Defining Palliative Care

Palliative care is a relatively new medical subspecialty, having arisen from a necessity for improved ways to manage patients with chronic diseases that may be “life-limiting.” Palliative care is similar in some ways to hospice care, yet it has enough distinct features to emerge as a sovereign entity. In fact, palliative care requires a paradigm shift away from the “hospice mindset,” in which death is a forthcoming and soon expected outcome (or “death is expected within 6 months”). This is not the case in palliative care, in which patients may seek similarly aggressive treatment of their symptoms, including pain, at any time during the disease course, for which death may not occur for many years. Here, successful management warrants more frequent patient reassessments and significant pharmacovigilance.

Despite similarity in the philosophy of care and services rendered, in the US health care system, palliative care and hospice services have 2 different payment systems and locations of services. Hospice is the only Medicare benefit that includes pharmaceuticals, medical equipment, 24-hours-a-day/7-days-a-week access to care, and support for loved ones following a death. Most hospice care is delivered at home, but it is also available to people in homelike hospice residences, nursing homes, assisted living facilities, veterans’ facilities, hospitals, and prisons. Palliative care services are often provided initially as part of an acute care hospital stay and may be organized around an interdisciplinary consultation service with or without an acute inpatient palliative care ward. Palliative care may also be provided in the dying person’s home as a “bridge” program between traditional home care services and hospice care, or it can be provided in long-term care facilities. In contrast, more than 80% of hospice care in the United States is provided in a patient’s home, with the remainder provided to patients residing in long-term care facilities or in free-standing hospice residential facilities. Hospice should be considered if the extent of disease and rate of decline indicate that the patient is likely to die within 6 months.

Why Is Palliative Care Appropriate for Neuromuscular Disease

Severely progressive neuromuscular disease (NMDs), including disorders like amyotrophic lateral sclerosis (ALS), spinal muscular atrophy, and Duchenne muscular dystrophy (DMD), pose unusual medical, ethical, and humanitarian considerations for the patient, the family, and the health care staff that are involved in their care. These patients often have high symptom burdens, including chronic pain, muscle cramping, fatigue, dyspnea, and constipation, among others. Moreover, these patients and their families must make challenging decisions regarding the potential use of life-prolonging therapies, such as mechanical ventilation. The significant portion of the impact may be felt by family caregivers who are simultaneously struggling with arduous physical, emotional, and financial stressors associated with the NMD. Although these diseases are considered to be fatal, unlike most cancers or other incurable illnesses, patients with advanced NMD may live for many years before succumbing. Despite our current research developments and the most aggressive treatment available, patients with these diseases will inexorably continue to debilitate as the disease progresses. However, the lack of available curative treatments does not mean that physicians should take a nihilistic attitude, because palliative care is an appropriate treatment of NMDs. Optimal care should maximize function and quality of life for patients with an NMD. Expert multidisciplinary care may improve both quality and length of life of patients with progressive NMD. Early involvement of palliative care specialists as part of the multidisciplinary team is theorized to likely improve the quality of life for patients with an NMD and their families, although this remains poorly studied.

Depending on the subtype, an NMD may encompass many conditions, each with differing presentations, symptoms, and complexities. People with an advanced NMD often have insufficient care during the last year or so of life. This infers that they may not have had a “good death” or did not die where they would have wished. People who are housebound cannot, by definition, attend clinic but they are seldom assessed at home either. Pain and other symptoms are often not adequately assessed or treated. Krivickas and colleagues studied home care in 98 patients with ALS. In that sample, 24 were receiving nonhospice home care, 9 were receiving hospice home care, and 7 were receiving both hospice and nonhospice home care. Remarkably, 58 patients received no outside help. Patients receiving hospice were older than those receiving nonhospice home care (68.9 vs 57.7 years, P <.05). Patients with home care assistance had a mean ALS Functional Rating score of 13, and those without home care assistance had a mean score of 26 ( P <.0001). Patients receiving nonhospice home care assistance received a median of 16 hours of care per week, whereas those with hospice received 5.5 hours of care per week ( P = .05). Patients on Medicaid received more hours of home care than those with any other insurance (median 61 vs 3.4 h/wk with Medicare and 5 h/wk with commercial insurance, P = .008). Not surprisingly, primary caregivers spent a median of 11 h/d caring for patients despite having home care assistance, and 42 (48%) of these primary caregivers felt physically ill and psychologically “burned out.” This study clearly identified that home care received by patients with advanced ALS is inadequate and too late to relieve the burden placed on family caregivers.

