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 .
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 Ninlaro® (also known as ixazomib, its generic drug name). Ninlaro was approved by the U.S. Food and Drug Administration (FDA) in 2015 for the treatment of adult patients with myeloma who have received at least 1 prior therapy. Ninlaro is the third proteasome inhibitor approved by the FDA since 2003, and it is the first proteasome inhibitor that is taken orally (by mouth).
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.
How Ninlaro works
Ninlaro works by blocking the activity of enzyme complexes called proteasomes. Both normal cells and cancer cells contain proteasomes, which break down damaged and unwanted proteins 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.
If the proteasome is stopped (inhibited), then the damaged and unwanted proteins build up in the cell’s nucleus and cytoplasm and cause it to die. Myeloma cells are more sensitive to proteasome inhibition than normal cells, so the myeloma cells die while normal cells are able to recover.
Indications for treatment with Ninlaro
Ninlaro is indicated in combination with the immunomodulatory agent Revlimid® (lenalidomide) plus the steroid dexamethasone, a “triplet” (3-drug) combination known as IRd. Therefore, we recommend that you also read these helpful IMF publications:
¡ Adherence to Oral Cancer Therapy tip card
¡ Understanding Dexamethasone in the Treatment of Myeloma
¡ Understanding REVLIMID® (lenalidomide) capsules
In April 2022, the FDA added a limitation of use for Ninlaro, stating that it is not recommended in the maintenance setting or in newly diagnosed myeloma in combination with Rd outside of controlled clinical trials.
Clinical trial experience with Ninlaro
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.
The FDA approval of Ninlaro was based on data from the TOURMALINE-MM1 clinical trial, a phase III study of IRd vs. placebo + Revlimid + dexamethasone [placebo-Rd] in 722 patients with relapsed or refractory myeloma who had received at least 1 prior line of therapy.
Patients who were refractory to prior treatment with Revlimid or proteasome inhibitors were not eligible to participate. Patients in both the IRd study arm and the placebo-Rd study arm were treated until their myeloma progressed or they were unable to tolerate the treatment.
Study patients were given a blood thinner to prevent blood clots, which is recommended for all myeloma patients taking Rd. Other medications were given as necessary at the discretion of the treating doctor to improve the patients’ tolerance of the treatment.
In June 2021, the Journal of Clinical Oncology published the final analysis of TOURMALINE-MM1 overall survival (OS) by Dr. Paul G. Richardson and colleagues. With a median follow-up of 85 months, median OS was 53.6 months [IRd] vs. 51.6 months [placebo-Rd]. Progression-free survival (PFS) benefit with IRd vs. placebo-Rd did not translate into a statistically significant OS benefit on intent-to-treat analysis.
Ninlaro continues to be studied in clinical trials as part of a wide spectrum of combination therapies and across myeloma disease settings. Contact the IMF InfoLine or visit clinicaltrials.gov for up-to-date information.
IRd dose and schedule
Ninlaro is taken orally. Therefore, it is the patient’s responsibility to take this medication as directed by the treating doctor. It is crucial that you understand the instructions and information your doctor provides to you.
¡ Each treatment cycle is 28 days.
¡ Ninlaro is taken orally on days 1, 8, and 15 of each treatment cycle. Your doctor will determine which starting dose of Ninlaro is best for you. If you have moderate or severe liver or kidney dysfunction, your dose of Ninlaro may be lowered. If you require dialysis, discuss the administration of Ninlaro and the timing of dialysis with your doctor.
¡ Revlimid is taken orally on days 1 through 21 of each treatment cycle. The recommended starting dose of Revlimid is 25 mg. If necessary, your dose of Revlimid can be lowered by your doctor.
¡ Dexamethasone is taken orally on days 1, 8, 15, and 22 of each treatment cycle. The recommended starting dose of dexamethasone is 40 mg. If necessary, your dose of dexamethasone can be lowered by your doctor.
IRd is an option for patients who can benefit from an all-oral combination therapy, especially older patients.
Herpes zoster
All patients taking a proteasome inhibitor, including patients taking IRd, are at an increased risk for the herpes zoster infection, also called “shingles.”
All patients taking IRd should receive preventive treatment with an antiviral medication to prevent the herpes zoster infection, which is caused
by the reactivation of the varicella-zoster virus (VZV), the same virus that causes varicella (also called “chickenpox”). When reactivated, the herpes zoster infection frequently affects nerves.
