Understanding Pomalyst (pomalidomide) capsules

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March 2024 Edition A publication of the International Myeloma Foundation Multiple Myeloma | Cancer of the Bone Marrow Understanding POMALYST ® (pomalidomide) capsules

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 Pomalyst works 5 Indication for treatment with Pomalyst 5 Dose and schedule of Pomalyst 6 How Pomalyst is taken 6 Warnings and precautions 6 Potential side effects of Pomalyst 10 In closing 12 Terms and definitions 13

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 Pomalyst® (also known as pomalidomide, its generic drug name). Pomalyst is an immunomodulatory agent taken by mouth in capsule form. It is the third immunomodulatory agent to receive approval from the U.S. Food and Drug Administration (FDA) for use in myeloma: thalidomide was approved in 2003, Revlimid® (lenalidomide) was approved in 2006, and Pomalyst was approved in 2013.

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 Pomalyst works

An immunomodulatory agent is a drug that can modify, enhance, or suppress the functioning of the immune system. Immunomodulatory agents have multiple actions. They have both anti-cancer and anti- inflammatory effects. Immunomodulatory agents are vascular endothelial growth factor (VEGF) inhibitors that interfere with angiogenesis, the formation of blood vessels that usually accompanies the growth of tumor cells.

Immunomodulatory agents also reduce the levels of cytokines and interleukins, which can promote the growth of cancer cells. Research shows that interleukin-6 (IL-6) stimulates and supports the growth of myeloma cells, and that Pomalyst “down-regulates” (reduces) the amount of IL-6. Down-regulation is the process by which a cell decreases the quantity of a cellular component.

In addition, immunomodulatory agents enhance the activation of specialized white blood cells (WBC), both the T cells (T lymphocytes) and “natural killer ” (NK) cells.

Indication for treatment with Pomalyst

Pomalyst is indicated for the treatment of adult patients with relapsed and refractory myeloma who have received at least 2 prior therapies, including the immunomodulatory agent Revlimid and a proteasome inhibitor, and have demonstrated disease progression on or within 60 days of completion of the last therapy.

Pomalyst is used in many regimens as therapy for previously treated myeloma, including the following combination therapies:

¡ Pomalyst + the steroid dexamethasone [Pd].

¡ Darzalex® (daratumumab) intravenous (IV) infusion + Pomalyst + dexamethasone [DPd].

¡ Darzalex Faspro® (daratumumab + hyaluronidase-fihj) subcutaneous (SQ) injection + Pomalyst + dexamethasone [DPd].

¡ Empliciti® (elotuzumab) + Pomalyst + dexamethasone [EPd].

¡ Ninlaro® (ixazomib) + Pomalyst + dexamethasone [IPd].

¡ Pomalyst + cyclophosphamide + dexamethasone [PCd].

¡ Pomalyst + Velcade® (bortezomib) + dexamethasone [PVd].

¡ Kyprolis® (carfilzomib) + Pomalyst + dexamethasone [KPd].

¡ Sarclisa® (isatuximab-irfc) + Pomalyst + dexamethasone [Isa-Pd].

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Dose and schedule of Pomalyst

Pomalyst capsules are given orally at 4 mg per day on days 1 through 21 of repeated 28-day cycles until disease progression. Pomalyst was approved in combination with dexamethasone. The recommended dose of dexamethasone is 40 mg per day on days 1, 8, 15, and 22 of each 28-day cycle.

If you are experiencing an unwanted or unexpected side effect or reaction, your doctor will determine if it is appropriate to adjust your dose of Pomalyst and/or dexamethasone. You must promptly report any and all new problems to your doctor.

How Pomalyst is taken

¡ Pomalyst is taken in capsule form by mouth.

¡ On the days that Pomalyst is taken, it should be taken at the same time of the day.

¡ Swallow Pomalyst with water. Pomalyst may be taken with or without food.

¡ Do not break, chew, or open the capsules.

¡ Do not handle the capsules more than needed.

¡ If you touch a broken Pomalyst capsule, wash the area of your body that came in contact with the medicine immediately and thoroughly with soap and water.

¡ If you miss a dose of Pomalyst, and it has been less than 12 hours since your regular time to take it, you should take it as soon as possible. If it has been more than 12 hours since your regular time, just skip your missed dose. Do NOT take two doses at the same time.

