Understanding Dexamethasone

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March 2024 Edition A publication of the International Myeloma Foundation Multiple Myeloma | Cancer of the Bone Marrow Understanding Dexamethasone in the Treatment of Myeloma

Founded in 1990, the International Myeloma Foundation (IMF) is the first and largest organization focusing specifically on myeloma. The IMF’s reach extends to more than 525,000 members in 140 countries. The IMF is dedicated to improving the quality of life of myeloma patients while working toward prevention and a cure through our four founding principles: Research, Education, Support, and Advocacy.

RESEARCH The IMF is dedicated to finding a cure for myeloma, and we have a range of initiatives to make this happen. The International Myeloma Working Group, which emerged from the IMF’s Scientific Advisory Board established in 1995, is the most prestigious organization with more than 300 myeloma researchers conducting collaborative research to improve outcomes for patients while providing critically appraised consensus guidelines that are followed around the world. Our Black Swan Research Initiative® is bridging the gap from long-term remission to cure. Our annual Brian D. Novis Research Grant Program is supporting the most promising projects by junior and senior investigators. Our Nurse Leadership Board, comprised of nurses from leading myeloma treatment centers, develops recommendations for the nursing care of myeloma patients.

EDUCATION The IMF’s webinars, seminars, and workshops provide up-to-date information presented by leading myeloma scientists and clinicians directly to patients and their families. We have a library of more than 100 publications for patients, care partners, and healthcare professionals. IMF publications are always free-of-charge, and available in English and select other languages.

SUPPORT The IMF InfoLine responds to your myeloma-related questions and concerns via phone and email, providing the most accurate information in a caring and compassionate manner. We also sustain a network of myeloma support groups, training hundreds of dedicated patients, care partners, and nurses who volunteer to lead these groups in their communities.

ADVOCACY We empower thousands of individuals who make a positive impact each year on issues critical to the myeloma community. In the U.S., we lead coalitions to represent the interests of the myeloma community at both federal and state levels. Outside the U.S., the IMF’s Global Myeloma Action Network works to help patients gain access to treatment. Learn more about the ways the IMF is helping to improve the

us at 1.818.487.7455 or 1.800.452.CURE, or visit myeloma.org .

quality of life of myeloma patients while working toward prevention and a cure. Call
Contents You are not alone 4 What you will learn from this booklet 4 How dexamethasone works 5 How dexamethasone is given 6 Dosages and scheduling of dexamethasone 6 Dexamethasone in clinical trials 6 Possible side effects of dexamethasone 8 Possible drug interactions 12 Other corticosteroids used to treat myeloma 13 In closing 14 Terms and definitions 14

You are not alone

The International Myeloma Foundation (IMF) is here to help you. The IMF is committed to providing information and support for patients with multiple myeloma (which we refer to simply as “myeloma”) and their care partners, friends, and family members.

We achieve this through a broad range of resources available on our website myeloma.org, and through numerous programs and services such as seminars, webinars, workshops, and the IMF InfoLine, which consistently provides the most up-to-date and accurate information about myeloma in a caring and compassionate manner. Contact the IMF InfoLine at 1.818.487.7455 or InfoLine@myeloma.org.

What you will learn from this booklet

Myeloma is a cancer that is not known to most patients at the time of diagnosis. To play an active role in your own medical care and to make good decisions about your care with your doctor, it is important and helpful to learn about myeloma, as well as its treatment options and supportive care measures.

The IMF’s Understanding-series publications address treatments for myeloma, supportive care measures, and the tests that are used to diagnose, monitor, and assess disease status throughout its course.

This booklet discusses the steroid dexamethasone (also called “dex” for short), one of the most frequently used medications in the treatment of myeloma. Dexamethasone is the generic drug name of this medication, which is also marketed under multiple brand names.

If you are newly diagnosed with myeloma, we suggest that you read the IMF’s publication Patient Handbook for the Newly Diagnosed, which will help you to better understand this complex disease.

To learn about myeloma in later disease settings, read the IMF’s publication Concise Review of Relapsed and Refractory Myeloma.

Words in bold+blue type are explained in the “Terms and definitions” section at the end of this booklet. A more comprehensive glossary can be found in the IMF’s publication Understanding Myeloma Vocabulary located online at glossary.myeloma.org.

If you are reading this booklet in electronic format, the light blue links will take you to the corresponding resources. All IMF publications are free-of-charge and can be downloaded or requested in printed format at publications.myeloma.org.