Venous thromboembolism (VTE)
Patients taking IRd are at an increased risk for VTE events. VTE is a condition that includes both deep vein thrombosis (DVT) and pulmonary embolism (PE). Risk factors include infection, age > 75, cancer, and a history of VTE. All patients taking IRd should receive preventive treatment with a blood thinner (anti-coagulant) to prevent a possible VTE event. Ask your doctor about getting regular physical activity to prevent blood clots.
Important instructions for taking Ninlaro safely
Read these safety instructions carefully BEFORE you take Ninlaro:
¡ Take the IRd combination therapy exactly as your doctor instructs.
¡ Take Ninlaro on the same day each week. This is important for efficacy and safety, and will help you establish a routine for remembering to take your medication.
¡ Take Ninlaro at about the same time of day each week.
¡ Take Ninlaro at least 1 hour before, or at least 2 hours after, eating (i.e., on an empty stomach).
¡ DO NOT take Ninlaro at the same time you take dexamethasone, because dexamethasone should be taken with food, and Ninlaro should not be taken with food.
¡ Store Ninlaro capsules at room temperature in their original packaging. Do not remove the capsule from the packaging until just before you take it.
¡ Swallow the whole Ninlaro capsule with a full glass of water.
¡ Do not crush, chew, or open the Ninlaro capsule.
¡ Avoid direct contact with the contents of the Ninlaro capsule. If you accidentally get powder from inside the capsule on your skin, wash the area well with soap and water. If you get it in your eyes, flush your eyes well with water.
¡ If you miss or delay a dose of Ninlaro, take the dose as long as the next scheduled dose is more than 3 days (72 hours) away. DO NOT take a missed dose of Ninlaro if it is within 3 days (72 hours) of your next scheduled dose.
¡ If you vomit after taking a dose of Ninlaro, DO NOT repeat the dose. Just take your next dose of Ninlaro on the next scheduled day at the usual time.
¡ If you take more Ninlaro than your doctor has prescribed, call your doctor immediately or go to the nearest hospital emergency room.
¡ Tell your doctor about all other medications and supplements that you’re taking before you take your first dose of Ninlaro.
¡ Tell your doctor about all of your medical conditions. Special precautions must be taken with your dosing if you have liver or kidney dysfunction or diabetes.
Be sure to promptly report any health issues to the doctor who is treating your myeloma.
Side effects noted during TOURMALINE-MM1
A side effect, also called an adverse reaction or adverse event (AE), is an unwanted or unexpected effect caused by a drug. All the side effects experienced by patients in both arms of the TOURMALINE-MM1 clinical trial were recorded for evaluation prior to the FDA approval of IRd.
The side effects discussed below occurred most commonly among the patients enrolled in the TOURMALINE-MM1 clinical trial, but other, less common side effects occurred as well. Back pain is also common while taking Ninlaro. In addition, serious side effects outside of clinical trials have been reported to the regulatory agencies. Some side effects can be life-threatening if not managed quickly and effectively.
You must promptly report any changes in your health to your doctor while you are being treated with IRd combination therapy.
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 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.
Both Ninlaro and Revlimid can cause platelet counts to drop. During treatment with IRd, the platelet count reaches its lowest point on days 14–21 of each 28-day cycle, but usually recovers to baseline by the beginning of the next cycle. In the TOURMALINE-MM1 clinical trial, 78% of the patients in the IRd arm and 54% of the patients in the placebo-Rd arm had thrombocytopenia, some of it severe enough to be life-threatening.
Prevention and treatment of thrombocytopenia
Your doctor should monitor your complete blood count (CBC) throughout your treatment with IRd. You should alert your doctor if you experience excessive bruising or bleeding. Management of thrombocytopenia may include holding your Ninlaro and Revlimid treatment until your platelet count recovers and then adjusting your Ninlaro and Revlimid doses. Some patients with persistent low platelet counts may require platelet transfusions.
Diarrhea
In the TOURMALINE-MM1 clinical trial, 42% of the patients in the IRd arm and 36% in the placebo-Rd arm experienced diarrhea. While no patient in the study had diarrhea that was life-threatening, 6% of the cases in the Ninlaro arm and 2% of the cases in the placebo-Rd arm were severe.
