THIS PAGE: You will learn about the different ways doctors use to treat people with this type of cancer. To see other pages, use the menu.

This section tells you the treatments that are the standard of care for this type of cancer. “Standard of care” means the best treatments known. When making treatment plan decisions, patients are also encouraged to consider clinical trials as an option. A clinical trial is a research study that tests a new approach to treatment. Doctors want to learn if it is safe, effective, and possibly better than the standard treatment. Clinical trials can test a new drug, a new combination of standard treatments, or new doses of standard drugs or other treatments. Your doctor can help you consider all your treatment options. Research for new myeloma treatments is very active, and many clinical trials are offered. To learn more about clinical trials, see the About Clinical Trials and Latest Research sections.

Treatment overview

In cancer care, different types of doctors often work together to create a patient’s overall treatment plan that combines different types of treatments. This is called a multidisciplinary team. Cancer care teams also include a variety of other health care professionals, including physician assistants, oncology nurses, social workers, pharmacists, counselors, dietitians, and others.

The treatment of multiple myeloma depends on whether the patient is experiencing symptoms (see the Stages section) and the patient’s overall health. In many cases, a team of doctors will work with the patient to determine the best treatment plan. The goals of treatment are to eliminate myeloma cells, control tumor growth, control pain, and allow patients to have an active life. While there is no cure for multiple myeloma, the cancer can be managed successfully in many patients for years.

Descriptions of the most common treatment options for multiple myeloma are listed below. They are divided into treatment options for people without symptoms and for people with symptoms. In addition, treatment options may depend on whether the patient is newly diagnosed with myeloma or is experiencing a recurrence of the disease. Your care plan may also include treatment for symptoms and side effects, an important part of cancer care. Take time to learn about all of your treatment options, including clinical trials, and be sure to ask questions about things that are unclear. Also, talk about the goals of each treatment with your doctor and what you can expect while receiving the treatment. Learn more about making treatment decisions.

Active surveillance for patients without symptoms

People with early-stage myeloma and no symptoms, called SMM (see Stages), may simply be closely monitored by the doctor through checkups. This approach is called active surveillance or watchful waiting. As noted previously, if there is evidence of bone thinning, or osteoporosis, periodic infusions of bisphosphonates to reverse this process may be recommended. There are also clinical protocols used to evaluate whether using targeted therapy or immunotherapy can prevent or delay myeloma from developing into a disease that requires treatment. If symptoms appear, then active treatment starts. Currently, clinical trials are being done to identify and treat patients with “high-risk” SMM, which means those who are at risk of developing symptoms within 18 months to 2 years. 

Overview of treatment options for patients with symptoms

Treatment for people with symptomatic myeloma includes both treatment for the disease as well as supportive therapy to improve quality of life, such as by relieving symptoms and maintaining good nutrition. Disease-directed treatment typically includes drug therapy, such as targeted therapy and/or chemotherapy, with or without steroids. Stem cell transplantation may be an option. Other types of treatments, such as radiation therapy and surgery, are used in specific circumstances. Each type of treatment is described below.

The treatment plan includes different phases.

  • Induction therapy for rapid control of cancer and to help relieve symptoms

  • Consolidation with more chemotherapy or stem cell transplant

  • Maintenance therapy over a prolonged period to prevent cancer recurrence

Targeted therapies

Targeted therapy is a treatment that targets the cancer’s specific genes, proteins, or the tissue environment that contributes to cancer growth and survival. This type of treatment blocks the growth and spread of cancer cells while limiting damage to healthy cells. In recent years, targeted treatment, sometimes called novel therapy, has proven to be increasingly successful at controlling myeloma and improving prognosis. Researchers continue to investigate new and evolving drugs for this disease in clinical trials.

Recent studies show that not all tumors have the same targets. To find the most effective treatment, your doctor may run tests on cancer cells to identify genes, proteins, and other factors. This helps doctors better match each patient with the most effective treatment whenever possible.