¡ If you take either too much or too little Pomalyst instead of your prescribed dose, promptly alert your doctor or poison control center.

¡ Store Pomalyst at room temperature.

¡ Keep Pomalyst and all medications out of the reach of children.

Warnings and precautions

Most of the side effects associated with Pomalyst are manageable and predictable. However, there are certain potential side effects that are serious enough to require a “Boxed Warning,” the highest safety-related warning that a medication can have assigned by the FDA. A Boxed Warning is intended to bring the patient’s attention to the risks of a drug.

Before starting treatment with Pomalyst, discuss the warnings, precautions, and side effects with the doctor treating your myeloma. After starting

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treatment with Pomalyst, promptly report to your doctor any problems or changes in your health so that appropriate steps can be taken. No problem is too small to report.

Pomalyst REMS

Pomalyst is available only through a restricted program called “Risk Evaluation and Mitigation Strategy (REMS).” The FDA requires a REMS program if a specific drug or treatment has serious safety concerns. REMS programs support the use of such drugs or treatments and help ensure that the potential benefits outweigh the risks. Prescribers and pharmacists must be certified with the REMS program, and patients must sign an agreement and comply with its requirements.

Females of reproductive potential

Pomalyst is contraindicated in pregnancy. Pomalyst is a chemical analog of thalidomide, which is known to cause severe birth defects or embryo-fetal death. During laboratory studies, it was discovered that Pomalyst harms the developing fetuses of laboratory animals.

Females of reproductive potential must have pregnancy tests weekly for 4 weeks, then every 4 weeks (if your menstrual cycle is regular) or every 2 weeks (if your menstrual cycle is irregular).

Patients must agree to use 2 acceptable forms of effective birth control at the same time for at least 4 weeks before starting Pomalyst, while taking Pomalyst or during any interruptions in treatment, and for at least 4 weeks after stopping treatment with Pomalyst.

If you become pregnant while taking Pomalyst, stop taking it and contact your doctor immediately. If your doctor is not available, contact FDA MedWatch at 1.800.FDA.1088.

Male patients

Pomalyst is present in the semen of male patients who take it, so they must also comply with mandatory contraceptive measures. If your female partner becomes pregnant, alert your doctor right away. In addition, do not donate blood because if a pregnant female gets your donated blood, her baby may be born with birth defects.

Nursing mothers

Because many drugs are excreted in human milk, a decision should be made whether to discontinue nursing or to discontinue the drug, taking into account the importance of the drug to the mother.

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Blood clots

Deep vein thrombosis (DVT) and pulmonary embolism (PE), as well as myocardial infarction (heart attack) and stroke can occur in myeloma patients treated with Pomalyst. In the clinical trial data submitted to the FDA for approval of Pomalyst, all study patients were required to receive antithrombotic medication to reduce the risk of blood clots forming and growing. Your doctor will assess your individual risk factors to determine which medication is most appropriate for you.

Before starting Pomalyst, tell your doctor about all the medicines you take, and if you have had a blood clot in the past, if you smoke, have high blood pressure, or have hyperlipidemia (high level of fat in your blood).

Get emergency medical help if you experience the following:

¡ Swelling of your lips, mouth, tongue, or throat,

¡ Trouble breathing or swallowing,

¡ Skin hives (raised red areas on your skin),

¡ A very fast heartbeat,

¡ Dizziness or fainting.

Get medical help right away if you experience the following:

¡ Arm or leg swelling,

¡ Shortness of breath,

¡ Feeling sweaty,

¡ Severe itching,

¡ Vomiting,

¡ Fever,

¡ Severe headache or confusion,

¡ Problems with vision, speech, or balance,

¡ A red skin rash or peeling of your skin or blisters,

¡ Chest pain (that may or may not spread to the arms, neck, jaw, back, or abdomen),

¡ Sudden numbness or weakness (especially if on one side of the body).

Neutropenia

Neutropenia is a reduced level of neutrophils, a type of white blood cell necessary to combat bacterial infection. Having too few neutrophils can lead to infection. Fever is the most common sign of neutropenia and indicates that you need immediate medical attention.