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How dexamethasone works

A steroid is a type of hormone. Steroidal hormones are produced by the body, and the synthetic analogues (equivalents) of some steroids can be manufactured in a laboratory. Dexamethasone is a synthetic steroid that has multiple effects and is used for many conditions, including myeloma. Dexamethasone is a synthetic adrenocortical steroid. In the body, adrenocortical steroids are produced naturally by the adrenal glands and are also known as glucocorticosteroids or corticosteroids. These compounds will be referred to as “steroids” throughout this booklet.

Adrenal glands produce both hormones and steroids. These steroids influence many actions of the body’s systems. They are involved in regulation of carbohydrates, proteins, and fats. They also inhibit inflammatory, allergic, and normal immune responses. Synthetic versions of steroids can imitate the actions of the naturally occurring compounds, or replace them in conditions that are associated with insufficient production of much-needed steroids that are normally produced by the adrenal glands.

Dexamethasone is available in many forms. To treat myeloma, dexamethasone can be given as either an oral tablet or as an injection, alone or in combination with other agents. Dexamethasone is used to treat a wide variety of medical conditions in addition to myeloma and other hematologic malignancies. Steroids are generally additive or synergistic with other treatments. Steroids as a component of treatment for myeloma may also help improve other conditions, such as the following:

¡ Endocrine disorders,

¡ Rheumatic or collagen disorders,

¡ Dermatologic diseases,

¡ Allergic states,

¡ Ophthalmic (eye) diseases,

¡ Gastrointestinal (GI) diseases,

¡ Respiratory diseases,

¡ Hematologic disorders,

¡ Other malignancies.

Dexamethasone and other steroids have many uses in the treatment of cancer. These steroids suppress certain actions of the immune system and also inhibit cytokines, which control inflammation. Dexamethasone decreases inflammation by stopping white blood cells (WBC), which normally fight infection, from traveling to areas of the body where there is swelling. Dexamethasone’s anti-inflammatory actions can stop the swelling

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around tumors and the resulting pain and other symptoms caused by tumors pressing on nerve endings. Dexamethasone can also alter normal immune system responses and is therefore useful in the treatment of conditions that affect the immune system.

How dexamethasone is given

To treat myeloma, dexamethasone can be given as either an oral tablet or as an injection, alone or in combination with other agents. Dexamethasone can irritate the stomach; taking it with food can reduce the chances of this happening.

Steroid therapy cannot be stopped abruptly. Abrupt discontinuation can lead to withdrawal symptoms. If steroid therapy must be discontinued, it must be done gradually and under the supervision of the doctor treating your myeloma.

Dosages and scheduling of dexamethasone

Many factors are taken into consideration when your myeloma doctor determines your dose of dexamethasone and how it is administered. Ask your doctor about the optimal overall treatment strategy and about finding a dosing regimen that is well tolerated and appropriate for the treatment of your individual disease.

Dexamethasone has demonstrated activity in myeloma as a single agent but it is typically given in combination with one or more other agents, especially during induction therapy. Dexamethasone is a component of nearly all combination therapies, as it appears to increase or “boost” the ability of other agents to destroy myeloma cells, thereby improving response to treatment. However, dexamethasone is associated with many short-term and long-term side effects.

Dexamethasone in clinical trials

A clinical trial is a medical research study with people who volunteer to test scientific approaches to a new treatment or a new combination therapy. Each clinical trial is designed to find better ways to prevent, detect, diagnose, or treat cancer and to answer scientific questions.

Dexamethasone has been part of the vast majority of myeloma clinical trials over a period of many years. There have been numerous clinical trials of different combination therapies with low-dose dexamethasone in patients with newly diagnosed multiple myeloma (NDMM), as well as with relapsed and refractory disease.

Low-dose dexamethasone in combination with other agents has been well established as the standard of care in myeloma. Depending upon the

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age and fitness of the patient, dexamethasone is usually prescribed at a dose of 20 mg to 40 mg once-weekly. For patients who cannot tolerate higher doses, dexamethasone has proven to be effective at doses as low as 4 mg once-weekly.

ECOG E4A03 clinical trial

In 2010, the results of the large ECOG E4A03 clinical trial were published in Lancet Oncology. Prior to this study, the standard of care in myeloma included 40 mg of dexamethasone administered 4 days per week (“highdose”). The E4A03 study is the legacy of Michael Katz, who was diagnosed with myeloma in 1990 and later became a myeloma support group leader and a member of the IMF Board of Directors.