  • Lenalidomide (Revlimid), pomalidomide (Pomalyst), and thalidomide (Synovir, Thalomid) are drugs that stop the growth of myeloma cells in the bone marrow. These drugs strengthen the immune cells so they attack cancer cells. They starve the cancer cells by blocking the formation of new blood vessels, a process called angiogenesis.

  • Bortezomib (Velcade), carfilzomib (Kyprolis), and ixazomib (Ninlaro) are classified as proteasome inhibitors. They target specific enzymes called proteasomes that digest proteins in the cells. Because myeloma cells produce a lot of proteins (see the Introduction), they are particularly vulnerable to this type of drug.

  • Panobinostat (Farydak) is an inhibitor of the enzyme histone deacetylase (HDAC). HDACs help keep the DNA tightly coiled, while panobinostat helps uncoil the DNA and activate genes that stop or slow the growth of cancer cells. It also blocks an escape route called the aggresome.

  • Elotuzumab (Empliciti) and daratumumab (Darzalex) are monoclonal antibodies that bind to myeloma cells and label them for removal by the patient’s own immune system.

Thalidomide, lenalidomide, and bortezomib are targeted, novel therapies approved to treat newly diagnosed patients. Lenalidomide, pomalidomide, bortezomib, carfilzomib, panobinostat, ixazomib, elotuzumab, and daratumumab are effective for treating recurrent myeloma. Targeted therapies may also be used in combination with chemotherapy or steroid medications (see below), because certain combinations of drugs can sometimes have a better effect than a single drug. For example, the drugs lenalidomide, bortezomib, and dexamethasone, as well as bortezomib, cyclophosphamide, and dexamethasone, are offered in combination as initial treatment. Clinical trials are exploring whether the combination of lenalidomide, bortezomib, and dexamethasone may be as effective as lenalidomide, bortezomib, and dexamethasone followed by stem cell transplant (see below).

Thalidomide, lenalidomide, and bortezomib can also be effectively used as maintenance therapy to extend the response to the initial therapy or after a stem cell transplant. However, the decision to undergo a stem cell/bone marrow transplant is complex and should be discussed carefully with your doctor.

Research has shown that maintenance therapy (the ongoing use of a drug) with lenalidomide and/or bortezomib increases how long patients survive and extends how long they live without active myeloma. Maintenance therapy has to be used with some caution, although recent studies have shown significant improvement with survival using this approach. Find out more about the basics of maintenance therapy.

In addition, many research studies are taking place now to find out more about specific molecular targets and new treatments directed at them. It is also important to talk with your doctor about possible side effects for a specific medication and how they can be better managed. Learn more about the basics of targeted treatments.

Chemotherapy

Chemotherapy is the use of drugs to destroy cancer cells, usually by stopping the cancer cells’ ability to grow and divide. Chemotherapy is given by a medical oncologist, a doctor who specializes in treating cancer with medication, or a hematologist, a doctor who specializes in treating blood disorders.

Systemic chemotherapy gets into the bloodstream to reach cancer cells throughout the body. Systemic chemotherapy is not targeted, in the sense that these drugs typically affect the cancer cells as a whole and their ability to divide. Common ways to give chemotherapy include an intravenous (IV) tube placed into a vein using a needle or in a pill or capsule that is swallowed (orally).

A chemotherapy regimen (schedule) usually consists of a specific number of cycles given over a set period of time. A patient may receive 1 drug at a time or combinations of different drugs at the same time.

Chemotherapy drugs that have been used successfully for the treatment of myeloma include cyclophosphamide (Cytoxan, Neosar), doxorubicin (Adriamycin, Doxil), melphalan (Alkeran), etoposide (Toposar, VePesid), cisplatin (Platinol), and carmustine (BiCNU).