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Neutropenia of any Grade was reported in 50% of clinical trial patients and was the most frequently reported serious side effect. Other common symptoms associated with a low neutrophil count include sore throat and mouth sores.

When patients with relapsed myeloma begin therapy with Pomalyst, neutropenia is often a problem that results from the combined effects of myeloma infiltration of the bone marrow, the impact of prior therapy, and the impact of the Pomalyst.

During the first 2–3 months of therapy, it is especially important to try to maintain the full dose of Pomalyst. As your neutrophils recover, your tolerance for the full dose of Pomalyst should improve. Your doctor may prescribe a colony-stimulating factor (CSF) to stimulate the development and growth of your blood cells.

For the first 8 weeks of treatment, patients should be monitored with a weekly complete blood count (CBC), and with a monthly CBC thereafter. Treatment can be modified if blood counts are too low. Tell your doctor if you have any cold- or flu-like symptoms, because neutropenia accompanying a viral infection may resolve quickly after the infection has cleared.

With ongoing Pomalyst therapy beyond the initial cycles, support for white blood cell production may be an ongoing concern. Mild neutropenia generally has no symptoms and may not need treatment.

Renal and hepatic impairment

Pomalyst is metabolized in the liver. Pomalyst and its metabolites are excreted by the kidneys. The influence of renal (kidney) and hepatic (liver) impairment on the safety, efficacy, and pharmacokinetics (PK) of Pomalyst has not been evaluated. Pomalyst should not be taken by the following patients:

¡ Patients whose serum creatinine is > 3.0 mg/dL.

¡ Patients whose serum bilirubin is > 2.0 mg/dL.

¡ Patients whose AST/ALT liver enzymes ratio in the blood is > 3.0 x ULN (upper limit of normal).

Hypersensitivity reactions

Patients with a prior history of serious sensitivity to thalidomide or Revlimid were excluded from studies with Pomalyst and may be at higher risk of allergic reactions to Pomalyst. Drug hypersensitivity results from the interaction of a drug and the immune system. Risk factors for drug hypersensitivity reactions include age, female gender, concurrent illnesses, and previous hypersensitivity to related drugs. Symptoms may include difficulty breathing, rash, hives, fever, swelling, vomiting, or diarrhea.

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Treatment is largely supportive and includes discontinuation of the medication and treatment of the symptoms. Contact your healthcare provider immediately if you experience any symptoms after taking Pomalyst.

Drug interactions

No formal drug interaction studies have been conducted with Pomalyst, but patients should not take Pomalyst with drugs that are strong inhibitors of CYP1A2, CYP3A, or P-gp. Cigarette smoking may reduce the efficacy of Pomalyst due to CYP1A2 activation. Any concerns or questions about possible drug interactions or smoking should be discussed with your doctor.

Age

Safety and effectiveness of Pomalyst in patients under the age of 18 have not been established. No dosage adjustment is required for Pomalyst based on age. However, clinical trial patients aged 65 years or older were more likely to experience pneumonia than patients younger than age 65.

Second primary malignancy

Cases of acute myelogenous leukemia (AML) have been reported in patients receiving Pomalyst in clinical trials, although none of these were patients in myeloma trials.

Potential side effects of Pomalyst

In a clinical trial of 219 patients who received Pomalyst alone or the Pd combination therapy, all patients experienced at least one side effect. The most common side effects occurring in 30% or more of patients included asthenia, neutropenia, anemia, constipation, nausea, diarrhea, difficulty breathing, upper respiratory tract infection, back pain, and fever.

The most common serious side effects were low blood counts and pneumonia. Your doctor may decide to interrupt your treatment with Pomalyst until your side effect resolves, then restart it at a lower dose.

Dizziness and confusion

In the clinical trial that led to FDA approval of Pomalyst, 18% of patients experienced dizziness and 12% of patients experienced some mental confusion.

Prevention of dizziness and confusion

Patients should be aware that dizziness and confusion are potential side effects and should avoid situations where this would pose a risk to, or problem for, themselves or others. You must discuss all your medications with your doctor because dizziness and confusion may be caused by other medications you are taking.

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Neuropathy

Symptoms of neuropathy that affect the sensory and motor nerves include numbness, tingling, and pain in the hands or feet, which 18% of patients experienced in the clinical trial of Pomalyst. Symptoms of neuropathy that affect the autonomic nervous system include dizziness and fainting. There were no cases of serious neuropathic side effects.

