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 Sarclisa® (also known as isatuximab-irfc, its generic drug name), a treatment agent that was approved for use in myeloma by the U.S. Food and Drug Administration (FDA) in 2020.
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.
FDA-approved indications for Sarclisa
The FDA has approved Sarclisa for the treatment of adult patients with relapsed or refractory myeloma as part of the following combination therapies:
¡ In combination with the immunomodulatory agent Pomalyst® (pomalidomide) + the steroid dexamethasone [Isa-Pd] for patients who have received at least 2 prior therapies, including the immunomodulatory agent Revlimid® (lenalidomide) and a proteasome inhibitor, such as Velcade® (bortezomib), Ninlaro® (ixazomib), or Kyprolis® (carfilzomib).
¡ In combination with Kyprolis + dexamethasone [Isa-Kd] for patients with relapsed or refractory myeloma who have received 1 to 3 prior lines of therapy.
How Sarclisa works
Sarclisa is a monoclonal antibody that binds to the CD38 (cluster of differentiation 38) receptor on the surface of myeloma cells. The CD system is designed to classify more than 200 proteins that can be expressed on (stick out of) the surface of a cell. CD38 is widely expressed on the surface of myeloma cells but is only expressed at low levels on the surface of other cells. Sarclisa is a targeted anti-myeloma therapy that binds to and kills myeloma cells directly, plus it also recruits surrounding immune cells to enhance its myeloma-killing process.
Clinical trial experience with Sarclisa
Clinical trials are medical research studies 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 Sarclisa was based on data from the ICARIA-MM phase III clinical trial, which compared Isa-Pd to Pomalyst + dexamethasone [Pd] alone. The study included 307 patients from 96 centers across 24 countries.
The main efficacy outcome measure was progression-free survival (PFS) using International Myeloma Working Group (IMWG) criteria. In patients treated with Isa-Pd, the improvement in PFS represented a 40% reduction in the risk of disease progression or death. Median PFS for the patients who received Isa-Pd was 11.53 months vs. 6.47 months for those who received Pd. The Isa-Pd regimen consistently prolonged PFS across subgroups, including Revlimid-refractory patients.
Adding Sarclisa to Pd improved PFS in both standard-risk patients and those with high-risk multiple myeloma (HRMM) which was defined in the ICARIA-MM clinical trial as myeloma with one or more of these chromosomal abnormalities: del(17p), t(4;14), or t(14;16).
Nearly 32% of patients in the Isa-Pd arm of the clinical trial had at least a 90% response to therapy (defined as VGPR, or very good partial response), as compared to only 8.5% of the patients in the Pd arm. Minimal residual disease (MRD) -negativity, a marker of deep response, occurred in 5.2% of Isa-Pd patients and in 0% of Pd patients.
In May 2022, results of the IKEMA phase III clinical trial, evaluating Sarclisa in combination with Kyprolis + dexamethasone (Isa-Kd) vs. Kd alone, were presented at the Controversies in Multiple Myeloma (COMy) congress.
Median follow-up was 44 months at the cutoff date of January 14, 2022. Patients in the Isa-Kd arm of the study had a median PFS of 35.7 months vs. 19.2 months in the Kd arm. This was the longest median PFS among clinical trials investigating treatment with a proteasome inhibitor in myeloma patients who relapsed after a prior therapy, including Revlimid.
In June 2022, results of an interim phase Ib clinical trial of subcutaneous (SQ, under the skin) administration of Sarclisa by a hands-free on-body delivery system (OBDS) were presented at the Annual Meeting of the American Society of Clinical Oncology (ASCO). This study of the Isa-Pd regimen in patients with relapsed/refractory myeloma demonstrated that SQ administration of Sarclisa by OBDS has a safety profile consistent with intravenous (IV, into a vein) infusion. Efficacy in the SQ cohort was comparable to the results of the ICARIA-MM phase III clinical trial.
In October 2022, the Clinical Lymphoma, Myeloma & Leukemia journal published updated data of the IKEMA phase III clinical trial, showing that treatment with Sarclisa yielded clinically meaningful improvements in duration of response (DOR) when compared to treatment without Sarclisa. Updated PFS showed significant benefit in favor of Isa-Kd. Primary PFS analysis using FDA censoring rules was 41.7 months vs. 20.8 months. Patients in the Isa-Kd arm achieved deeper responses, with final complete response (CR) rate of 44.1% vs. 28.5% in the Kd arm. The rate of patients who achieved both CR and MRD-negativity was 26.3% with Isa-Kd vs. 12.2% with Kd. These results are unprecedented for non-Revlimid regimens.