Often, more than 1 drug is used at a time. It may also be recommended to combine chemotherapy with other types of treatment, including targeted therapies (see above) or steroids (see below). For instance, the combination of melphalan, the steroid prednisone, and a novel targeted therapy called bortezomib is approved by the U.S. Food and Drug Administration (FDA) for the initial treatment of multiple myeloma. This is because this combination increases survival when compared with melphalan and prednisone. A patient may also be given a combination of melphalan, prednisone, and thalidomide. Additional combinations of drugs are being evaluated in clinical trials.

The side effects of chemotherapy depend on the individual and the dose used, but they can include fatigue, risk of infection, nausea and vomiting, hair loss, loss of appetite, and diarrhea or constipation. Other side effects include peripheral neuropathy (tingling or numbness in feet or hands), blood clotting problems, and low blood counts. These side effects usually go away once treatment is finished. Occasionally an allergic reaction such as skin rash or urticaria may occur and the drug may have to be stopped.

The length of chemotherapy treatment varies from patient to patient and is usually given until the myeloma is well controlled.

Learn more about the basics of chemotherapy and preparing for treatment. The medications used to treat cancer are continually being evaluated. Talking with your doctor is often the best way to learn about the medications prescribed for you, their purpose, and their potential side effects or interactions with other medications. Learn more about your prescriptions by using searchable drug databases.

Other drug therapy

Steroids, such as prednisone and dexamethasone (multiple brand names), may be given alone or at the same time as other drug therapy, such as with targeted novel therapy or chemotherapy (see above).

For example, lenalidomide (Revlimid) and dexamethasone as induction and maintenance therapy is recommended for those who are not able to have stem cell transplantation. Adding bortezomib (Velcade) to this combination has recently been shown to be effective in a clinical trial. In addition, most patients with myeloma receive monthly infusions of bisphosphonate therapy, which are drugs that help to prevent the progression of bone disease from myeloma. Learn more about the basics of bisphosphonate therapy.

Stem cell transplantation/bone marrow transplantation

A stem cell transplant is a medical procedure in which bone marrow that contains cancer is replaced by highly specialized cells, called hematopoietic stem cells, that develop into healthy red blood cells, white blood cells, and platelets in the bone marrow. Hematopoietic stem cells are blood-forming cells found both in the bloodstream and in the bone marrow. Today, this procedure is more commonly called a stem cell transplant, rather than bone marrow transplant, because it is the stem cells in the blood that are typically being transplanted, not the actual bone marrow tissue.

Before recommending transplantation, doctors will talk with the patient about the risks of this treatment and consider several other factors, such as the type of cancer, results of any previous treatment, and the patient’s age and general health.

There are 2 types of stem cell transplantation depending on the source of the replacement blood stem cells: allogeneic (ALLO) and autologous (AUTO). ALLO uses donated stem cells, while AUTO uses the patient’s own stem cells. For multiple myeloma, AUTO is more commonly used. ALLO is being studied in clinical trials. In both types, the goal is to destroy all of the cancer cells in the marrow, blood, and other parts of the body using high doses of chemotherapy (usually melphalan) and then allow replacement blood stem cells to create healthy bone marrow.

Learn more about the basics of stem cell and bone marrow transplantation.

Radiation therapy

Radiation therapy is the use of high-energy x-rays or other particles to destroy cancer cells. A doctor who specializes in giving radiation therapy to treat cancer is called a radiation oncologist. The most common type of radiation treatment is called external-beam radiation therapy, which is radiation given from a machine outside the body. A radiation therapy regimen (schedule) usually consists of a specific number of treatments given over a set period of time.

Doctors may recommend radiation therapy for patients with bone pain when chemotherapy is not effective or in order to control pain. However, using radiation therapy is not an easy decision. In many instances, pain (especially back pain) is due to structural damage to the bone. Radiation therapy will not help this type of pain and may affect the bone marrow's response to future treatment.

Side effects of radiation therapy may include fatigue, mild skin reactions, upset stomach, and loose bowel movements. Most side effects go away soon after treatment is finished. Learn more about the basics of radiation therapy.

Surgery

Surgery is not usually a disease-directed treatment option for multiple myeloma, but it may be used to relieve symptoms (see below). Surgery is used to treat bone disease, especially if there are fractures, and recent plasmacytomas, especially if they occur outside the bone.

