Multiple myeloma is the second most common blood cancer in the United States, with about 26,850 new cases diagnosed annually, representing about 2% of all cancers.1 Over the past 10 years, dramatic progress has been made in expanding treatment options for patients with multiple myeloma, and ongoing research is focused on the development of newer and better therapies. Patients with multiple myeloma are living longer and are dealing with a new set of challenges. A roundtable discussion of patients with multiple myeloma was held in Boston on July 14, 2015, with the goal of gaining insights into the challenges and gaps that patients and their loved ones are facing.
This article summarizes the views of the patients regarding their experiences with current treatments and other issues related to cancer care. The participating patients were Jack Aiello, Cheryl Boyce, Yelak Biru, Cynthia Chmielewski, and Gary Petersen. They have all had multiple myeloma for many years and have expressed their perspectives based on their individual struggles with the disease. In addition, as leaders of their communities of patients with multiple myeloma, they have gained insights into critical issues that patients with multiple myeloma face from diagnosis and throughout their survivorship.
Developments in Multiple Myeloma
All participating patients acknowledged that, although the disease is incurable, the increased availability of innovative treatment options compared with 10 years ago is a major development in multiple myeloma care. For patients, knowing that more treatments are available to fight their disease is a great source of hope. As one patient said, “I think there is much more hope now, because there are many more treatment options available to us. I think we’re feeling more hopeful.”
However, patients also noted that the expansion of treatment options was a great source of uncertainty and anxiety. They cited several challenges and concerns, such as not knowing which of the many treatment options is best for the individual patient ata particular disease stage; what is the best sequence of the available therapies; and which of them will promote the best quality of life for the patient.
The Chart describes the continuum of care and the potential treatment options. In many instances, patients also share in the decision-making process with their physician. Furthermore, considerations of medication efficacy, safety, administration, and quality-of-life issues, are taken into account to make informed choices.
In this context, the Internet was credited as a key resource in providing access to information on the latest advances in therapy and thus addressing patients’ uncertainty and fears. All of this information was thought to have contributed to two things: “the birth of many of the online patient communities,” as well as patient empowerment. According to one patient, “The only acceptable option we have today is to be our own advocates and learn as much as we can about treatment, and side effects, and options that are available to us.”
While acknowledging that patients are not medical experts and must learn to trust their providers, the group concluded that the educated patient must be involved in the decision-making process by considering the benefits and trade-offs of each treatment.
Patient Education and Development of a Treatment Plan
The group agreed that only a fraction of the patients are “empowered,” whereas another group of patients is disenfranchised and underserved. In the words of one patient, “I guess the story of myeloma to me is a story of extremes. In terms of patients, you have people who are very well educated and articulate in the issue, and then you have people at the other end who seem to be disenfranchised, and not connected anywhere, and feeling really alone, like they’re the only person on the planet diagnosed with the disease.”
For both groups of patients, few educational materials are available on the latest advances in the treatment of multiple myeloma that are adequately translated for the patient, which was mentioned as a source of frustration and identified as a major unmet need.
As one patient, who is also an educator, explained, “As a health educator, I’m really frustrated with the literature. We’ve not articulated nor translated the rest of the information for the general mass that’s out there, that is not quick and tethered to the technology. There are a lot of people who feel isolated and who only know what it is that their clinician provides for them.”
Providing such information to the minority of patients was thought to be a particular obstacle that needed to be addressed. The group indicated that patients would benefit greatly from summaries for patients of new data and other medical information from clinical meetings and peer-reviewed publications.
The group indicated that there are several key considerations for developing a treatment plan. These may include whether there are efficacy and safety data to support the treatment decision, whether the treatment is aligned with clinical practice guidelines recommended by national cancer societies, and how it affects the patient’s quality of life.
The group emphasized the importance of multiple myeloma support communities in providing education and overall support for patients.
The group suggested that an integrated framework to help them better understand their treatment plan may limit their fears and uncertainties. The treatment plan must include relevant factors to individualize the plan to the patient, based on the patient’s response to previous steps of therapy, disease aggressiveness, other coexisting diseases and conditions, and genetic factors. Because the first disease remission (improvement in response to treatment) lasts the longest, choosing the right first treatment is particularly important. In addition, recognizing that disease relapse (comeback) is inevitable, the treatment plan should include ways to help patients prepare for these setbacks before the disease returns and progresses.
Such a framework may broadly be organized by the different steps of treatment, including primary treatment (called “induction,” which is the main treatment given to kill multiple myeloma cells), maintenance therapy (treatment given after the primary treatment to maintain the response to treatment and prevent the cancer from returning), and additional treatment given after previous treatments if the cancer returns (relapses) or continues to grow (progress), as described in the Chart.
