The International Myeloma Foundation (IMF) is the global leader in multiple myeloma, reaching more than 525,000 patients in 140 countries. The IMF mission is to improve the quality of life of myeloma patients while working toward prevention and a cure. The IMF vision is to realize a world where every myeloma patient can live life to the fullest, unburdened by the disease. Since 1990, the IMF has been serving the myeloma community through the following four pillars:
RESEARCH At the IMF, finding a cure for myeloma is our top priority. The IMF Scientific Advisory Board (SAB) of leading myeloma experts identifies key opportunities to drive research forward. The IMF Black Swan Research Initiative® is pushing the boundaries with early screening for a precursor condition of myeloma as well as cure-focused myeloma clinical trials. The IMF International Myeloma Working Group (IMWG) provides trusted guidelines for diagnosing, treating, and managing myeloma. The IMF also funds innovative research through its Brian D. Novis Research Grants.
EDUCATION Myeloma is a complex and unique journey for each patient. The IMF offers hundreds of free publications in multiple languages to help navigate the myeloma journey. IMF seminars, webinars, and workshops directly connect patients with expert clinicians. The IMF Nurse Leadership Board (NLB) provides recommendations for managing myeloma. The IMF M-Power Project works to break down barriers and ensure health equity in underserved populations.
SUPPORT The IMF offers more than 160 myeloma support groups across North America, including specialized groups for Spanish-speakers, people with smoldering disease, care partners of patients with myeloma, and patients without care partners. The IMF InfoLine helps with your myeloma-related questions. The IMF “Myelo” AI chatbot helps you find the right resources. You don’t have to face myeloma alone. Studies show that social support can greatly improve the quality of life of people with cancer.
ADVOCACY In the U.S., the IMF Advocacy team represents your interests at the federal and state levels. Internationally, the IMF Global Myeloma Action Network (GMAN) works to improve patient access to treatments.
Visit myeloma.org or contact the IMF InfoLine at 1.818.487.7455 (worldwide) or 1.800.452.CURE (U.S. and Canada), or infoline@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. If you have myeloma, it is important and helpful for you to learn about your disease, its treatment options, and supportive care measures in order to play an active role in your own medical care and to make good decisions about your care in partnership with your doctor.
If you are a patient with myeloma, we suggest that you read the IMF’s publication, Patient Handbook for Multiple Myeloma, which will help you to better understand this disease. In addition, this booklet will direct you to resources that may be relevant in your particular case. All IMF publications are free-of-charge and can be read, downloaded, or requested in printed format at publications.myeloma.org.
The IMF’s Understanding-series publications address specific drugs, drug classes, and combination therapies used to treat myeloma. These booklets also discuss supportive care measures that may help manage the symptoms and side effects of myeloma and its treatments. The IMF’s publication, Understanding Your Test Results, explains how myeloma is diagnosed, monitored, and assessed throughout the disease course.
Words in bold+blue type are explained in the IMF’s companion publication, Understanding Myeloma Vocabulary, a comprehensive glossary that also can be helpful in discussions with your doctor. Myeloma is complicated, but the language that describes it doesn’t have to be hard to understand.
If you are reading this booklet in electronic format, the light blue links will take you to the corresponding resources.
This booklet is intended for myeloma patients in the United States who are in discussions with their treating doctors about the possibility of having an autologous stem cell transplant (ASCT) as part of their overall treatment strategy. This booklet describes the typical journey of a patient with myeloma in the U.S. who might undergo ASCT, and provides information that may be helpful in decision-making.
The importance of consulting with an expert
Myeloma is a highly individualized disease. Do NOT compare your myeloma to that of other patients as each case of myeloma is unique and each patient’s myeloma has its own distinct characteristics. This is why it is so important to seek an opinion – or a second opinion – from an experienced myeloma specialist. You may be able to do this in person or your doctor can consult with a myeloma expert remotely.
Please note that while a consensus among myeloma experts about which patient will benefit most from which treatment may continue to evolve, data exists that some patients with myeloma are more likely to benefit from specific treatment approaches than other patients, and this includes ASCT. However, it is important to remember that even if you are a good candidate for a specific treatment approach – such as ASCT – there are always many factors to consider and the ultimate decision is always for the patient to make.