Michael lost his battle with myeloma 25 years after diagnosis, but it was his perseverance and insight that led to the evaluation of the “Rd” frontline therapy of the immunomodulatory agent Revlimid® (lenalidomide) with either high-dose or low-dose dexamethasone. The 1 day per week (“low-dose”) schedule of 40 mg dexamethasone demonstrated better survival at 1 year, with significantly fewer side effects than the 4 days per week schedule.

Rd-R regimen vs. continuous Rd

In 2021, the journal Blood published the results of a clinical trial designed specifically for treatment of older and less fit patients with myeloma, a group usually excluded from clinical trials. Newly diagnosed patients who were 65–80 years old and who were “intermediate-fit” on the International Myeloma Working Group (IMWG) frailty score were randomized to receive 9 months of Rd followed by maintenance therapy of Revlimid (without dexamethasone) at 10 mg per day [Rd-R] or to a study arm that received continuous Rd.

Side effects were mainly related to dexamethasone and were more frequent with continuous Rd. After 9 cycles of Rd, switching to reduced-dose Revlimid maintenance therapy without dexamethasone was feasible, with similar outcomes to standard continuous Rd.

Rd regimen vs. DR regimen

In December 2022, at the annual meeting of the American Society of Hematology (ASH), the efficacy and safety analysis of the IFM2017-03 phase III clinical trial became a point of interest for its limited use of dexamethasone in frail or elderly patients with newly diagnosed myeloma. The Rd regimen was compared to a combination of Darzalex® (daratumumab) + Revlimid [DR], in which patients received only 2 months of dexamethasone. The DR regimen had deeper responses, with overall response rate (ORR) of 96% and complete response (CR) rate of 37%. The Rd regimen had ORR of 85% and CR of 10%.

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Secondary Analysis of SWOG S0777 and S1211

A study presented at the ASH meeting in December 2023 investigated outcomes from two large clinical trials, SWOG S0777 and SWOG S1211, and showed that reducing the dose of dexamethasone did not have a negative impact on patients with NDMM. Dexamethasone was reduced in 76% of the 541 evaluable patients during induction therapy. Progression-free survival (PFS) and overall survival (OS) were comparable between full-dose patients (40 mg once per week or 20 mg on Days 1, 2, 4, 5, 8, 9, 11, and 12 of 21-day cycles), “dex-lowered” patients (end-of-induction dexamethasone dose and/or frequency were lower than at the start), and “very-low-dex” subgroup of patients (≥ 50% reduction in dexamethasone dose during induction). Median OS in the “very-low-dex” subgroup was not reached.

Possible side effects of dexamethasone

Dexamethasone can cause side effects. Few patients get all of the possible side effects described in this section. Some patients do not experience any side effects at all while taking dexamethasone. Ask your doctor how to best prevent, minimize, or treat possible side effects.

You and your doctor can take precautionary measures in order to reduce or avoid side effects. The most important precautions are described in this booklet, and your doctor can provide greater detail about these and other possible side effects, and make recommendations about their management.

The longer you take a steroid, and the higher the dose, the greater your chances of experiencing side effects, but most side effects will go away when treatment is completed. Alert your doctor if you are experiencing side effects or if you notice changes in your health.

Do not stop taking any of your medications or reduce your doses on your own. Discuss your concerns with the doctor who is treating your myeloma.


Dexamethasone is a component of nearly all combination therapies used in myeloma. Any drug that suppresses normal immune responses can make you susceptible to infections, and patients who are taking dexamethasone or other steroids have an increased risk of all types of infections (bacterial, viral, or fungal).

Steroids block white blood cells from reaching sites of infection, and may cause existing infections to get worse or allow new infections to begin. Steroids can mask signs that an infection is present and may also decrease your immune system’s ability to fight the start of a new infection.

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Prevention and treatment of infections

Generally, steroids should not be administered to a patient who has a known infection. Nevertheless, there are some situations in which steroids may be important or necessary during the time that an active infection is being treated appropriately. For example, steroids are useful in the treatment of septic shock, an infection that involves the whole body, and in treating any serious infection that causes a major inflammatory response and/or tissue destruction.