In this plan, potential options available for each treatment step, such as radiation therapy, targeted therapy, chemotherapy, corticosteroids, and stemcell transplantation, may be included, and specific medication options discussed.
Variation in Care
However, the patients said that a large area of concern for them was the practice variations between care settings that are seen between specialized multiple myeloma care centers and local community cancer centers, or among multiple myeloma specialists. One patient stated, “I really have to differentiate between the experience I have with my local oncologist as opposed to the experience I have with my myeloma specialist.” This variation was attributed to the fact that local community practices see patients with different types of cancer and have an opportunity to treat only a limited number of patients with multiple myeloma. As summarized by one patient, “There are some doctors who just know what to do, and it’s practice. Practice makes perfect. If you only see a few patients, and are confronted with everything, you just don’t have the skills to do it correctly.”
For these reasons, the group indicated that healthcare professionals in the community setting, including oncologists and nurses, may show knowledge gaps in the management of patients with multiple myeloma. Some physicians may struggle with providing personalized treatment, because of the lack of frequent exposure to this disease; for example, they are uncertain about providing an actionable treatment recommendation based on the particular genetic mutation (changes) of a patient. Nurses may also not be as well-informed as they would like about the side effects of common drugs that fight multiple myeloma.
Given the range of potential treatment options available for patients with multiple myeloma, patients also find a wide variation in treatment philosophies even among multiple myeloma specialists, with some specialists adopting a conservative treatment approach and others favoring more aggressive treatments. The participants said that this can be a cause for confusion and uncertainty for them, as expressed by one patient, “Should I have the kitchen sink thrown at me, or should I go for the minimalist? We don’t know, and that’s what’s confusing and frustrating.”
The group explained that the practice variations even extend to how physical exams are conducted in different specialized multiple myeloma centers. As one patient said, “I see 2 myeloma specialists, and depending on which doctor I go to, I get treated differently. In one of the specialist groups, they don’t just ask me, they actually do. They listen to my heart. They listen to my bowel sounds. They measure my height. They don’t just go through the checklist. They check my feet and see if I have edema. The other group, they just ask, ‘Any swelling? How do you feel?’ Even between specialists, your physical exam is different.”
Barriers to Clinical Trial Enrollment
There was much discussion on patient barriers to clinical trial enrollment. Although clinical trial enrollment is a valid treatment option in certain treatment settings and is often a part of the treatment plan,2 patients are reluctant to enroll in them. The group cited several factors for this hesitation, including lack of awareness of a trial, inconvenience of traveling to a trial center, or “feeling like a guinea pig.”
Another reason cited was that “many patients go to community doctors who don’t have those trials available, and there are a whole slew of reasons as to why people don’t participate in trials.”
The patients urged pharmaceutical companies and clinicians to increase awareness about clinical trials. As one patient said, “How clinical trials are designed, executed, and governed needs to really be communicated with everybody, because clinical trial patients immediately assume it’s an experiment.”
It was suggested that the best time to initiate such a conversation is “when people are in remission… those are the times that I think, whether in a passive or aggressive way, that we have an opportunity to get people more acclimated to the idea of participating in clinical trials. When you’re eligible for one, it is a horrible time to introduce a concept that historically has been so negative.”
Other barriers for clinical trial participation were cited as very strict exclusion criteria of the trials, and patients’ unwillingness to risk receiving a potentially inferior treatment.
The group suggested reinforcement to patients that the cost of treatment may be covered by commercial insurance, Medicare, and the company sponsoring the trial.
The Oncology Care Team and Barriers to Care Coordination
The group went on to define the key members of the oncology care team, and who they considered the most critical team member. According to the patients, the care team should, at a minimum, consist of the patient, medical oncologist, nurse/patient navigator, primary care physician, advanced practitioner, nurse, pathologist, pharmacist, and payers or practice managers.
Other healthcare providers that the patients thought may be included in the care team are health plan case managers, societies and support groups, practice administrative staff besides practice managers, social workers, supportive care team, mental health specialists, complementary and alternative medicine (integrative medicine) specialists, nutritionists, and spiritual support.
Given the distinctive roles played by community oncologists and oncology specialists during the disease continuum of multiple myeloma, it was suggested that both types of oncologists be included as separate entities on the care team.
Although all members of the care team play important roles, the group noted that patients’ need for each of these members changes during the disease continuum. The primary “go-to” member of the care team differed, depending on their need, the stage of the disease, geographical location, and how “myeloma educated” they were.