Rationale for ASCT in myeloma
The overall strategy for a patient with myeloma is to have as many treatment options available as possible, both short-term and long-term, in order to pursue the treatment option most likely to attain a deep and durable response. ASCT is one treatment option to consider for patients with myeloma, and it is the type of transplant used most frequently in myeloma for patients who are eligible.
The medical term for ASCT is “high-dose therapy (HDT) with stem cell rescue” and the medical term for the immature cells from which all blood cells develop is “hematopoietic stem cells.”
Normal stem cells give rise to normal blood components, including red blood cells (RBC), white blood cells (WBC), and platelets. Normal stem cells originate in the bone marrow, the soft and spongy tissue in the center of bones.
Figure 1. Blood composition
Red blood cells
White blood cells
Slaybaugh Studios
In patients with myeloma, the myeloma cells are present in the same bone marrow microenvironment as the normal stem cells. As myeloma cells build up in the bone marrow, they become intermixed with normal stem cells.
Chemotherapy is the use of drugs to kill cancer cells. HDT is more effective at eradicating myeloma cells from the bone marrow than standard-dose chemotherapy. But any treatment that reaches the bone marrow to kill myeloma cells is also capable of damaging your normal stem cells. With reduced bone marrow, your body is less able to produce blood cells that carry oxygen into organs and tissues (RBC), help your blood to clot (platelets), and defend against infection (WBC).
In the ASCT procedure, your peripheral blood stem cells (PBSC) are first collected (“harvested”) from the circulating blood after a mobilizing agent triggers the release of bone marrow stem cells into the bloodstream – this is why ASCT is a “stem cell transplant” and not a “bone marrow transplant.” After HDT has been administered, your own harvested stem cells are reinfused (“transplanted”) into you to “rescue” your bone marrow from the effects of HDT.
Prior to making a decision about ASCT, consider the following key points:
¡ Discuss the benefits and risks of ASCT and other treatment options with your doctor.
¡ ASCT can improve the depth of response and duration (length) of remission, known as progression-free survival (PFS). Transplant doctors usually require at least a 50% reduction in levels of monoclonal protein (myeloma protein, M-protein) as well as other indicators prior to harvesting normal blood stem cells.
¡ While the rates of PFS may be improved with ASCT, the rates of overall survival (OS) are the same with or without transplant.
¡ Myeloma patients who have no minimal residual disease (MRD) after treatment, also called being MRD-negative, have improved OS whether they have undergone ASCT or not. MRD-negativity improves outcomes with or without ASCT.
¡ Quality of life (QoL) and overall costs of treatment favor early ASCT because this produces higher rates of sustained PFS and MRD-negative status.
¡ Discuss with your doctor the option of harvesting your stem cells, then freezing them without undergoing immediate ASCT. If there is no plan for immediate ASCT, discuss and develop a treatment plan with your doctor.
¡ Ask your doctor to explain the proposed treatment plan for your myeloma. Please continue reading for a more detailed discussion of ASCT.
2. ASCT factors to discuss with your doctor
Eligibility and decision-making Assessment
In the U.S., eligibility criteria for ASCT varies among institutions. In 2014, the IMF’s International Myeloma Working Group (IMWG) published a consensus statement, which concluded that the arbitrary age of 65 is no longer sufficient to define whether a patient is eligible or ineligible for ASCT. Physiologic age is a more important factor than chronological age, but this does not mean that age is not a factor at all. Your general fitness and any concurrent illnesses will be part of the evaluation to determine appropriate treatment.
In the U.S., there is no upper age limit set in the Medicare National Coverage Determination for ASCT in myeloma, and the Centers for Medicare & Medicaid Services (CMS) have now removed the upper age limit on coverage, leaving the question of eligibility to the patient’s doctor and the Medicare administrative contractor within the patient’s jurisdiction. Visit cms.gov/Medicare/Coverage/DeterminationProcess for more information or speak with your healthcare team.