You must tell your doctor as soon as possible if you have been exposed to any infectious illnesses, or if you have any signs or symptoms of an infection. In addition, your doctor must know your entire vaccination history to date. Also, make sure to wash your hands frequently, especially after being in public places.

Cardiac conditions and fluid retention

Use of dexamethasone and other steroids can cause increases in blood pressure, salt and water retention, and potassium and calcium excretion. These changes are more likely to occur when steroids are taken in large doses. Salt retention may lead to edema or swelling. You may notice that your ankles and feet are swollen. Fluid retention and loss of potassium can be a problem for patients who have cardiac conditions, especially congestive heart failure and hypertension.

Prevention and treatment of cardiac conditions and fluid retention

Discuss with your doctor if changes to your diet may be needed, such as restricting your salt intake or replacing the potassium and calcium that you may be losing. Consult with your healthcare team to make sure that you are eating the right foods.

Dermatologic effects

Patients taking dexamethasone or other steroids may notice that it takes longer than usual for wounds to heal. Patients may develop acne and rashes while taking dexamethasone. Increased sweating is seen in some patients during steroid therapy.

Prevention and treatment of dermatologic conditions

Proper hygiene is important. If your dermis (skin) is injured, administer first aid and contact your healthcare team.

Endocrine effects

Steroids, including dexamethasone, may interfere with the way patients metabolize carbohydrates and can cause blood glucose levels to rise. This is especially important in patients who have diabetes. Patients with

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diabetes can take steroids, but additional treatment, including insulin therapy, may be needed to control blood sugar levels. Steroids can also cause menstrual irregularities.

Prevention and treatment of endocrine effects

Patients with diabetes may need to monitor their blood glucose levels more frequently. These patients may need to adjust the doses of their insulin or diabetes medications. This decision needs to be made by healthcare professionals and not by patients themselves. If you have diabetes, tell the doctor who is treating your diabetes that you have been prescribed dexamethasone.

Females of childbearing potential, especially those experiencing menstrual irregularities, should take added precautions not to become pregnant while taking dexamethasone, and should speak with their doctor about the potential effects of steroids on the developing child.

Gastrointestinal (GI) effects

Steroids can have various effects on your GI tract, such as increasing the risk of GI perforations (holes). Therefore, patients who have peptic ulcers, diverticulitis, and ulcerative colitis should use corticosteroids cautiously to minimize the risk of perforation. For these reasons, many physicians automatically recommend antacid therapy of some type for patients taking steroids. Other possible GI side effects seen with dexamethasone therapy are increased or decreased appetite, stomach bloating, nausea, vomiting, hiccups, and heartburn.

Prevention and treatment of gastrointestinal effects

Tell your doctor if you experience any GI side effects while taking dexamethasone and ask for advice on how to manage or avoid these events. To avoid or minimize GI irritation, dexamethasone should be taken with food or after meals. Alcoholic beverages, which may also irritate the stomach, should be avoided while taking dexamethasone. Limiting intake of caffeine-containing foods and drinks (e.g., colas, coffee, tea, and chocolate) may also help. Eating small, frequent meals may decrease nausea. Antacids taken between meals may also be helpful, but should not be taken unless approved by your healthcare team. Treatment for persistent hiccups may require such prescription drugs as baclofen, chlorpromazine, or promethazine.

Musculoskeletal effects

Because steroids decrease calcium absorption and increase its excretion, they affect bones. These effects can lead to pain and osteoporosis in adults. Patients with myeloma who are already subject to severe bone

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loss and bone pain must be watched carefully and given appropriate supportive care to prevent further bone damage. Patients taking steroids may also experience muscle pains because they may be losing potassium.

Prevention and treatment of musculoskeletal effects

Consult with your doctor before taking any supplements or changing your diet. Do not take any supplements or make changes to your diet on your own. Your doctor may recommend supplements or that you increase your intake of calcium and potassium. The best dietary sources of calcium are dairy products, dark-green leafy vegetables, peas and beans, canned fish such as sardines and salmon, and calcium-fortified juices and cereals. Potassium is available in many fruits and vegetables, leafy greens, beans, nuts, dairy foods, and starchy vegetables.

Many patients with myeloma receive bisphosphonate therapy as treatment for myeloma-related bone disease. Bisphosphonate therapy also combats the negative effects of steroids on bone strength and density.