Prevention and treatment of neuropathy

We strongly urge you to contact your doctor if you experience new or worsening symptoms of this condition. Many myeloma patients have underlying neuropathy from their disease before they are diagnosed and treated for myeloma. Early detection and dose modification can prevent progression of neuropathy. For more information, read the IMF’s publication Understanding Peripheral Neuropathy in Myeloma.

Fatigue

Fatigue caused by cancer or cancer treatment is a distressing, persistent, subjective sense of tiredness or exhaustion that is not proportional to recent activity and interferes with usual functioning. Although many of the patients in clinical trials with Pomalyst felt fatigued, very few had severe fatigue that prevented them from performing the activities of daily living. As a general rule, any patient suffering from fatigue should exercise caution if operating machinery, including automobiles.

Prevention and treatment of fatigue

If your fatigue is severe, your doctor may have to intervene with appropriate supportive care measures. There are medications to treat this problem. The effects of fatigue may be minimized by maintaining:

¡ A moderate level of activity (because inactivity breeds greater fatigue).

¡ A healthy diet and proper fluid intake.

¡ A consistent sleeping schedule with enough rest.

¡ Regularly scheduled visits with your healthcare providers to discuss your fatigue issues.

For more information, read the IMF’s publication Understanding Fatigue.

Asthenia

Asthenia is a condition in which the body lacks or has lost strength either as a whole or in any of its parts. It is a common complication of a number of drugs that are used to treat myeloma, and of cancer treatments in general.

Prevention and treatment of asthenia

First and easiest, try taking your Pomalyst at night before you go to bed in order to limit the side effects to the hours when you’re asleep. If you

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are still experiencing asthenia during your waking hours, your doctor may reduce your dose of Pomalyst or may prescribe medication to be taken in the mornings that may help you be more active during the day. Finally, asthenia may improve by itself with prolonged therapy or may improve as your myeloma disease burden is decreased.

Thrombocytopenia

Thrombocytopenia is a low number of platelets in the blood. Platelets help blood to clot; fewer platelets can lead to easier bruising, bleeding, and slower healing. The “normal” level of platelets varies from laboratory to laboratory. For example, at Mayo Clinic the “normal” level is 150,000 or more platelets per microliter of circulating blood. If the platelet count is less than 50,000, bleeding problems could occur. Major bleeding is usually associated with a reduction to less than 10,000.

Prevention and treatment of thrombocytopenia

You should inform your doctor if you experience excessive bruising or bleeding. Management may include platelet transfusions at the discretion of your doctor.

Anemia

Red blood cells contain hemoglobin, a protein that carries oxygen to the body’s tissues and organs. Anemia is usually defined as a decrease in hemoglobin < 10 g/dL or as a decrease of ≥ 2 g/dL from the normal level for an individual. More than 13–14 g/dL is considered normal. Low levels of oxygen in the body may cause shortness of breath and feelings of exhaustion. Many newly diagnosed myeloma patients have anemia.

Prevention and treatment of anemia

In addition to lowering the dose of Pomalyst, if this is deemed appropriate, your doctor may determine that a treatment regimen for anemia may be best suited and safest for you.

The following are options for treatment of anemia:

¡ Adjusting medications.

¡ Blood transfusions.

¡ Erythropoietic (red blood cell-making) agents.

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

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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.

Autonomic nervous system: The part of the nervous system that regulates functions of organs over which we have no conscious control. The autonomic nerves connect the spinal cord to the internal organs, including the blood vessels, stomach, intestines, lungs, liver, kidneys, bladder, and heart.

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.

Complete blood count (CBC): Many cases of MGUS, SMM, and myeloma are identified as the result of this routine blood test, which quantifies all the cells that make up the solid parts of blood. The CBC is usually performed as part of an annual medical exam, and it is also one of the tests needed for diagnosing and monitoring patients with myeloma.

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.

Deep vein thrombosis (DVT): A condition that occurs when a blood clot (thrombus) forms in one or more of the deep veins in the body, usually in the legs. A blood clot from a DVT can break loose (embolize) and travel to the heart or lungs. An embolus is potentially life-threatening. DVT can occur without any symptoms, or it can cause leg pain or swelling.