The group concurred that the medical oncologist is considered the primary care team member. One patient said, “Early on, the type of oncologist that I needed is very different. The community‑based oncologist was appropriate. It was more of a relationship, maybe a bedside manner, a connection. Later on, a more research‑based oncologist that is looking further.”
From a patient perspective, the primary care physician also plays an important role in their care. The primary care physician is the most critical person at the beginning of the cancer journey, because of his or her involvement in the diagnosis. Moreover, as patients with multiple myeloma live longer, the care by the primary care physician continues to be vital to the overall health of the patient in providing supportive care and treating other comorbid conditions.
However, the group pointed out that a lack of coordination often occurred among the different stakeholders in the care team, which led to fragmented care. One patient elaborated on the lack of coordination, “Even information flowing from the oncologist to the internist. If I have somebody on two systems, it really is up to me to provide that bridge of information between those two different organizations, as well.” The group stressed that since all the members of the multiple myeloma care team interact with the patient at different stages of the disease, care coordination is critical to providing the best possible patient care. They concurred that having one person, such as a healthcare assistant or a nurse navigator, coordinating care would be of great benefit for providing the needed continuity of care.
Supportive Care and Survivorship Care Programs
The group noted that many cancer care teams lacked a dedicated supportive care component, which plays a critical role in side effect management and prevention. One patient stressed, “Supportive care, being able to understand the importance of taking care of yourself, and never ever taking a cold or anything like that for granted, to always be prepared, that’s critical. That saved my life twice.” Elaborating on the importance of supportive care, another patient stated, “They go through, and they’ll train you. If you have a temperature, don’t wait until your temperature is 102. When it’s 100.5, this is what you do…”
The group also lamented the lack of survivorship care programs to help patients maintain continuity of care and address survivorship issues with life course implications. Expanding on the lack of discussion on survivorship issues relevant to many young patients with multiple myeloma who undergo stem-cell transplant, one patient stated, “I have a couple of young guys that I was talking to that nobody told them, ‘Hey, what you need to do is go get some [semen] frozen, if you plan on having any kids,’ or women, ‘You need to get a couple of eggs removed if you really want to have kids,’ if you’re a 20‑, 30‑, or even 40‑year‑old woman.”
The group envisioned a survivorship care plan to be a complete document or “blueprint” that contained information on patient diagnosis, treating physicians, treatment history, including pathology, treatment plan, reported side effects and their management, and other screenings, which could be assessed electronically and by all stakeholders.
The group emphasized the importance of multiple myeloma support communities in providing education and overall support for patients. One of the patients explained, “I also felt a big part were my support communities, whether it was my support group, or the online support communities. They were the people that really educated me, and because I was educated, I could participate more in my care.”
They also shared that there were practice variations in terms of recognizing the immense benefit patients derived from this resource. Although the academic oncology teams offered patients avenues to pursue this support resource, community practices did not.
Education and Management of Side Effects
One session was dedicated to discussion of treatment-related side effects that patients with multiple myeloma experience, and the appropriate approach to management of these side effects. The group revealed that some patients may be reluctant to report side effects, because of the fear that an active drug may need to be stopped, which may compromise their treatment outcome.
The patients conveyed that a key message to communicate to the patient is that many side effects can be managed, and that the emergence of side effects during cancer treatment does not mean the drug must be discontinued.
According to the patients, nurses should be proactive in having these discussions with patients. Moreover, patients who are using oral therapy may not recognize and readily communicate side effects to the care team, because of the reduced interaction with the practice when taking oral medications.
The group conveyed that it is often difficult for patients to decide whether the side effects are related to the treatment or to the disease. They reinforced the importance of having open communications with the myeloma care team to distinguish between the sources of side effects.
The group also suggested making available informative materials to educate patients on drug specific side effects, and the frequency with which they occur. It was suggested to list just the major side effects, instead of a long list of all side effects, preferably, as an acronym that can be easily remembered.
In addition, the group expressed that baseline side effects for each patient are seldom documented. As one patient explained, “Getting a baseline is very important. For example, if you know what your baseline pain is, then you tell your physician when it’s worse than usual. Subsequent testing should be done in relation to that baseline.”
With the understanding that nearly all patients with multiple myeloma experience medicationrelated side effects at some point during treatment, the participants identified the following 4 categories of multiple myeloma treatment-related side effects that they struggle with the most:
- Cognitive side effects (“chemo brain”)
- Gastrointestinal side effects
- Fatigue
- Neuropathy, which involves pain, tingling, burning sensations, and numbness in the hands and feet
Pain was mentioned as an ever-present side effect that patients with multiple myeloma contend with but is not adequately treated. It was noted that different patients have different pain thresholds, and that it can be difficult to assess pain using self reporting methods, which highlights the need for routine pain assessments for all patients with multiple myeloma, using standard assessment methods.