Prevention and treatment of diarrhea
Your doctor may direct you to take medication to control diarrhea. If you have diarrhea, alert your doctor and take precautions to prevent dehydration by drinking a sufficient amount of water. Your doctor will monitor your electrolytes and correct abnormalities if they’re detected. Contact your doctor immediately if you get dizzy or lightheaded, or experience fainting. If needed, your doctor may withhold or adjust your doses of Ninlaro and Revlimid, or administer anti-diarrheal medication or intravenous (IV) hydration.
Constipation
The medical definition of constipation is 3 or fewer bowel movements in one week. The stool may be hard, dry, and difficult to pass. You may also have stomach cramps and bloating. Not eating and drinking enough fluids and being less active can contribute to the problem.
In the TOURMALINE-MM1 clinical trial, 34% of the patients in the IRd arm and 25% in the placebo-Rd arm experienced constipation. Less than 1% of cases in either arm were considered serious. Sometimes patients cycle back and forth between the discomfort of constipation and diarrhea. Talk to your doctor about strategies to regulate your bowel health.
Prevention and treatment of constipation
¡ Drink sufficient fluids.
¡ Try to eat high-fiber foods.
¡ Try to be active every day, even if you exercise in a chair. Moving your body increases the rhythmic contractions that move food through your intestines.
¡ Report your constipation to your doctor or nurse.
Nausea and vomiting
Nausea affected 26% and 21% of the patients in the IRd and placebo-Rd arms of the TOURMALINE-MM1 clinical trial, respectively, and vomiting occurred in 22% and 11%, respectively. These episodes were not life-threatening.
Prevention and treatment of nausea
Your doctor will give you medication to help prevent nausea and vomiting prior to each dose of Ninlaro. Precautions should be taken to prevent dehydration caused by vomiting. You must drink a sufficient amount of water and other fluids. Contact your doctor immediately if you get dizzy or lightheaded, or experience fainting. You may be given medication to prevent vomiting or IV hydration if required.
Peripheral neuropathy
Peripheral neuropathy (PN) is a serious condition that affects nerves in the hands, feet, lower legs, and/or arms. Symptoms of PN may include a feeling of numbness, tingling, burning, and/or pain. Patients may experience PN from the effects of myeloma itself and/or from treatments for myeloma. For more information, read the IMF’s publication Understanding Peripheral Neuropathy in Myeloma.
If you have PN before beginning therapy with Ninlaro, it is important that you promptly report to your treating doctor any increase in your PN discomfort.
In the TOURMALINE-MM1 clinical trial, 28% of the patients in the IRd arm reported PN, of which 18% was Grade 1. In the placebo-Rd arm, 21% of the patients reported PN, of which 14% was Grade 1. Only 2% of patients in either study arm reported PN that caused severe pain, weakness, or numbness that interfered with the activities of daily living.
Prevention and treatment of peripheral neuropathy
The best approach to treating PN is to prevent it from occurring or worsening. By promptly reporting any signs of numbness or tingling to your doctor, you can avoid potentially painful or disabling neuropathy.
Peripheral edema
Edema is an abnormal accumulation of excess fluid under the skin in the interstitial spaces within the tissues. Peripheral edema is accumulation of fluid that causes swelling in the ankles, feet, and legs. If you have swelling in one leg only, you must alert your doctor immediately, as this might signal the presence of a blood clot.
Peripheral edema can be a side effect of long-term use of anti-inflammatory medications (such as dexamethasone), which increase fluid pressure from
sodium and water retention, and thereby upset the balance of inflow and drainage of interstitial fluid. Peripheral edema can also result from other causes.
In the TOURMALINE-MM1 clinical trial, peripheral edema affected 25% of the patients in the IRd arm and 18% of the patients in the placebo-Rd arm. The majority of the cases were mild and none were life-threatening.
Prevention and treatment of peripheral edema
Patients should be evaluated for underlying causes of peripheral edema and provided supportive care, as necessary. A reduction in dietary salt intake may be required. The dose of dexamethasone may be modified, and if the edema is severe, the dose of Ninlaro should also be adjusted.
Rash
In the TOURMALINE-MM1 clinical trial, rash was reported in 19% of the patients in the IRd arm and 11% of the patients in the placebo-Rd arm.
The majority of these cases were mild, and fewer than 1% of the patients in either arm discontinued one or more of the three drugs because of a skin reaction. However, rash can be a serious concern, as a rash may be mild initially and then escalate in severity. Drug rashes vary in severity from mild redness with tiny bumps over a small area to peeling of the entire skin. Rashes may appear suddenly within minutes after a person takes a drug, or they may be delayed for hours or days.