While ASCT is an option for most myeloma patients upon completion of frontline therapy, not all patients are candidates for this intensive approach. Decisions regarding transplant should be made based on a risk-benefit assessment, as well as the needs and wishes of the patient. You and your doctor must consider all the relevant myeloma-related factors and patient-related factors. The factors to discuss with your doctor include, but are not limited to, the following:
¡ Myeloma-related factors include the stage of disease, its level of aggressiveness, response to prior treatments, beta-2 microglobulin (β2-microglobulin, β2M, or β2M), serum albumin, and chromosomal abnormalities.
Figure
¡ Patient-related factors include age, general physical condition, the presence of other medical conditions, and function of the kidneys, heart, lungs, and liver.
¡ Patient preference, as well family and work situations, should be factored into the discussion.
Your doctor will perform a series of tests, and the gathered data will help assess your candidacy for ASCT. Please discuss the following with your doctor before making your decision:
1. Confirm your diagnosis of myeloma and that your myeloma is active and that it requires treatment. If there is any doubt, this is a key moment to seek an opinion from a myeloma expert before moving ahead with a treatment strategy. Discuss with your doctor the treatment strategy optimal for you.
2. After each cycle of induction therapy (usually every 3 to 4 weeks), ask your doctor about your response to treatment. Learn to understand the complete evaluation of the level of response, including bone marrow testing and imaging studies. If your response to therapy is deemed inadequate by your doctor, then other therapy may be recommended before proceeding to ASCT.
3. Ask your doctor if you may run the risk of serious complications.
For information about tests used to monitor and assess myeloma status throughout the disease course, read the IMF’s publication Understanding Your Test Results.
Timing
ASCT may be performed as part of a frontline therapy regimen or at the time of myeloma relapse. When to perform ASCT is an important decision. Most transplant doctors believe that it is better to perform ASCT early in the disease course. However, there is also evidence that if a patient has a deep response to induction therapy, stem cells can be collected and frozen, but the ASCT can be delayed until relapse. Discuss your optimal timing for an ASCT with your doctor.
Chemotherapy
If your doctor considers ASCT to be an option for you, then your initial therapy should be with drugs that don’t damage your normal stem cells prior to harvesting. For example, therapy with Revlimid® (lenalidomide) for more than 4 cycles may impair stem cell collection.
It is also important to note that the use of alkylating agents should be avoided because they can lead to damage of normal stem cells. Alkylating
agents are chemotherapy drugs that cross-link the DNA of myeloma cells and block cell division. Alkylating agents were the earliest effective drugs used in the treatment of myeloma.
Preparing for the experience of ASCT
You should feel comfortable and reassured before you begin your ASCT process. You can do a lot to get ready for your ASCT. The IMF has a library of publications about the therapies used for the treatment of myeloma at every stage of the disease. All are free-of-charge and can be downloaded or requested in printed form at publications.myeloma.org.
Be empowered to ask your doctor any questions you may have. Some transplant centers may even pair you up with a fellow patient who has been through the ASCT process and can share a firsthand account of their experience. If possible, bring a friend or a family member to your doctor appointments so that they can help take notes. If available, ask your doctor to provide an “after-visit summary.”
Share what you learn with your loved ones so that they know what to expect – and how they can best help in the weeks and months ahead.
Your transplant center and team
Ask your doctor whether you will receive treatment on an inpatient or outpatient basis. Visit the transplant center and see the rooms where the ASCT procedure and recuperation would take place. If the transplant center location is far from your home, visit the accommodations where you will stay. Many transplant centers have accommodations nearby or can help you find suitable accommodations.
Ask to meet your myeloma transplant team in advance – the doctors, nurses, social workers, psychologists, and allied healthcare professionals. This will help you know who’s who, as well as what resources are available to you and your care partners. ASCT is a complicated medical procedure and you want your transplant team to have both experience and expertise.
Support resources
Having a support network is very important. ASCT can place overwhelming stresses on patients and their loved ones before, during, and after the procedure. Physical, psychological, emotional, and financial stresses may trigger feelings of anxiety, depression, or anger. We urge you to take advantage of the support resources offered through your hospital, the IMF’s InfoLine and support groups, and through other patient-centric organizations. Ask your doctor about the benefits of psychological counseling or psychiatric consultation.
The process of ASCT in myeloma
Induction therapy
Depth of response after induction therapy is generally thought to influence the depth of response after ASCT. However, please note that even lesser degrees of response to induction therapy may be sufficient for effective stem cell collection and for proceeding with ASCT.
