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Information regarding treatments for the disease is constantly changing, and the MMRF will update this section as new discoveries take place. The MMRF advises patients and caregivers to work with their physicians regarding specific questions relating to treatment and urges health care professionals to share this information with their patients.
Use the links on the right to browse treatments by category, or go directly to information about a specific treatment using the pull-down directory below.
Treatment Decisions in Myeloma
Deciding on a particular treatment for myeloma is a complex process. Treatment is tailored to each patient according to several factors, including:
- results of the physical exam
- results of laboratory tests
- the specific stage or classification of their disease
- their age and general health
- their symptoms
- whether complications of the disease are present
- whether they have previously received treatment for their disease
- their lifestyle and their view and philosophy on quality of life
Goals of Treatment
Treatment regimens may be designed to meet one or more different therapeutic goals, which can include:
- Eradicating all evidence of disease, which may require accepting higher levels of toxicity
- Controlling disease activity to prevent damage to other organs of the body, using a regimen with an acceptable toxicity level
- Preserving normal performance and quality of life for as long as possible with minimal intervention
- Providing lasting relief of pain and other disease symptoms
- When applicable, managing myeloma that is in remission over the long-term
With the many promising therapies in clinical trials, we hope to include the goal of a cure for myeloma in the near future!
Potential Outcomes of Treatment
There are several potential outcomes of treatment in myeloma, which are summarized in the table below. Although cures have not been documented in patients with myeloma, molecular complete responses have been achieved with some of the new therapies in clinical trials. Relapses still occur after molecular complete response, usually after a longer period of event-free survival. Evolving therapies may offer patients a greater chance of achieving a molecular complete response. However, there is currently no evidence that the quality of a response affects how long a patient lives.
Potential Outcomes of Treatment in Myeloma | ||||||||||||||||||||
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* Note that in all outcomes, response may also be referred to as remission. These terms are interchangeable.
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We have made much progress in our understanding of myeloma and in these promising therapies. However, we have yet to achieve the maximal benefit possible with regard to patient survival and improvement in quality of life. For these reasons, patients with myeloma should strongly consider participating in a clinical trial if it is an appropriate option for them.
Management Options for Myeloma
Myeloma therapy is tailored to each patient. It is important to note that there is no one "standard therapy" for active myeloma. The treatment regimens that are often referred to as standard therapies are those that have been traditionally used for the treatment of the disease and have an established track record of usefulness as documented through publication in reputable scientific journals.
Treatment of myeloma can be a complex process because many variables must be taken into account, such as the patient's overall state of health, other medical issues/diseases, and how well the disease is currently controlled, as well as the type, number, and response to previous therapy. Moreover, there is no single test result that can lead to a diagnosis of myeloma and determine its prognosis; many factors must be considered.
Many centers have developed their own guidelines for treating myeloma and these may vary from center to center. The National Comprehensive Cancer Network (NCCN) has also developed a set of general practice guidelines to aid physicians in treating myeloma. These guidelines indicate particular therapies that have been deemed appropriate based on a review of the literature and on the expertise and clinical experience of a panel of experts. (NCCN Practice Guidelines in Oncology - Version 1.2004. Multiple Myeloma. Available at www.nccn.org.)
To further advance therapy for multiple myeloma, it is highly suggested that all eligible patients be included on clinical trials. Clinical trials, especially Phase II and Phase III trials, are designed to be at least as effective as what is considered standard therapy.
Management options depend on a patient?s disease classification and disease status.
Treatment of myeloma can be a complex process because many variables must be taken into account, such as the patient's overall state of health, other medical issues/diseases, and how well the disease is currently controlled, as well as the type, number, and response to previous therapy. Moreover, there is no single test result that can lead to a diagnosis of myeloma and determine its prognosis; many factors must be considered.
Many centers have developed their own guidelines for treating myeloma and these may vary from center to center. The National Comprehensive Cancer Network (NCCN) has also developed a set of general practice guidelines to aid physicians in treating myeloma. These guidelines indicate particular therapies that have been deemed appropriate based on a review of the literature and on the expertise and clinical experience of a panel of experts. (NCCN Practice Guidelines in Oncology - Version 1.2004. Multiple Myeloma. Available at www.nccn.org.)
To further advance therapy for multiple myeloma, it is highly suggested that all eligible patients be included on clinical trials. Clinical trials, especially Phase II and Phase III trials, are designed to be at least as effective as what is considered standard therapy.
Management options depend on a patient?s disease classification and disease status.
Disease Classification
Patients with myeloma can classified as having either inactive disease or active disease.
Inactive disease is asymptomatic disease that does not require immediate treatment. Patients with inactive disease do not have evidence of any myeloma-related organ or tissue impairment (also known as end-organ damage). Inactive disease includes the following classifications subtypes of myeloma:
Inactive disease is asymptomatic disease that does not require immediate treatment. Patients with inactive disease do not have evidence of any myeloma-related organ or tissue impairment (also known as end-organ damage). Inactive disease includes the following classifications subtypes of myeloma:
Disease Status
The treatment options available to a patient also take into account their disease status, that is, whether they have already received therapy and if so, what was the outcome.
Patients with newly diagnosed disease are individuals who have myeloma that has not yet been treated.
Patients who have received therapy may fall into several categories:
Patients with newly diagnosed disease are individuals who have myeloma that has not yet been treated.
Patients who have received therapy may fall into several categories:
- Responsive disease refers to myeloma that is responding to therapy. There has been a decrease in M protein of at least 50%. Some myeloma groups consider a decrease in M protein between 25% and 50% to be a minimal response whereas others consider this to be stable disease.