The group discussed other concerns, including practice variations and unmet needs related to side effect management. Foremost, the patients reported that in contrast to larger academic settings, their community practices provided minimal side effect education and trivialized the side effects they were experiencing. One patient said, “Teach them [patients] about side effects, how to dose-reduce when necessary, not follow just the standard protocol, but ask questions. Not one nurse has ever asked me, ‘How’s your neuropathy?’ or given me a test to see if I could hold things. Unless I bring it up, I’m not asked.”
Echoing these sentiments, another patient stated, “If you look okay, and your ‘numbers’ are doing okay, they don’t even want to entertain some of the side effects that you may be experiencing. Everything that I was saying was being dismissed, because my blood counts looked good, my platelets were okay.”
One patient went on to elaborate about how her complaints about fatigue were not addressed. “No one bothered to tell me that maybe if I took my medication at night and used it as a sleeping pill, I might have less fatigue. I learned that in the support group. I didn’t join a support group until maybe 4 or 5 months after my diagnosis.”
Moreover, the group mentioned that some physicians may prescribe antiemetic (antivomiting and nausea drugs) and antiviral agents for prevention of side effects, but others do not.
It was also expressed that each patient has a different outlook on the risks and benefits of a treatment, and the care team must therefore make an effort to determine the patient’s viewpoint before designing the treatment plan. One patient mentioned a negative experience with steroids, which formed the basis for her unwillingness to accept the trade-offs associated with their use.
Oral Therapy and Medication Adherence
Oral therapy is an important trend in oncology, with 4 new-generation oral drugs currently approved by the FDA for patients with multiple myeloma.3 The patients agreed that oral therapy is more convenient than injections and infusions, has no injection or intravenous (IV)-related complications, may be easier and faster to take, saves time by requiring fewer visits to the healthcare team, allows more independence, and provides travel convenience.
Out-of-pocket expense was mentioned as a major issue for patients, with some attempting to "stretch out" the time between prescription fills by under-dosing or missing doses.
The patients noted that oral treatment can be liberating for patients, can restore their dignity, and change their perception of their sickness. As expressed by patients, “Doing the oral, besides what it gave me back in terms of time, it gave me back something in terms of dignity, too, so I preferred it.”
Another patient said, “I thought I was a sicker person when I had to have infusions than when I took a pill.”
However, a shift in burden with oral therapy is well-recognized, with the patient assuming the burden of medication adherence.4 Unfortunately, many patients are not adhering to their treatment and often fail to fill their prescription, or take the drug as prescribed, or they discontinue treatment earlier than prescribed.4 This lack of adherence to prescribed therapy is known to be a major contributor to suboptimal drug efficacy and poor patient outcomes.5
The group cited several patient barriers to medication adherence. Foremost, the group stated that patients perceive oral drugs as being more expensive. Out-of-pocket expense was mentioned as a major issue for patients, with some attempting to “stretch out” the time between prescription fills by underdosing or missing doses.
The group stressed that it was important to educate patients about drug-drug interactions and drug-food interactions, particularly regarding oral drugs.
One patient explained that many patients may not have the same coverage for oral cancer drugs as for IV drugs, which are paid for by different types of insurance in relevant states, and for those who are covered for oral drugs, the out-of-pocket costs may still be higher than what some patients can afford to pay for drugs covered under their pharmacy benefit.
Although patient assistance programs offered by drug manufacturers and nonprofit foundations are available for such patients, it was noted that many practices and patients are unaware of the existence of such financial resources, and consequently do not avail of these opportunities.
Another financial barrier cited was that many practices consider that it is time-consuming and resource-intensive for them to help patients determine their financial assistance options, so these options are not offered to patients. For these same reasons, and because they want to initiate treatment quickly, the practices opt to initiate an IV treatment instead of oral therapy.
The group also cited other practical and patient-related barriers to medication adherence; side effects, cognitive issues such as forgetting to take oral medication, and delays in obtaining insurance approvals can interrupt therapy. It was noted that home delivery of drugs can be a challenge, because a signature is often required. Several patients liked the idea of in-office dispensing, especially if a nurse or a pharmacist can provide on-site education.
Sometimes, patients find coordinating refills with the blood test to be challenging, and may result in suboptimal adherence. It was noted that dosing schedules may also impact adherence, and once-daily dosing may be easier to adhere to compared with once-weekly dosing, because it is easier to remember.