Prevention and treatment of rash
Promptly alert your doctor if you experience a new or worsening skin rash. It may be possible to manage and reverse your skin rash, but it may be necessary to withhold your myeloma treatment regimen until the rash resolves and then resume at an adjusted dose, or your regimen may be discontinued.
Liver toxicity (hepatotoxicity)
In the TOURMALINE-MM1 clinical trial, drug-induced liver damage was reported in 6% of the patients in the IRd arm and 5% of the patients in the placebo-Rd arm. Signs of liver toxicity include yellowing of your skin or the whites of your eyes and/or pain in your right upper-stomach area.
Prevention and treatment of hepatotoxicity
Your doctor will monitor your liver enzymes with regular blood tests while you are being treated with IRd. If you have moderate to severe liver impairment, your dose of Ninlaro should be reduced.
Eye disorders
In the TOURMALINE-MM1 clinical trial, eye disorders of different types were reported in 26% of the patients in the IRd arm and 16% of the patients in the placebo-Rd arm. The most common disorders were blurred vision, dry eye, and conjunctivitis (pinkeye), an inflammation of the thin, clear tissue that lies over the white part of the eye. More serious eye disorders occurred in 2% of the patients in the IRd arm and 1% of the patients in the placebo-Rd arm.
Prevention and treatment of eye disorders
Eye disorders are readily detectable, so reporting and seeking medical attention for them can and should be done as soon as you experience a problem. Members of your healthcare team may provide supportive care or refer you to an eye specialist.
Embryo-fetal toxicity
Embryo-fetal toxicity is an exposure of an embryo or a fetus to a toxic substance. Based on animal studies, Ninlaro can cause fetal harm when administered to a pregnant female.
Prevention of embryo-fetal harm
Females of reproductive potential and males with female partners of reproductive potential should use effective contraception during treatment with Ninlaro and for 90 days following the final dose. You should not become pregnant while taking Ninlaro. You should not breastfeed while taking Ninlaro.
Access to Ninlaro and other resources
Takeda Oncology, the company that developed Ninlaro, has established the website here2assist.com to help you with the following:
¡ Working with your insurance company to help get you started on Ninlaro. Getting you started on Ninlaro treatment if there is a delay in insurance coverage determination.
¡ Providing information about specialty pharmacies to help fill and ship your prescriptions appropriately.
¡ Identifying available financial assistance that may be right for you, and connecting you to additional support services and resources.
¡ Connecting you with nurse navigators to support your Ninlaro education journey.
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.
Baseline: The initial known data that is gathered and used for comparison with later data.
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.
Cell: The basic unit of any living organism. Millions of microscopic cells comprise each organ and tissue in 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.
Cytoplasm: The jellylike material inside the membrane of a human cell, excluding the cell’s nucleus.
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.
Electrolytes: Minerals in your blood and other body fluids that carry an electrical charge and are essential for life. Electrolytes include sodium, potassium, calcium, magnesium, chloride, phosphate, and bicarbonate. They affect the amount of water in the body, the acidity of the blood (pH), nerve and muscle function (including the heart), and other important processes.
Enzyme: A protein molecule manufactured by a cell. An enzyme acts as a catalyst that increases the rate of a specific biochemical reaction in the body.
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.
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.
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.”
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.”
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.
Multiple myeloma: A cancer of the bone marrow plasma cells, white blood cells that make antibodies. Cancerous plasma cells are called myeloma cells.
Nucleus: In advanced organisms, the nucleus of the cell is the control center of the cell. It stores all the genetic material (DNA) of the cell and it coordinates the cell’s activities, including growth and reproduction (cell division).
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.
Placebo: An inert (inactive) substance often used in clinical trials for comparison with an experimental drug. No clinical trial for cancer patients in the U.S. can ethically or legally randomize patients to receive a placebo alone when they require treatment. In the placebo arm of a cancer treatment trial, patients receive treatment with approved therapy plus a placebo.
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.”
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.”
Proteins: Substances composed of amino acids. Proteins are an essential part of all living organisms, especially as structural components of body tissues such as muscle, hair, collagen, etc., as well as enzymes and antibodies.
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.
• 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).
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.
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
IMF booklets, tip cards, guides, and periodicals –subscribe to stay in the know!
Sign up at subscribe.myeloma.org for our quarterly journal Myeloma Today and weekly e-newsletter Myeloma Minute, as well as alerts about IMF news, events, and actions. And engage with us on social media!
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