- Stable disease refers to myeloma that has not responded to treatment (ie, the decrease in M protein has not reached 50%), but has not progressed (gotten worse). Some myeloma groups consider a decrease in M protein between 25% and 50% to be a minimal response.
- Progressive disease refers to active myeloma that is worsening (ie, increasing M protein and worsening organ or tissue impairment [end organ damage]). In most cases, relapsed and/or refractory disease can be considered to be progressive disease.
- Relapsed disease refers to myeloma disease that initially responded to therapy but has then begun to progress again. Patients may be further classified as having relapsed after initial therapy or after subsequent therapy.
- Refractory disease refers to myeloma that has not responded to initial therapy, as well as relapsed myeloma that does not respond to subsequent treatment. In this last instance, the myeloma may also be referred to as relapsed and refractory disease.
- Refractory disease can be of two types:
- Non-responding progressing refractory disease, which refers to refractory disease that is progressing. This is a difficult type of myeloma to treat and some form of innovative therapy is usually suggested as a treatment option.
- Non-responding non-progressing refractory disease, which refers to refractory disease that is not worsening. Patient with this type of disease often do as well as patients with responsive disease.
Treatment Approaches
Treatment approaches to myeloma depend on whether a patient has inactive or active disease. This section provides an overview of the various treatment approaches and therapeutic options for myeloma.
Inactive Disease
Patients with inactive (asymptomatic) disease are typically observed and not treated unless their disease begins to progress. No therapy is recommended outside of a clinical trial unless there is evidence of active disease with end organ damage. However, in some cases, bisphosphonates and other forms of supportive care may be appropriate for patients with smoldering or indolent myeloma or Stage 1 disease. Participation in a clinical trial is also an option. For example, agents such as thalidomide are being tested in patients with inactive disease. You can access the MMRF Clinical Trials Monitor (CTM) to see what clinical trial options might be appropriate for you.
- Find out more about Bisphosphonates
- Find out more about Supportive Care
- Search the MMRF's Clinical Trials Monitor
Active Disease
Patients with newly-diagnosed, active (symptomatic) myeloma typically receive some form of initial therapy, as well as bisphosphonates and supportive care as required to treat bone disease and other complications of the disease. Subsequent treatment options are often decided based on previous treatments received and the outcome.
The pathway below and the text that follows outline the typical options available to a newly-diagnosed patient with symptomatic myeloma requiring treatment. Participation in a clinical trial is an option at virtually every step in the pathway.
The pathway below and the text that follows outline the typical options available to a newly-diagnosed patient with symptomatic myeloma requiring treatment. Participation in a clinical trial is an option at virtually every step in the pathway.
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(Adapted from Rajkumar et al. Mayo Clin Proc. 2002;77:814.) |
The choice of initial therapy is dependent on whether a patient is a candidate for high-dose chemotherapy and autologous stem cell transplant, a therapeutic strategy that offers improved response rates and survival in myeloma. Patients under the age of 65 in good physical condition with adequate kidney, lung, and heart function are potential transplant candidates. However, older patients may be eligible if they are in very good health.
Non-transplant Candidates
If a patient is not a candidate for an autologous transplant, the most common initial treatment is a combination of melphalan, a type of chemotherapy, and prednisone, a type of corticosteroid. Cyclophosphamide and prednisone (CP) or other combinations of agents may also be used. High-dose dexamethasone (another corticosteroid) is often used in older patients who may be unable to tolerate other therapies, as is combination Thalomid® (thalidomide, Celgene) and dexamethasone.
Non-transplant candidates typically continue initial therapy for about a year or until their response reaches a plateau. At that time they may receive some form of maintenance therapy, such as corticosteroids, and continue to receive bisphophonates and supportive care as required. Patients who initially respond to therapy, however, eventually relapse (that is, disease begins to progress again). Patients who relapse shortly following completion of initial therapy may no longer respond to the initial medications used. These patients, as well as those who do not respond to initial therapy, are said to have refractory disease. Treatments for relapsed or refractory disease are described below.
Transplant Candidates
Certain chemotherapy agents, such as melphalan (which is also known as an alkylating agent) may impair the ability to collect stem cells for use in an autologous transplant and should be used with care. For this reason, patients who are candidates for autologous transplant often receive other agents as initial therapy or receive melphalan for only a brief period before their stem cells are collected. Options include combination thalidomide and dexamethasone, which is now the most frequently used initial therapy in transplant candidates, as well as dexamethasone or the chemotherapy regimen known as VAD (vincristine, doxorubicin, dexamethasone). A modification of the VAD regimen known as DVd (Doxil® [liposomal doxorubicin, Ortho Biotech], vincristine, short-schedule Decadron [dexamethasone]), which can be administered on an outpatient basis, may also be used. After 3 or 4 cycles of therapy, which minimizes the tumor burden, transplant candidates have their stem cells collected for use in their transplant.
After stem cell collection, transplant candidates may proceed directly to an autologous transplant ("early transplant"). Alternatively, they can continue to receive their initial therapy until plateau and then receive their transplant at the time of relapse ("late transplant"). Some prefer early transplantation because it minimizes a patient's time on chemotherapy and overall quality of life may be improved.
Treatments for Relapsed or Refractory Disease
Treatments for relapsed and/or refractory disease are often referred to as salvage therapy. If a relapse occurs after 6 months of discontinuing therapy, the initial therapy may be reconsidered. Other options for treating relapsed or refractory disease include participation in a clinical trial, various salvage chemotherapy agents or combinations, thalidomide-based regimens, bortezomib (Velcade®), or stem cell transplant if possible.
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