Ophthalmologic effects

Prolonged steroid treatment may produce elevated intraocular pressure that could lead to glaucoma, optic nerve damage, eye infections, and cataracts. Cataracts occur commonly in older age and usually take years to develop to the point where surgery is indicated. Steroids can speed up this process. With ongoing steroid treatment, it is not uncommon for myeloma patients to develop mature cataracts requiring surgery. This involves removal of the cataract and implantation of a new lens in the eye, which usually allows for enhanced vision.

Prevention and treatment of ophthalmologic effects

Have your eyes checked regularly. Any change in vision should be reported immediately to your healthcare team.

Psychiatric and neurologic effects

Steroids can also cause irritability, mood swings, personality changes, insomnia, and severe depression. Emotional instability or psychotic tendencies are aggravated and may become worse during steroid therapy. Patients also have reported experiencing headaches and dizziness.

Prevention and treatment of psychiatric and neurologic effects

If you are having problems sleeping, ask your healthcare team if you can adjust the time you take dexamethasone so it doesn’t interfere with your sleep during the night. Taking steroids before going to bed can be very effective in allowing sleep during the night, with increased activity delayed until morning. However, regular sleep medications can be helpful or necessary for some patients.

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Do not hesitate to contact your doctor if you are experiencing any mood or personality effects. Your doctor may need to reduce or stop your steroid therapy temporarily or permanently. Do not stop steroid therapy on your own without consulting your doctor.

Family members should be advised that you may be more irritable and difficult to live with when you are receiving steroid therapy. Counseling may be a good option at this time, both for the patient and for the care partners. The stresses and pressures of a cancer diagnosis added to life’s other challenges may lead to psychological overload not only for a patient who is receiving steroids, but for the patient’s family members as well. A consultation with a family counselor can be most helpful.

Allergic reactions

Allergic and hypersensitivity reactions to steroids are possible in patients who are susceptible or have had allergic reactions to other drugs. Allergic reactions can include difficulty breathing, closing of the throat, swelling of the lips and tongue, and hives. Such allergic reactions to steroids are exceedingly rare.

Prevention and treatment of allergic reactions

Special precaution should be used before administering dexamethasone or any other corticosteroid to patients who have histories of any type of allergic reactions to medications. Be sure to alert your healthcare team if you have a history of allergic responses when given any medication.

General effects

Some patients may experience coughing or hoarseness. Resting the voice can help with this condition.

Use of steroids, including dexamethasone, can cause weight gain.

Prevention and treatment of weight gain

Some weight gain is to be expected during steroid therapy. Dexamethasone has a tendency to increase patients’ appetites. Patients may need to control their caloric intake. Reduced carbohydrate intake is especially helpful during steroid therapy. Let your healthcare team know immediately if there is a sudden, large weight gain (more than 5 pounds over a day or two).

Possible drug interactions

Interactions are possible with dexamethasone and other medications. Patients with myeloma typically need to take a number of medications to treat the disease as well as other medical conditions that also may be present. Chances of drug interactions increase with multiple medications. Below is a partial list of medications or classes of medications that may interact with dexamethasone. These interactions may increase or decrease

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the actions of any of the drugs. It is very important to tell your healthcare team about all prescription and over-the-counter medications, as well as any herbal preparations or vitamins that you are taking.

Drugs that can interact with dexamethasone and other corticosteroids

¡ Amphotericin B and diuretics that affect potassium levels (such as amiloride, spironolactone, and triamterene).

¡ Antibiotics (such as erythromycin, clarithromycin, rifampicin, and azithromycin).

¡ Anticoagulant medications (such as warfarin and aspirin).

¡ Barbiturates (such as amobarbital, butalbital, pentobarbital, and secobarbital).

¡ Diabetes medications (e.g., insulin, glibenclamide, and metformin).

¡ Cyclosporine.

¡ Digitalis.

¡ Ephedrine, which is most commonly found in weight-loss products.

¡ Estrogen-containing medications, including oral contraceptives and hormone-replacement therapy products.

¡ Nonsteroidal anti-inflammatory drugs (NSAIDs), including aspirin, ibuprofen, indomethacin, and naproxen.

¡ Phenytoin.

Other corticosteroids used to treat myeloma

Ask the doctor treating your myeloma if any steroid other than dexamethasone might be more effective or more appropriate in your care.