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.

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Grade: The toxicity criteria adopted in the United States by the National Cancer Institute (NCI) for cancer clinical trials includes:

• Grade 0 – no symptoms,

• Grade 1 – mild symptoms,

• Grade 2 – moderate symptoms,

• Grade 3 – symptoms requiring treatment,

• Grade 4 – symptoms requiring urgent intervention,

• Grade 5 – symptoms resulting in death.

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.

Inflammation: A protective response of the body against injury or disease.

Interleukin: A naturally produced chemical released by the body, or a substance used in biological therapy. Interleukins stimulate the growth and activities of certain kinds of white blood cells. Interleukin-2 (IL-2) is a type of biological response modifier that stimulates the growth of certain blood cells in the immune system that can fight some types of cancer.

Interleukin-6 (IL-6) is a cytokine that is a potent stimulus to osteoclast and plasma cell growth.

Intravenous (IV) infusion: Administered into a vein.

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.

Natural killer (NK) cell: NK cells, also known as large granular lymphocytes, are a type of white blood cell. They are responsible for tumor surveillance; able to recognize cells that have been transformed by tumors and to induce a strong response against tumors through the release of cytokines. Unlike cytotoxic T cells, NK cells can do this without the need of a “trigger” antigen

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on the tumor. This can result in a faster defensive response. In patients with active myeloma, NK cells are reduced both in number and in function.

Pharmacokinetics (PK): The study of the processes by which a drug is absorbed, distributed, metabolized, and eliminated by the body.

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.

Proteasome: A joined group (“complex”) of enzymes (“proteases”) that break down the damaged or unwanted proteins in both normal cells and cancer cells into smaller components. Proteasomes also carry out the regulated breakdown of undamaged proteins in the cell, a process that is necessary for the control of many critical cellular functions. These smaller protein components are then used to create new proteins required by the cell. This is important for maintaining balance within the cell and for regulating cell growth.

Proteasome inhibitor: Any drug that interferes with the normal function of the proteasome. See “ Proteasome.”

Pulmonary embolism (PE): A potentially life-threatening condition that occurs when a blood clot in a vein (deep vein thrombosis, or DVT) breaks loose, travels through the bloodstream, and gets stuck in an artery in a lung, blocking blood flow.

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.

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• 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).

Stable disease: Response to treatment that does not meet criteria for CR, VGPR, PR, or progressive disease. Myeloma can remain stable for many years. See “ Response” and “ Progressive disease.”

Steroid: A type of hormone. Steroidal hormones are produced by the body. Synthetic analogues (equivalents) of some steroids can be manufactured in a laboratory. Dexamethasone, prednisone, and methylprednisolone are synthetic steroids that have multiple effects and are used for many conditions, including myeloma.

Subcutaneous (SQ) injection: A method of administering medication under the skin by a short needle that injects a drug into the tissue layer between skin and muscle.

T cell (T lymphocyte): A type of white blood cell that plays a central role in the immune system. T cells can be distinguished from other lymphocytes, such as B cells and natural killer (NK) cells, by the presence of a T-cell receptor (TCR) on the cell surface. They are called T cells because they mature in the thymus, although some also mature in the tonsils.

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 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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17 myeloma.org Notes
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INTERACTIVE RESOURCES AT A GLANCE

Use the hyperlinks and web addresses included in this publication for quick access to resources from the IMF.

infoline.myeloma.org

Contact the IMF InfoLine with your myeloma-related questions and concerns

medications.myeloma.org

Learn about FDA-approved therapies for myeloma

diversity.myeloma.org

Diversity and inclusion are integral aspects of the myeloma community

videos.myeloma.org

The latest on myeloma research and clinical practice, as well as IMF webinars and other events

support.myeloma.org

Robin Tuohy

rtuohy@myeloma.org will help you find a multiple myeloma support group

publications.myeloma.org

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© 2024, International Myeloma Foundation. All rights reserved. 4400 Coldwater Canyon Avenue, Suite 300 Studio City, CA 91604 USA Telephone: 1.800.452.CURE (USA & Canada) 1.818.487.7455 (worldwide) Fax: 1.818.487.7454 TheIMF@myeloma.org myeloma.org u-pom_en_2024_s1-01

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