How Palliative Care Can Help

Unlike hospice, palliative care is applicable early in the course of illness, in conjunction with other therapies that may indeed prolong life. The key to effective palliative care is communication. It is essential to ensure that the patient with NMD is able to understand as much as he or she wants to know about the disease and to be aware of what may happen as his or her disease progresses. This enables the patient to feel empowered to make informed choices regarding future care. This must be a 2-way process, with the person feeling that his or her views and preferences have been heard and understood by the clinician.

The basic tenets of palliative care include providing relief from pain, shortness of breath, nausea, and other distressing symptoms. This is done in the context of affirming life yet acknowledging that dying is a normal process. The intent of palliative care is to neither hasten nor postpone death but instead to offer a support system to help patients live as fully as possible through the integration of medical, biopsychosocial, and spiritual aspects of patient care. This involves a multidisciplinary team to provide care for the patient. In this setting, pain is not simply treated as a singular entity but is approached as the sum of 4 components: physical noxious stimuli, affect or emotional discomfort, interpersonal conflicts, and the psychological aspects of accepting one’s own death. These 4 components may individually or in combination affect patients’ perception of their total pain. Failure on the physicians’ part to fully understand and address each of these 4 components may result in less-than-optimal pain management at this stage of illness.

What Are the Challenges That Need to Be Addressed?

Given the advancements in health care, people with even severe NMDs like DMD are living longer now, often well in to adulthood, yet the need for care increases with time. Young adults with DMD often report high life satisfaction. Ideally, access and use of services increase in tandem with disease severity. However, this is not necessarily the case. Parker and colleagues found discrepancies in service use in their study of 25 adult patients with DMD. Medical care and other services may lag behind increases in severity for a variety of reasons. For example, the family may not be aware of existing services or they may not understand the medical implications of a young patient’s increased disease severity. Availability of services, distance to care, family resources, and transportation may all serve as barriers to care. Ideally, the need for services should predict their use; family resources, for example, should not factor into this. The need for services should equate roughly to disease severity, whereas limited family resources and lack of knowledge of available resources reflect inequitable access to these services. Yet service use models typically explain no more than 25% of the variance, whether predicting the use of individual services or the total number of services. Prior studies have shown considerable variance in other disabled patient populations in terms of use of palliative care services. Family resources should not serve as barriers to palliative care services because insurance should cover most of these costs. The ability to access palliative care may be more dependent on patient and caregiver knowledge of what is available. Patients with NMD may not be aware of all services that are available that could potentially help them through the advanced stages of the disease process. Given that service use seems to be based more on resources and/or greater knowledge than on need is inequitable. Clearly, this dictates policy changes that include strategies to increase awareness of available services for all patients with NMD.

There are many other unanswered questions, including what aspects of care matter most for patients with an NMD who are receiving palliative services and what are the most effective strategies to alleviate suffering in patients with an NMD. Research has yet to identify the best practice standards in pain and symptom management for patients with an NMD with chronic pain who are in a palliative medicine program. The available literature indicates that patients with an advanced NMD have frequent, unplanned hospital admissions, which increase as the disease progresses. Many factors account for this, including the long and unpredictable duration of advanced NMDs, the wide range of symptoms, the complex multidisciplinary care issues in DMD and ALS, neuropsychiatric problems (eg, behavioral and cognitive changes) that may negatively affect communication and care planning, and the common use of assisted ventilation aids in patients with NMD. It is worth considering that many patients with an NMD die of comorbid problems, instead of the disease itself. That is, they die with, but not of, their neuromuscular condition. This highlights the need, in the more rapidly advancing diseases, to obtain palliative care involved early in the disease progression.