In December 2023, a plenary session at the annual meeting of the American Society of Hematology (ASH) presented the results from the ISKIA phase III clinical trial of 302 patients with newly diagnosed multiple myeloma (NDMM)
who were eligible for an autologous stem cell transplant (ASCT). Patients were randomized to two study arms with 151 patients in each arm. One arm received Kyprolis® (carfilzomib) + Revlimid® (lenalidomide) + dexamethasone [KRd] and the other arm received Isa-KRd. The study assessed efficacy and safety of Isa-KRd as pre-ASCT induction therapy and post-ASCT consolidation therapy. Compared to KRd alone, the addition of Sarclisa to KRd [Isa-KRd]
induction and consolidation therapies significantly increased the rates of MRD-negativity in every treatment phase and with no new safety concerns, including in patients with HRMM.
Safety precautions
Before starting treatment with Sarclisa, be sure to tell your doctor about all of your medical conditions, especially heart problems. Tell your doctor about all the medicines you take, including prescription and over-the-counter.
You should not receive Sarclisa if you are pregnant or plan to become pregnant, because Sarclisa may harm your unborn child. Females of reproductive potential and males with female partners of reproductive potential should ask the treating doctor if the use of effective contraception is necessary before treatment with Sarclisa begins, during treatment, and/or after the last dose of treatment is administered. Tell the doctor treating your myeloma if you become pregnant.
You should not receive Sarclisa if you are breastfeeding or plan to breastfeed, because it is not known if Sarclisa passes into your breast milk.
Dose and schedule of Sarclisa Isa-Pd regimen
¡ Sarclisa is given as an IV infusion at a dose of 10 mg/kg once-weekly for the first 4 weeks, then once every other week. The first infusion takes about 3–4 hours, the second infusion is about 2 hours, and the subsequent infusions are typically 75 minutes.
¡ Pomalyst is taken by mouth in capsule form at a dose of 4 mg on days 1–21 of every 28-day cycle. For more information about Pomalyst, read the IMF publication Understanding POMALYST® (pomalidomide) capsules.
¡ Dexamethasone can be taken by mouth or as an IV infusion at a dose of 40 mg once-weekly, or at 20 mg once-weekly for patients 75 years or older. For more information about dexamethasone, read the IMF publication Understanding Dexamethasone in the Treatment of Myeloma.
Isa-Kd regimen
¡ Sarclisa is given as an IV infusion at a dose of 10 mg/kg once-weekly for the first 4 weeks, then once every other week. The first infusion takes about 3–4 hours, the second infusion is about 2 hours, and the subsequent infusions are typically 75 minutes.
¡ Kyprolis is administered as a once-weekly IV infusion at a dose of 20 mg/m2 in the first cycle and once every other week at 56 mg/m2 during subsequent cycles. For more information about Kyprolis, read the IMF publication Understanding KYPROLIS® (carfilzomib) injection.
¡ Dexamethasone is administered on days 1, 2, 8, 9, 15, 16, 22, and 23 of each 28-day cycle at a dose of 20 mg. It is given as an IV infusion on days of Sarclisa and/or Kyprolis and taken orally by tablet on other days.
Possible side effects of Isa-Pd
Based on data from the ICARIA-MM clinical trial, the most common side effects occurring in 20% or more of patients include upper respiratory tract infection, infusion-related reactions, pneumonia, and diarrhea. The most common hematology laboratory abnormalities occurring in 80% or more of patients include decreased levels of hemoglobin, neutrophils, lymphocytes, and platelets. Possible serious side effects include pneumonia (26%), upper respiratory tract infections (7%), and febrile neutropenia (7%). Reported fatal adverse events occurring in more than 1% of patients are pneumonia and other infections (3%).
Neutropenia
Neutrophils, the most abundant type of white blood cell (WBC), are the body’s “first responders” in fighting infections. Having too few neutrophils can therefore increase the risk of infection. Approximately 85% of the patients in the Isa-Pd arm of the ICARIA-MM clinical trial had severe neutropenia vs. 70.1% of the patients in the Pd arm. Neutropenia was the most frequently reported adverse event.
Prevention and treatment of neutropenia
Your neutrophil count will be monitored frequently while you are receiving treatment with Isa-Pd. The doses of your medications may be interrupted and/or lowered if your neutrophils are too low. Your doctor may also give you a colony-stimulating factor (CSF) to increase production of your white blood cells.