The group stressed that it was important to educate patients about drug–drug interactions and drug–food interactions, particularly regarding oral drugs. They also mentioned the need for drug-specific checklists to help them monitor their side effects and stay on track with their oral medications. This was thought to be particularly useful to patients using oral therapy, because they have fewer interactions with the practice.
Tools and Resources
The group stated that patients had little knowledge of drug manufacturer–provided tools and resources for multiple myeloma, such as discussion guides that are available online, because they rarely visit drug companies’ websites. The group advised that drug manufacturers are not part of the patient mindset; an exception is for commercially insured patients who require financial assistance or when drug company speakers host a program to showcase drug services. According to the patients, some patients have the impression that “big pharma is bad.”
Participating patients communicated a need for comprehensive electronic tools and resources to help them manage their treatment and to communicate with their treatment team. The Myeloma Manager software (from the International Myeloma Foundation)6 was cited as a “go-to” resource that allowed patients to keep track of their laboratory test results, drugs, side effects, appointments, and dosing schedules, which may be more beneficial if it can be developed into a mobile application.
Other helpful resources cited by the group included Smart Patients (formerly ACOR), which is offered by the International Myeloma Foundation. It is an online community where patients and caregivers learn from each other; another tool is MyChart,7,8 which provides patients access to personal medical information and clinic services.
The group expressed their interest in ASCO’s CancerLinQ,9 a database that collects treatment information from actual patients with cancer that helps oncologists make treatment decisions for their patients.
In particular, the group was receptive to tools to help track medication side effects and send medication reminders to patients. One patient explained, “The ability to have, or a means to have to record your side effects over a period of time, that actually change, and be able to present that in a meaningful way to your doctor when you are visiting him on a monthly basis for your lab results or something like that, I think would be very helpful as well.”
The group stated that the tool must be flexible in terms of frequency of contact (weekly, monthly), and that the mode of communication must be tailored to patient needs and desires, with periodic follow-up to see if their needs or desires have changed.
It was also cautioned that disease severity may impact how patients embrace tools and resources, and that patients would be more likely to embrace a resource if they have an immediate need. Also, because patients are typically using multiple drugs, an integrated application, developed in partnership with other manufacturers and/or third parties, may be warranted.
The group expressed the need for resources that help all providers, including oncologists, primary care physicians, other specialists, pharmacists, and midlevel providers, coordinate care effectively. The group recommended that drug manufacturers should incorporate an educational component when introducing tools and resources. They noted that they would likely not be used if resources are “dumped” on patients without appropriate support; most patients cannot figure them out on their own, and become frustrated.
Conclusions
This article highlights the key challenges and unmet needs of patients with multiple myeloma, and their unique recommendations to remedy these shortcomings. Recognizing the knowledge gaps between academic centers and community providers, the group recommended the development of peer-to-peer educational programs that target the community oncology provider and nurses. To address deficits in side effect management and education, measures such as regular side effect assessments, educating patients on treatment specific side effects and side effect management strategies, and appropriate education level, treatment-specific checklists were recommended.
Because multiple myeloma care is often fragmented, participating patients indicated that a single point of contact to help guide them through the journey would be most beneficial. Toward that end of the discussion, institution of navigator initiatives in conjunction with the development of educational tools and resources directed specifically for navigators was recommended.
To truly improve overall patient outcomes, concerted efforts across the healthcare delivery system are needed to implement these recommendations and correct the identified deficits in the care of patients with multiple myeloma.
References
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2. National Comprehensive Cancer Network. NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines): multiple myeloma. Version 1. 2016. September 4, 2015. www.nccn.org/professionals/physician_gls/pdf/myeloma.pdf. Accessed September 10, 2015.
3. CenterWatch. FDA approved drugs for oncology. www.centerwatch.com/drug-information/fda-approved-drugs/therapeutic-area/12/oncology. Accessed August 27, 2015.
4. Accordino MK, Hershman DL. Disparities and challenges in adherence to oral antineoplastic agents. Am Soc Clin Oncol Educ Book. 2013:271-276.
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6. International Myeloma Foundation. Myeloma Manager Personal Care Assistant Version 4.0.2. http://myeloma.org/ArticlePage.action?tabId=0&menuId=0&articleId=2316&aTab=-1&tBack=&tDisplayBack=true. Accessed August 27, 2015.
7. Smart Patients. www.smartpatients.com/. Accessed August 27, 2015.
8. Epic Systems Corporation. University of Arkansas for Medical Sciences MyChart. www.uamshealth.com/MyChart. Accessed August 27, 2015.
9. ASCO Institute for Quality. CancerLinQ. www.instituteforquality.org/cancerlinq. Accessed August 27, 2015.