In addition to dexamethasone, other corticosteroids are used to treat patients with myeloma. Because these drugs all belong to the glucocorticosteroids class of drugs, they act very similarly and can be used to treat many of the same medical conditions. They behave the same way chemically in the body to treat diseases. Because they are so similar in their mechanisms of action, many of the side effects and associated precautions are the same. Some of the steroids may be better tolerated than others, depending on the patient and the drug.

The uses, side effects, precautions, and considerations described previously for dexamethasone are relevant for the entire class of corticosteroids and thus pertain to prednisone, prednisolone, and methylprednisolone. Prednisolone is a metabolite of prednisone. Methylprednisolone, although structurally similar, may be less toxic and appears to be associated with less sodium and fluid retention than prednisolone.

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In closing

This booklet is not meant to replace the advice of your doctors and nurses who are best able to answer questions about your specific healthcare management plan. The IMF intends only to provide you with information that will guide you in discussions with your healthcare team. To help ensure effective treatment with good quality of life, you must play an active role in your own medical care.

We encourage you to visit myeloma.org for more information about myeloma and to contact the IMF InfoLine with your myeloma-related questions and concerns. The IMF InfoLine consistently provides the most up-to-date and accurate information about myeloma in a caring and compassionate manner. Contact the IMF InfoLine at 1.818.487.7455 or InfoLine@myeloma.org.

Terms and definitions

The following selected terms are used in this booklet, while a more complete glossary can be found in the IMF’s publication Understanding Myeloma Vocabulary located online at glossary.myeloma.org.

Adrenal glands: Glands located at the top of the kidneys that are chiefly responsible for releasing sex hormones and cortisol, a hormone that helps human beings respond to stress.

Bisphosphonate: A type of drug that protects against osteoclast activity (bone breakdown) and binds to the surface of bone where it is being resorbed or destroyed.

Blood glucose: A type of blood sugar that the body produces from the food in our diet. Glucose is transported via the bloodstream to all the cells in our body. It is our primary source of energy. Certain medications can affect our blood glucose levels. There are tests that measure and monitor blood glucose.

Calcium: A mineral found mainly in the hard part of bone matrix (hydroxyapatite). If produced or released in excess, it can build up in the bloodstream. See “ Hypercalcemia.”

Cancer: A term for diseases in which malignant cells divide without control. Cancer cells can invade nearby tissues and spread through the bloodstream and lymphatic system to other parts of the body.

Congestive heart failure: A condition that occurs when the heart’s pumping function is weakened, causing a series of events that result in the body retaining fluid and salt. If fluid builds up in the arms, legs, feet, ankles, lungs, or other organs, the body becomes congested.

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Cytokine: A protein that circulates in the bloodstream, usually in response to infection. Cytokines can stimulate or inhibit the growth or activity in other cells.

Frontline therapy: A general term for the initial treatment used in an effort to achieve response in a newly diagnosed myeloma patient. See “Induction therapy ” and “ Response or remission.”

Generic drug name: A brand name identifies a drug as property of the company that receives approval for it from a governmental regulatory agency, such as the U.S. Food and Drug Administration (FDA). After a drug goes “off patent,” other companies may make generic versions of the drug under a generic name that refers to the chemical makeup of a drug.

Glaucoma: A disease associated with the buildup of pressure inside the eye that, if untreated, can result in vision loss and blindness.

Hematologic malignancy: A cancer of the bone marrow or blood cells.

Hormones: Chemicals produced by various glands that regulate the actions of certain cells or organs in the body.

Hypercalcemia: A higher than normal level of calcium in the blood. In myeloma patients, it usually results from bone breakdown with release of calcium from the bone into the bloodstream. This condition can cause a number of symptoms, including loss of appetite, nausea, thirst, fatigue, muscle weakness, restlessness, and confusion. See “Calcium.”

Hypersensitivity reaction: Undesirable reactions, sometimes in response to a medication, produced by the normal immune system, including allergies and autoimmunity. These reactions may be uncomfortable, damaging, or fatal.

Hypertension: A chronic medical condition in which the blood pressure in the arteries is elevated. Also known as high blood pressure.

Immune system: A complex network of cells, tissues, organs, and the substances they make. The immune system helps the body defend itself by destroying infected and diseased cells and removing cellular debris, while protecting healthy cells.

Immunomodulatory agent: A drug that can modify, enhance, or suppress the functioning of the immune system. An immunomodulatory agent is sometimes called an “immunomodulatory drug (IMiD®).”