Infection
Severe or life-threatening infections occurred in 42.8% of the patients in the Isa-Pd arm of the ICARIA-MM clinical trial vs. 30.2% in the Pd arm.
The most commonly reported infections were upper respiratory, including pneumonia. Immediately contact your doctor or healthcare team if you are showing any signs or symptoms of infection, including:
¡ Fever,
¡ Flu-like symptoms (body aches, sweating, chills),
¡ Sore throat,
¡ Cough,
¡ Shortness of breath,
¡ Chest pain when you breathe or cough.
Prevention and treatment of infection
You must report your symptoms to your doctor, who will determine how they should be managed and if you need to receive an antibiotic or other medications. If you develop an infection, the doses of your medications may be interrupted and/or lowered.
Infusion-related reaction
An infusion-related reaction (IRR) can occur with many IV-administered cancer therapies. IRR is caused by cytokines, small proteins released from cells targeted by the monoclonal antibody in order to affect the behavior of other cells, and also from immune cells that are recruited to the area. Reactions are often flu-like, and include nasal congestion, fever, chills, cough, throat irritation, shortness of breath, low blood pressure, nausea, and rash.
IRR occurred in 38.2% of the patients in the Isa-Pd arm of the ICARIA-MM clinical trial, but only 2.6% of those cases were severe. The overwhelming majority of IRRs were during the first dose.
Prevention and treatment of IRR
To minimize the risk of IRR, medications are given before and during Sarclisa infusions. If a reaction of any kind occurs during your infusion of Sarclisa, the infusion will be stopped.
Possible side effects of Isa-Kd
Based on data from the IKEMA clinical trial, the most common side effects occurring in 20% or more of patients include upper respiratory tract infection, IRR, fatigue, hypertension, diarrhea, pneumonia, shortness of breath, insomnia, bronchitis, cough, and back pain. The most common hematology laboratory abnormalities include decreased levels of hemoglobin, lymphocytes, and platelets. Reported fatal adverse reactions that occurred in more than 1% of patients during treatment were pneumonia (1.7%) and cardiac failure (1.1%).
Serious treatment emergent adverse events (TEAEs) were reported in 70.1% of the patients in the Isa-Kd arm vs. 59.8% in the Kd arm of the IKEMA clinical trial. The most common, any Grade, non-hematologic TEAEs with Isa-Kd were IRR (45.8%), diarrhea (39.5%), hypertension (37.9%), and upper respiratory tract infection (37.3%).
Infusion-related reaction
An infusion-related reaction (IRR) can occur with many IV-administered cancer therapies. IRR is caused by cytokines, small proteins released from cells targeted by the monoclonal antibody in order to affect the behavior of other cells, and also from immune cells that are recruited to the area. Reactions are often flu-like, and include nasal congestion, fever, chills, cough, throat irritation, shortness of breath, low blood pressure, nausea, and rash.
IRR occurred in 46% of the patients in the Isa-Kd arm of the IKEMA clinical trial, but only 0.6% were Grade 3 and none were Grade 4. The occurrence of IRR in the Kd arm was 3.3%, and none were Grade 3 or 4.
Prevention and treatment of IRR
To minimize the risk of IRR, medications are given before and during Sarclisa infusions. If a reaction of any kind occurs during your infusion of Sarclisa, the infusion will be stopped.
Diarrhea
Diarrhea is defined as 3 or more loose stools per day. Severe diarrhea is defined as 7 or more loose stools per day requiring treatment with IV fluids or hospitalization. Diarrhea occurred in 36% of the patients in the Isa-Kd arm vs. 29% in the Kd arm of the IKEMA clinical trial. Grade 3 diarrhea was noted in 2.8% vs. 2.5% of patients, respectively. No Grade 4 cases were reported.
Prevention and treatment of diarrhea
Diarrhea might occur while taking Sarclisa, causing dehydration. Precautions should be taken to prevent dehydration caused by either excessive or persistent diarrhea. Be sure to drink a sufficient amount of water. Contact your doctor if you experience dizziness, light-headedness, and/or fainting. Your doctor may administer medication or IV hydration.
Hypertension
Hypertension (HTN) is a medical condition in which the blood pressure (BP) in the arteries is elevated; also known as high blood pressure (HBP). HTN occurred in 37% of the patients in the Isa-Kd arm vs. 32% in the Kd arm of the IKEMA clinical trial.
Prevention and treatment of hypertension
If you have HTN prior to starting treatment with Kyprolis, your doctor should optimize treatment of your HTN in advance. Monitor BP carefully when receiving your myeloma therapy, especially during the first month. If very high BP or complications develop, report this to your doctor immediately. Your doctor will determine if your medication should be reduced, interrupted, or discontinued.