Patients have many options for induction therapy prior to ASCT, including the following:
¡ Darzalex Faspro® (daratumumab + hyaluronidase-fihj) + Velcade® (bortezomib) + Revlimid + dexamethasone [DVRd] regimen was approved by the FDA in July 2024 based on data from the PERSEUS phase III clinical trial of DVRd vs. VRd. Patients were 70 years or younger and had a performance status of 0 to 2 based on the scale by the Eastern Cooperative Oncology Group (ECOG), now part of the ECOG-ACRIN Cancer Research Group.
The PERSEUS study demonstrated a 60% reduction in risk of disease progression or death with the use of DVRd. Further, there was a significant advantage in overall MRD negativity rate in the DVRd study arm (57.5%) vs. the VRd study arm (32.5%). Additionally, MRD negativity among those who also achieved a complete response (CR) or better was 76.6% in the DVRd study arm vs. 58.5% in the VRd study arm.
¡ Velcade + Revlimid + dexamethasone [ VRd] was studied in the two largest ASCT studies, the IFM clinical trial and the DETERMINATION clinical trial. However, in 2024, the 4-drug DVRd regimen (see above) demonstrated its clinical superiority to the 3-drug VRd regimen.
¡ Kyprolis® (carfilzomib) + Revlimid + dexamethasone [KRd] has been used and studied before and after ASCT. The FORTE clinical trial demonstrated high efficacy with a favorable safety profile of KRd + ASCT in newly diagnosed myeloma patients.
¡ Darzalex + KRd [DKRd] is a four-drug combination being used more frequently now. Clinical data has shown that the DKRd induction regimen is both safe and yields deep responses prior to ASCT, and can lead to a high rate of MRD-negativity in newly diagnosed myeloma patients after ASCT.
While the vast majority of myeloma patients who proceed to ASCT are being treated with the above-mentioned induction therapies, additional treatment options are listed by the National Comprehensive Cancer Network (NCCN) in its guidelines for the management of myeloma:
¡ Revlimid + dexamethasone [Rd],
¡ Velcade + cyclophosphamide + dexamethasone [ VCd or CyBorD],
To circumvent potential damage of normal blood stem cells in the bone marrow, the stem cells are harvested before you receive chemotherapy with an alkylating agent. This is done through a process called apheresis. A thin flexible catheter is inserted through the skin and into a vein, and blood from the patient is passed through a special machine to remove the stem cells. The rest of the blood is immediately returned to the patient. Apheresis is usually done as an outpatient procedure for 1 to 5 days, lasting from 3 to 4 hours each day.
One of the following protocols may be used prior to harvesting your stem cells:
¡ Protocol 1: The patient receives subcutaneous (SQ) injections with a colony-stimulating factor (CSF) to mobilize the release of stem cells from the bone marrow into the bloodstream. The daily injections are followed by the daily harvesting of stem cells until a sufficient quantity is obtained. Typically, the aim is to harvest enough stem cells for two transplants. The amount of stem cells reinfused into the patient has an impact on the recovery of the patient’s blood cell counts.
¡ Protocol 2: The patient receives both CSF and chemotherapy. Ask your doctor to explain the potential benefits and side effects of using chemotherapy in addition to growth factors. The most commonly used drug to enhance the release of stem cells from the bone marrow into the bloodstream is cyclophosphamide, but there are other drugs that can be used instead. An advantage of cyclophosphamide is that it is also used to treat myeloma, although it usually requires hospitalization while stem cell production is being stimulated. A disadvantage of cyclophosphamide is that it lowers WBC counts and infection might result, possibly requiring another hospitalization. After chemotherapy is completed, a WBC growth factor is given daily by injection for approximately 10 days, then the stem cells are harvested over 2 to 5 days, while the patient is still receiving growth factor injections.