Induction therapy: The initial treatment given to a patient in preparation for an autologous stem cell transplant (ASCT). See “ Frontline therapy ” and “ Line of therapy.”

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Inflammatory: Relating to inflammation, a protective response of the body against injury or disease.

Line of therapy: A term used to calculate the number of therapies a patient has received. A line of therapy is 1 or more complete cycles of a regimen that can consist of a single agent, a combination of several drugs, or a planned sequential therapy of various regimens. Also see “ Induction therapy.”

Maintenance therapy: Drug or drugs given to patients to prolong remission.

Median: The mean (middle) of two central numbers in a series of numbers. For example, “median progression-free survival (mPFS)” means that half the patients had remissions that were shorter and half the patients had remissions that were longer than the mPFS.

Metabolism: The conversion of one compound into another compound, which occurs during a living organism’s life-sustaining chemical processes. See ”Metabolite.”

Metabolite: Any substance that is formed during metabolism or that is necessary for metabolism. See ”Metabolism.”

Metabolize: When the body or an organ of the body converts one compound into another by the process of metabolism. See “Metabolism.”

Multiple myeloma: A cancer of the bone marrow plasma cells, white blood cells that make antibodies. Cancerous plasma cells are called myeloma cells.

Osteoporosis: A progressive bone disease that is characterized by a decrease in bone mass and density, leading to an increased risk of fracture. Diffuse involvement of bones with myeloma produces what looks like osteoporosis on X-ray and bone density measurement.

Overall response rate (ORR): In myeloma clinical trials, the percentage of patients whose monoclonal protein decreased by at least 50% in response to treatment.

Overall survival (OS): The median number of individuals in a group who are alive after a particular duration of time. OS is often used as a measure of treatment efficacy in clinical trials. The lengthening duration of OS in myeloma trials makes it a difficult endpoint to use, leading to the effort to validate minimal residual disease (MRD) status as a new endpoint.

Progression-free survival (PFS): The length of time during and after the treatment of myeloma that a patient lives with the disease but the myeloma does not get worse. In a clinical trial, PFS is one way to measure how well the treatment is working. See “ Progressive disease.”

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Progressive disease: Myeloma that is becoming worse or relapsing, as documented by tests. Defined as an increase of ≥ 25% from the lowest confirmed response value in the myeloma protein level and/or new evidence of disease.

Refractory: Disease that is no longer responsive to standard treatments. Myeloma is refractory in patients who have had progressive disease either during treatment or within 60 days following treatment. Most clinical trials for advanced disease are for patients with relapsed and/or refractory myeloma.

Relapse: The reappearance of signs and symptoms of myeloma after a period of improvement. Patients with relapsed disease have been treated, then developed signs and symptoms of myeloma at least 60 days after treatment ended. Most clinical trials for advanced myeloma are for patients with relapsed and/or refractory disease.

Response or remission: Interchangeable terms to describe the complete or partial disappearance of the signs and symptoms of cancer.

• Stringent complete response (sCR) – sCR is CR (as defined below) plus normal FLC ratio and absence of clonal cells in bone marrow by immunohistochemistry or immunofluorescence.

• Complete response (CR) – For myeloma, CR is negative immunofixation on serum (blood) and urine, and disappearance of any soft tissue plasmacytomas, and ≤ 5% plasma cells in bone marrow. CR is not the same as a cure.

• Very good partial response (VGPR) – VGPR is less than CR. VGPR is serum M-protein and urine M-protein detectable by immunofixation but not on electrophoresis, or 90% or greater reduction in serum M-protein, plus urine M-protein less than 100 mg per 24 hours.

• Partial response (PR) – PR is a level of response in which there is at least a 50% reduction in M-protein, and reduction in 24-hour urinary M-protein by at least 90% (or to less than 200 mg per 24 hours).

Side effect: An unwanted or unexpected effect caused by a drug. Also known as adverse reaction or adverse event (AE).

Synergistic: When two or more elements produce a combined effect that is greater than the sum of their separate effects.

Tumor: An abnormal mass of tissue that results from excessive cell division. In myeloma, a tumor is referred to as a plasmacytoma.

White blood cells (WBC): General term for a variety of leukocytes responsible for fighting invading germs, infections, and allergy-causing

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agents. These cells begin their development in bone marrow and then travel to other parts of the body. Specific white blood cells include neutrophils, basophils, eosinophils, lymphocytes, and monocytes.


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