Infection
Upper respiratory tract infection occurred in 67% of patients in the Isa-Kd arm vs. 57% in the Kd arm of the IKEMA clinical trial. Pneumonia occurred in 36% of patients on Isa-Kd vs. 30% on Kd. Bronchitis occurred in 24% of patients on Isa-Kd vs. 13% on Kd.
Immediately contact your doctor or healthcare team if you are showing any signs or symptoms of infection, including:
¡ Fever,
¡ Flu-like symptoms (body aches, sweating, chills),
¡ Sore throat,
¡ Cough,
¡ Shortness of breath,
¡ Chest pain when you breathe or cough.
Prevention and treatment of infection
You must report your symptoms to your doctor, who will determine how they should be managed. If you develop an infection, the doses of your medications may be reduced, interrupted, or discontinued.
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. Fatigue that is related to cancer and its treatments is different from – and more severe than – normal fatigue. It tends to last longer, and includes the feeling of overall weakness.
Fatigue occurred in 42% of the patients in the Isa-Kd arm vs. 32% in the Kd arm of the IKEMA clinical trial. Grade 3 fatigue was 5% vs. 3.3%, respectively. No Grade 4 cases were reported in either study arm.
Prevention and treatment of fatigue
The effects of fatigue may be minimized by maintaining the following:
¡ A moderate level of activity,
¡ A healthy diet and proper fluid intake,
¡ A consistent sleeping schedule,
¡ Regularly scheduled visits with your doctor to monitor your red blood cell count (low red blood cells, or anemia, can cause fatigue) and to discuss issues that may contribute to your fatigue, and
¡ A careful review of the side effects of any other medications you are taking to ensure that they are not contributing to your fatigue.
For more information about this side effect, read the IMF’s publication Understanding Fatigue.
Access to Sarclisa and other resources
CareAssist by Sanofi Genzyme helps eligible patients with access and support for Sarclisa. To explore the services and resources that may be available for you, please call 1.833.930.2273 or visit SanofiCareAssist.com/Sarclisa.
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.
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.
Colony-stimulating factor (CSF): Proteins that stimulate the development and growth of blood cells. Neupogen® (filgrastim), Neulasta® (pegfilgrastim), and Leukine® (sargramostim) are colony-stimulating factors that are used to mobilize stem cells from the bone marrow into the bloodstream prior to apheresis. These may also be used after the transplant to hasten blood count recovery, or to treat low white cell count caused by therapy.
Consolidation therapy: Treatment that may be given to deepen the patient’s response after an autologous stem cell transplant (ASCT). Usually, consolidation therapy is the same regimen used for induction therapy.
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.
Duration of response (DoR): The length of time from onset of response to disease progression or death.
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.”
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.
High-risk multiple myeloma (HRMM): Myeloma that is more likely to relapse quickly after treatment or to be refractory to treatment, as defined by the cytogenetic (chromosomal) abnormalities t(4;14), t(14;16), t(14;20), del 17p, and 1q gain, along with Revised International Staging System (R-ISS) Stage III disease, and/or a high-risk gene expression profile (GEP) signature.
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.”
Infusion: Delivering fluids or medications into the bloodstream over a period of time.
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.
Minimal residual disease (MRD): The presence of residual tumor cells after treatment has been completed and complete response (CR) has been attained. Even patients who have attained a stringent CR (sCR) may have MRD. Highly sensitive testing methods are able to detect 1 myeloma cell among 1,000,000 sampled cells in blood or bone marrow. See “MRD-negative.”
Monoclonal antibody: An antibody manufactured in a lab rather than produced in the human body. Monoclonal antibodies are specifically designed to find and bind to cancer cells and/or immune system cells for diagnostic or treatment purposes. Monoclonal antibodies can be used alone, or they can be used to deliver drugs, toxins, or radioactive material directly to tumor cells.
MRD-negative: Minimal residual disease-negative. Depending on the test, not even one myeloma cell found in 100,000 or 1,000,000 sampled bone marrow plasma cells. See “Minimal residual disease (MRD).”
Multiple myeloma: A cancer of the bone marrow plasma cells, white blood cells that make antibodies. Cancerous plasma cells are called myeloma cells.
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.”
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).
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.
Treatment emergent adverse event (TEAE): An event that emerges during treatment, having been absent before treatment, or an event that worsens relative to the pretreatment state.
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.
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!
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