¡ Protocol 3: The patient receives CSF plus a mobilizing agent. In 2008, the FDA approved Mozobil® (plerixafor) for stem cell mobilization in combination with a growth factor. Treatment with growth factors lasts 4 days, then Mozobil is injected under the skin approximately 11 hours before stem cell collection begins. Mozobil is particularly helpful for patients who have difficulty generating enough stem cells for harvesting. There is also the likelihood of fewer apheresis procedures, with reduced number of days on the apheresis machine. In September 2023, the FDA approved Aphexda® (motixafortide) to mobilize hematopoietic stem cells for ASCT. One dosage of Aphexda + filgrastim (a growth factor) enabled a majority of myeloma patients in the clinical trial to achieve the collection goal of ≥ 6 million hematopoietic stem cells.
Next, the collected stem cells are taken to a processing laboratory, where they are frozen (cryopreserved) in liquid nitrogen and stored at a temperature of –80°C (–112°F) for later use within days, weeks, or years. Excellent function of stem cells is retained for at least 10 years.
Note: Scientific evidence indicates that “purging” myeloma cells from the harvested stem cells is not effective in ASCT for myeloma.
High-dose therapy
When you are ready to proceed with ASCT, your doctor will try to first reduce your tumor burden with myeloablative HDT, which destroys myeloma cells in the bone marrow where they grow. However, normal cells are also destroyed. The most common type of HDT used in myeloma is melphalan, administered at a dose of 200 milligrams per square meter (mg/m2) of body surface area (size of patient).
Medications are given to prevent or lessen the anticipated side effects of HDT. Common side effects of HDT include nausea, vomiting, diarrhea, mouth sores, skin rashes, hair loss, fever or chills, and infection. Patients are monitored very closely during and after the administration of HDT, including daily measurement of weight, blood pressure, heart rate, and temperature.
Stem cell rescue
Approximately 36 to 48 hours after the HDT is administered, the levels of melphalan in your blood and tissue are very low and do not harm the reinfused stem cells. Your frozen stem cells are thawed in a warm water bath and reinfused back into your bloodstream over a period of 1 to 4 hours through a catheter. The chemical used to keep stem cells fresh has a garlic smell – you may even experience the taste of garlic.
Engraftment
Engraftment is the process by which the reinfused stem cells migrate from the bloodstream to your bone marrow, where they begin to produce new
blood cells to replace the normal stem cells destroyed by HDT. You may receive SQ injections of growth factors to help stimulate your bone marrow to produce normal blood cells. Your stem cells will begin to grow back within 10 to 14 days after reinfusion, and your blood counts will begin to recover. You may receive transfusions if necessary.
Some transplant centers may require you to remain in the hospital on an inpatient basis after the reinfusion, and some centers have facilities nearby where you may stay while being monitored daily at the hospital on an outpatient basis. The length of stay varies patient-to-patient but is usually around 2 to 3 weeks. If you live near the transplant center, you may be able to sleep at home and come to the hospital for daily monitoring on an outpatient basis.
When you are discharged, your recovery will continue at home for about 2 to 4 months. Often, the most difficult time is waiting for the reinfused stem cells to engraft, for blood counts to return to safe levels, and for side effects to resolve. On some days you may feel better, and on other days you may feel too weak to do much more than sleep.
Recovery may feel like a rollercoaster ride. Frequent visits to the hospital may be required to monitor your progress. It is important to take things one day at a time. As your bone marrow produces new blood cells, symptoms resolve, the risk of serious infections is reduced, and transfusions may no longer be needed.
Depth of response
HDT with stem cell rescue delivers further improvement in the level of response achieved by induction therapy. More than half the time, partial response (PR) will be improved to either very good partial response (VGPR) or complete response (CR). The ultimate goal of HDT with stem cell rescue is to eliminate all residual myeloma cells that have not been killed during induction therapy.
Consolidation therapy
Consolidation therapy is treatment that may be given after ASCT to further deepen response, usually with the same drug regimen used for induction. Several protocols in current clinical practice include additional cycles of induction therapy after ASCT and prior to commencing maintenance therapy. Some transplant centers pursue an additional ASCT, but this is less common in the United States.
Maintenance therapy
Maintenance therapy is treatment that may be given after ASCT to prolong response. Currently, Revlimid is the only myeloma treatment approved
by the FDA for maintenance after an ASCT. Multiple clinical trials have reported higher rates of PFS and OS in patients who received Revlimid as maintenance therapy post-ASCT (vs. placebo as maintenance therapy), regardless of the depth of response following ASCT.