Getting care for symptoms and side effects

Cancer and its treatment often cause side effects. In addition to treatment to slow, stop, or eliminate the cancer, an important part of cancer care is relieving a person’s symptoms and side effects. This approach is called palliative or supportive care, and it includes supporting the patient with his or her physical, emotional, and social needs.

Palliative care is any treatment that focuses on reducing symptoms, improving quality of life, and supporting patients and their families. Any person, regardless of age or type and stage of cancer, may receive palliative care. It works best when palliative care is started as early as needed in the cancer treatment process. People often receive treatment for the cancer and treatment to ease side effects at the same time. In fact, patients who receive both often have less severe symptoms, better quality of life, and report they are more satisfied with treatment.

Palliative treatments vary widely and often include medication, nutritional changes, relaxation techniques, emotional support, and other therapies. For people with myeloma, supportive care to reduce symptoms and complications may include:

  • Antibiotics and intravenous immunoglobulins (a blood product given through an IV and known as IVIG) to treat or prevent infections.

  • Pain medications, or analgesics, are given to patients who need help managing bone pain. They can include a wide range of drugs, from acetaminophen (Tylenol) to opiates. Non-steroidal anti-inflammatory drugs (NSAIDs) should be avoided.

  • Antiviral medications may treat or prevent viral infections such as herpes zoster, particularly for those patients who are being treated with a proteasome inhibitor.

  • A blood thinner will help prevent blood clots for patients who are being treated with a combination of an immunomodulatory agent, such as thalidomide (Thalomid), lenalidomide (Revlimid), or pomalidomide (Pomalyst), and dexamethasone, which is a steroid.

  • Antidepressants are frequently prescribed not only to help patients cope with the normal reaction to a cancer diagnosis, but also sometimes to treat painful neuropathy that can be caused by the myeloma itself or by treatments for myeloma.

  • Antacids or proton pump inhibitors are frequently given to patients who are taking steroids to prevent or treat gastrointestinal problems.

  • Plasmapheresis is a procedure used to reduce the thickness of the blood and relieve the related symptoms. During this procedure, blood is taken from a vein. The plasma is then removed from the body after it is separated from the red and white blood cells. Plasma is the liquid part of the blood. The blood cells are then mixed with a plasma substitute, usually an albumin solution, and placed back into the patient. This procedure is often done using a blood cell separator.

  • Bisphosphonates are drugs that increase bone density. They are given to help with bone pain and reduce the risk of fractures. These drugs also prevent high levels of calcium in the blood, which reduces the effects of having too much calcium circulating in the blood.

  • Exercise is recommended to maintain bone strength, reduce the loss of calcium, and help prevent fatigue.

  • Drinking an adequate amount of water is very important. Patients are encouraged to drink water and other healthy fluids, so the body can flush the kidneys and help them filter impurities from the blood.

  • A balanced diet that is high in calories and protein helps prevent fatigue and infection, as does getting plenty of rest and reducing stress.

  • Emollient/moisturizing cream and supplements may also help with specific side effects such as peripheral neuropathy.

You may also receive palliative treatments similar to those meant to eliminate the cancer, such as chemotherapy, surgery, or radiation therapy. For example, surgery may be used to relieve pressure from a plasmacytoma on the spine or other organs. More recently, procedures have been investigated to relieve pain, restore lost height due to a collapsing vertebra, and strengthen the spine. One such procedure is kyphoplasty, which inflates a balloon between vertebrae to clear the space, then removing the balloon and injecting bone cement to stabilize the spine. Another procedure is vertebroplasty, which involves injecting bone cement to stabilize fractures in the spine. Talk with your doctor about the goals of each treatment in your treatment plan.