How can end-of-life care be improved for people with an NMD? Communication is critical. Listen to what the person has to say about his or her own problems, strengths, and circumstances. The clinician must adequately assess the current problems and look for viable solutions. The multidisciplinary team should manage symptoms as part of holistic care. This improves an individual’s quality of life and can ease burden on the family, both before and after death.

Consider advance care planning, including advance decisions to refuse treatment and lasting power of attorney, and sensitively discuss with the person and caregivers their future care options. Clinicians can initiate open discussion around end-of-life care (ie, when talking about potentially life-prolonging treatment). However, the clinician must recognize that the person could die soon, thus justifying a more palliative approach. If a clinician is not used to thinking in these terms, he or she should ask himself or herself, “Considering the extent of disease, and rate of decline, would you be surprised if this person died in the next year?” If the answer is no, this would not be surprising and is expected; then consider introducing advance care planning and initiate discussions about managing the time the patient has left to them. For most clinicians, including most physicians, this involves a major paradigm shift in thinking. Physicians are often not very skilled at detecting subtle signs that would indicate that a palliative approach or even end-of-life care might be more appropriate and have not received training in how to approach these often very sensitive, uncomfortable discussions.

Possible triggers that indicate it is time to consider a palliative care approach for a patient with advanced NMD include (1) a marked decline in pulmonary function, particularly forced vital capacity and peak cough flow; (2) marked weight loss; (3) recurrent infections (typically pulmonary or urinary); (4) inability to heal lesions, including pressure ulcers; (5) swallowing problems; and (6) cognitive decline.

The savvy clinician will prepare the patient with an NMD well in advance of late-stage disease and facilitate end-of-life discussions by posing questions such as, “If you were to get a serious infection, where would you like to be treated?” “How aggressively do you want to be treated?” “Do you want all available medical interventions, including those that may be life prolonging?” Done properly, these questions would be asked when the patient is stable and doing well. The patient and family may need coaching, education, and time to consider the differing options and care pathways. Avoiding these conversations, whether early or late in the disease process, does the patient a disservice and may result in the loss of autonomy when, in the setting of an acute problem such as aspiration or sepsis, the person ends up being hospitalized and is given aggressive life-prolonging treatment, regardless of preference.

The Next Step: Integration of Palliative Care in NMD Management

We hope that we have presented a clear and logical argument that allows the reader to recognize need for palliative care in the management of progressive NMD. It is a better way to manage the complexity of care in this patient population. Although there is a great need for prospective research in this area, a recent study published in The New England Journal of Medicine showed that patients with lung cancer who received early palliative care experienced less depression and increased quality of life and survived almost 3 months longer than those receiving standard oncologic care. We can do the same for palliative care in NMDs.

On the other hand, barriers still exist. Given the qualitative nature of these modalities of treatment, palliative care research has been hampered by methodologic challenges. Much of this relates to variations in clinical trial design and care strategies, attrition, and missing data caused by patient death. The variation in palliative interventions and the wide variations in focus and extent of services make comparisons across trials difficult. Despite these limitations, accumulating evidence confirms that palliative care interventions improve patients’ quality of life, satisfaction with care, and end-of-life outcomes.

Patients with incurable NMDs experience a considerable burden of physical and psychological distress, which often negatively impacts their quality of life. Palliative care clinicians are ideally suited to alleviate this suffering and to help patients with an NMD make informed decisions to direct their health care toward maximizing quality of life and improving satisfaction with care, family caregiver outcomes, health service use, and quality of end-of-life care.