There is a trend of more frequent use of “doublet” (2-drug) maintenance therapy regimens, especially in patients with higher-risk myeloma. In patients with high-risk multiple myeloma (HRMM), a more intense maintenance therapy may be considered. Post-ASCT maintenance therapy remains an area of study and data is evolving.
A 2014 meta-analysis of 3,218 patients in 7 clinical trials showed that an increase in second primary malignancies (SPM) could arise from the use of Revlimid in combination with melphalan. There has been no increase in SPMs reported among relapsed or refractory patients treated with Revlimid in the absence of an alkylating agent.
Given the advantages and potential risks of post-ASCT maintenance therapy with Revlimid, discuss with your doctor your individual risk factors and your response to ASCT before making any decision.
The role of tandem ASCT
ASCT can be performed once (a “single” autologous transplant) or twice (“double” or “tandem” autologous transplants done in succession). Tandem ASCTs are usually planned with an interval of 3 to 6 months between the two transplants. Tandem transplantation for myeloma has become less common in the U.S. due to the emergence of effective novel therapies.
The role of a second ASCT
Long after a patient’s first ASCT, a second transplant may be an option for relapsed myeloma. A second ASCT appears to confer benefit. It is also a viable option for patients who achieved response of at least an 18-month duration following a first ASCT, but then relapsed. This is one of the reasons that enough stem cells for two ASCTs may be collected in advance.
Possible side effects of ASCT
Side effects are a possibility with every type of medical treatment or procedure. Each patient reacts differently at each step of the ASCT process. No two patients share exactly the same side-effect profile. The following are the most common potential side effects following HDT with stem cell rescue: nausea, mouth sores, hair loss, infection, and fatigue.
In part, the appropriate management of side effects is why it is so important to have your ASCT performed at a transplant center where the doctors, nurses, and allied healthcare professionals have performed the ASCT
procedure many times on many myeloma patients. Such a team is more likely to have the experience and the expertise to care for each individual patient’s needs.
Until engraftment of the reinfused stem cells takes place, patients are very susceptible to developing infections, and infections can cause serious complications and even be potentially life-threatening. Even a minor infection like the common cold can lead to serious complications because the body’s immune system is weakened by the effects of HDT. Special precautions are necessary during recovery. To protect the patient and prevent infection, the following supportive care measures may be required:
¡ Antibiotics may be prescribed to help prevent infection.
¡ Visitors may be asked to wash their hands, and wear masks and rubber gloves.
¡ Fresh fruits, vegetables, and flowers may be prohibited from the patient’s room as these can carry infectious agents such as bacteria and fungi.
If infection or fever occurs as a result of lowered WBC counts, the patient may be given an intravenous (IV) infusion of antibiotics.
Other side effects to be aware of and discuss with your transplant team include nausea, fatigue, mouth sores, diarrhea, and skin rash.
Life after ASCT
Data from the Health Resources and Services Administration (HRSA) demonstrate that 99.1% of myeloma patients in the U.S. are alive at 100 days following ASCT.
ASCT is more than just a medical procedure, and you will need to rely upon your doctor and other members of the transplant team, as well as on the support of your family and friends. It is not uncommon for patients to experience a loss of the sense of independence and control, while at the same time experiencing feelings of isolation, depression, and helplessness. Patients and their care partners may consider seeking assistance from a trained counseling professional. There may also be benefits to participating in patient support groups either in-person or virtually.
On average, it takes 3 to 6 months to recover from an ASCT. By this time, your bone marrow will be producing healthy blood cells and your immune system will once again be able to fight infection. Your hair will grow back, but some foods that tasted good before your ASCT may no longer taste good. In most cases, patients are able to return to normal daily activities, but it may take 6 months or more after ASCT for some patients to return to normal functioning or to full-time work.
There will be bad days and good days, and they won’t necessarily come in that order. Patients may feel differently each day during the recovery process. Patients and their care partners must take it one day at a time.
Some people believe in the intended benefits of alternative and complementary therapies, but it’s important to remember that all medicines –synthetic and natural – may interact and create unanticipated effects. Patients should always inform their doctor of all medications and therapies being taken in addition to the doctor’s prescribed protocol. Even over-thecounter products may be harmful to a patient with myeloma.