Before treatment begins, talk with your health care team about the possible side effects of your specific treatment plan and palliative care options. And during and after treatment, be sure to tell your doctor or another health care team member if you are experiencing a problem so it can be addressed as quickly as possible. Learn more about palliative care

Refractory myeloma

The disease is called relapsed and refractory myeloma if the cancer no longer responds to the most recent treatment. If this happens, it is a good idea to talk with doctors who have experience in treating refractory myeloma. Doctors can have different opinions about the best standard treatment plan. Also, clinical trials might be an important option.

Sometimes drugs that have gone through advanced phases of clinical trial and are awaiting FDA approval are made more readily available to patients with refractory myeloma through FDA’s Expanded Access Clinical Trial Program. One such drug that has gone through this process is daratumumab (Darzalex). It was first approved in 2015 to be used alone to treat relapsed and refractory myeloma after other treatments had failed. Most recently, in November 2016, the FDA approved the use of daratumumab in combination with lenalidomide and dexamethasone or with bortezomib and dexamethasone to treat people who have already received at least 1 previous treatment. You may want to talk to your physician about availability of such drug(s). 

Learn more about getting a second opinion before starting treatment, so you are comfortable with your chosen treatment plan.

For people with refractory myeloma, palliative care may also be important to help relieve symptoms and side effects.

For most patients, a diagnosis of relapsed and refractory myeloma is very stressful and, at times, difficult to bear. Patients and their families are encouraged to talk about the way they are feeling with doctors, nurses, social workers, or other members of the health care team. It may also be helpful to talk with other patients, including through a support group.

Remission and the chance of recurrence

A remission is when cancer cannot be detected in the body and there are no symptoms. A remission may be temporary or permanent. This uncertainty causes many people to worry that the cancer will come back. It’s important to talk with your doctor about the possibility of the cancer returning, which is almost certain with myeloma, despite recent advances in therapy. Understanding your risk of recurrence and the treatment options may help you feel more prepared if the cancer does return. Learn more about coping with the fear of recurrence.

If the cancer does return after the original treatment, it is called recurrent myeloma or relapsed myeloma. When this occurs, a cycle of testing will begin again to learn as much as possible about the recurrence. After testing is done, you and your doctor will talk about your treatment options. Often the treatment plan will include the treatments described above such as targeted therapy and chemotherapy, but they may be used in a different combination or given at a different pace. Recent advances in targeted, novel therapy mean that the chances of effective treatment for relapsed disease are increasing.

Your doctor may also suggest clinical trials that are studying new ways to treat recurrent or relapsed myeloma. There are multiple drugs currently being researched in the late stages of clinical trials that have shown promise as treatments for recurrent or relapsed myeloma. See the Latest Research section for more information. Whichever treatment plan you choose, palliative care will also be important for relieving symptoms and side effects.

People with relapsed myeloma often experience emotions such as disbelief or fear. Patients are encouraged to talk with their health care team about these feelings and ask about support services to help them cope. Learn more about dealing with cancer recurrence.

If treatment fails

Recovery from and a cure for myeloma are unlikely. The disease is still considered incurable despite recent progress. If the cancer cannot be controlled even with the newer treatments available, the disease may be called advanced or terminal.

This diagnosis is stressful, and advanced cancer is difficult to discuss for many people. However, it is important to have open and honest conversations with your doctor and health care team to express your feelings, preferences, and concerns. The health care team is there to help, and many team members have special skills, experience, and knowledge to support patients and their families. Making sure a person is physically comfortable and free from pain is extremely important.

Patients who have advanced cancer and who are expected to live less than 6 months may want to consider a type of palliative care called hospice care. Hospice care is designed to provide the best possible quality of life for people who are near the end of life. You and your family are encouraged to think about where you would be most comfortable: at home, in the hospital, or in a hospice environment. Nursing care and special equipment can make staying at home a workable alternative for many families. Learn more about advanced cancer care planning.

After the death of a loved one, many people need support to help them cope with the loss. Learn more about grief and loss.

The next section in this guide is About Clinical Trials. It offers more information about research studies that are focused on finding better ways to care for people with cancer. Or, use the menu to choose another section to continue reading this guide.