Vaccination and immunization
These terms are often used interchangeably, but vaccination is the act of introducing a vaccine into a body to produce protection from a specific disease, and immunization is the process by which a body becomes protected against that disease through vaccination. If you are planning for an ASCT, you must also make plans with your doctor to be re-immunized on a set schedule. Your re-immunization plans should be based upon local guidelines.
Current guidelines of the American Society for Transplantation and Cellular Therapy (ASTCT), European Bone Marrow Transplant (EBMT), and the Infectious Disease Society of America (IDSA) advise that re-vaccination should start between 6 and 12 months after ASCT. Additionally, these guidelines state that myeloma patients on active therapy should not be vaccinated. Please ask your doctor how this applies to your specific case of myeloma.
Relapse
Unfortunately, the majority of patients with myeloma experience a relapse after transplant is completed. The prevailing theory is that it is not the myeloma cells among the harvested stem cells that cause disease relapse, but rather the myeloma cells that remain in the body after systemic treatment.
In 2007, the journal Haematologica published the findings of the EBMT group from a large, randomized clinical trial. The study definitively demonstrated that there was no difference in relapse rate between myeloma patients who received grafts with detectable myeloma cells vs. patients receiving grafts with no detectable myeloma cells.
In December 2023, at the annual meeting of the American Society of Hematology (ASH), long-term followup results were presented of the GMMG “ReLApsE” phase III randomized clinical trial of salvage therapy with ASCT + Revlimid maintenance vs. continuous Rd for relapsed myeloma. No significant PFS or OS difference was observed in the study participants.
Other types of stem cell transplants
In addition to ASCT, there are other types of transplants.
Allogeneic (allograft) transplant
This type of transplant uses stem cells or bone marrow harvested from a donor who has been determined to be a compatible match with a recipient by means of a human leukocyte antigen (HLA) test . The donor cells are infused into the patient after myeloablative HDT. The donor’s immune system cells recognize the recipient’s myeloma cells as foreign and attack them. Unfortunately, the donor cells also attack other tissues in the recipient’s body, causing graft-versus-host disease (GVHD), which may cause complications or may even be fatal.
A type of an allogeneic transplant, sometimes called “mini-allo” for short. RIC transplant is a newer and, for myeloma, a safer technique than a full allogeneic (allograft) transplant because RIC transplant is non-myeloablative. RIC transplant is usually performed within 180 days after a standard ASCT.
Bone marrow transplant
A type of an autologous transplant where stem cells are collected from a patient’s bone marrow, not from a patient’s circulating peripheral blood. Currently, bone marrow transplantation is used infrequently in myeloma because the ASCT procedure is preferred. But bone marrow transplantation may be considered if stem cells are not able to be collected from the peripheral blood.
Syngeneic transplant
A type of an allogeneic transplant where bone marrow or stem cells from one identical twin sibling (donor) are infused into the other identical twin (recipient).
Matched unrelated donor (MUD) transplant
A type of an allogeneic transplant where stem cells are genetically matched to the patient but are not from a donor who is a family member. In myeloma, this type of transplant carries a high rate of GVHD and is therefore very rarely used.
Umbilical cord stem cell transplant
A type of allogeneic transplant where stem cells are harvested from multiple umbilical cords of newborns in order to obtain enough stem cells for an adult transplant. In myeloma, this type of transplant carries a high rate of GVHD and is therefore very rarely used.
ASCT in clinical trials
The use of HDT was introduced in myeloma in the 1990s, followed by the introduction of new treatment options in the 2000s. This led to improved outcomes in ASCT-eligible myeloma patients. The early clinical trials established a therapeutic approach of induction, followed by ASCT, consolidation therapy, and maintenance therapy. More recently, the use of novel therapies in each phase of myeloma treatment has significantly improved the efficacy of ASCT, including MRD-negativity.
For myeloma patients considering ASCT, the key takeaways from this booklet remain relevant as of the time of its printing, although new and updated data from clinical trials continues to be produced and presented.
If you have any questions or concerns about ASCT, speak with your doctor and visit myeloma.org/online-resources/transplant to familiarize yourself with resources that may be of interest to you.
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.
