2 The New and Changing Landscape of Children Receiving Pediatric Palliative Care in the US

An Evidence Base Emerges

Bryan Sisk, Kimberley Widger, and Chris Feudtner

PPC did not become a widely recognized clinical specialty until the 21st century, but research into the experiences of children with serious illnesses dates back to the 1950s.¹ In the 1950s and 1960s, pediatricians and psychologists began publishing accounts of the psychological experiences of children with “terminal” illnesses (most commonly cancer) and their mothers. For example, Bozeman, Sutherland, and Orbach published one of the earliest studies of this kind in 1955, in which they described the blunted responses of children with cancer.

Others similarly described the experiences of these children and their parents over the ensuing decade.^3,4,5

By the 1970s, this psychological research shifted toward more rigorous studies, exemplified by the work of Spinetta and Bluebond-Langner. Spinetta, a psychologist, explored children’s awareness of their impending death and their adaptation to this knowledge. He focused on children’s anxiety, highlighting their perceptions that nurses and doctors distanced themselves as their disease progressed. Spinetta’s work contributed to the development of psychosocial standards for the International Society for Pediatric Oncology (SIOP).^6,7,8,9,10 Bluebond-Langner, an anthropologist, performed ethnographic observations in a pediatric cancer ward, culminating in her landmark book, The Private Worlds of Dying Children.¹¹ Bluebond-Langner’s work explored the role of relational and social networks in the way families processed and worked through their children’s fatal cancer diagnoses. Notably, her work further explored the concept of mutual pretense that was originally proposed by Glaser and Strauss.¹²

Infants fared even worse than young children. In the mid-1980s, Anand and colleagues published a series of studies demonstrating that infants have robust hormonal and biochemical stress responses after surgery, thus indicating that infants could feel pain in a meaningful way.^16,17,18 Prior to this work, many physicians commonly believed that infants were unable to experience pain. Some thought that a high pain threshold was evolutionarily adaptive to protect infants from the pain of birth.¹⁹ Additionally, pain prior to that time was viewed as “a subjective phenomenon.¹⁹ Given that infants had limited cognitive capacity, many believed they were unable to experience pain in the same way as adults. Instead, most clinicians believed the infant’s manifestations of “pain” to be merely physiological reflexes, similar to tapping the knee with a reflex hammer and watching the leg kick. Even if infants did experience painful stimuli, some argued they were probably unable to remember it after the event. (Surprisingly, this lack of a memory for painful events was reassuring to some clinicians.) As a result of these beliefs about pain, infants routinely underwent major surgeries, even thoracotomies, with paralytics and sedatives but no analgesia.²⁰

By 2000, a growing body of literature had begun to explore the physical, psychological, and social experiences of children with life-threatening illnesses. Yet few studies explored actual clinical practices. Wolfe and colleagues published a pair of landmark studies in 2000 that highlighted inadequacies in communication and symptom management for children with cancer at the end of life.^21,22 Soon after, Hilden and colleagues found that pediatric oncologists endorsed “a strikingly high reliance on trial and error in learning to care for dying children.”²³ In 2002, Contro and colleagues studied the experiences of bereaved parents, finding evidence of “confusing, inadequate, or uncaring communications regarding treatment or prognosis; preventable oversights in procedures or policies, failure to include or meet the needs of siblings . . . and inconsistent bereavement follow-up.”²⁴ Concurrently, children began to be involved and included in their own care, leading to open communication and engagement in decision-making, especially advanced directives.

From this foundation in the early 2000s, PPC research has expanded rapidly into a growing evidence base. The initial focus on pediatric cancer in PPC research has broadened to include many complex diseases, thus contributing to a shift in PPC from a focus on end of life to a focus on quality of life (QOL), no matter how long or short it might be.

In the following sections, we summarize the current state of research in PPC, and we end by identifying future directions for investigation and development of interventions.

Patient Experience

While each child receiving PPC services is unique, there are discernable patterns of conditions and diseases, mortality and prognosis, and concerns about symptoms and QOL.

Not all children who die receive PPC services, but national profiles of children who die provide the strongest epidemiological evidence to characterize the conditions and diseases of children receiving PPC. Examining mortality data in the United States between 1999 and 2006, one notices immediately that infant deaths are due mostly to specific perinatal conditions, such as premature birth and congenital syndromes or chromosomal disorders, whereas older children are most likely to die from external causes, such as traumatic injury (Figure 2.1).

We can examine the number of children who die from complex chronic conditions (CCCs) as one statistic that can inform the field of PPC. CCCs are defined as

• Any medical condition that can reasonably be expected to last at least 12 months unless death intervenes,

• Any medical conditions that involves either several organ systems or one organ system severely enough to require specialty pediatric care, with a likelihood of some period in a tertiary care center.

For childhood deaths in the United States, congenital and chromosomal abnormalities were associated with 20.3% of infant and 4.4% of child deaths, while diseases of the nervous system were attributed to 1.3% of infant and 4.8% of child deaths. Deaths from cancer, which is perhaps the condition most strongly associated with the imagery of palliative care in the mind of the public and much of the healthcare professions, accounted for only 0.5% of infant deaths and 8.9% deaths past infancy.

A few studies of patients enrolled in PPC programs support the evidence of diverse conditions in this population. In a study of 13 PPC programs in Canada, patients had as their primary diagnosis nervous system disorders (26.8%), cancer (15.8%), or perinatal-onset conditions or congenital anomalies (36%).^25,26 At a large children’s hospital in the United States, 41.8% of patients who were referred for specialist PPC services had a primary diagnosis of cancer and 38% had congenital or genetic disorders.²⁷ Moreover, another study evaluated the complexity of patients admitted to a large tertiary children’s hospital, finding that nearly half of all children admitted to the hospital suffered from CCCs, and 82% of children were diagnosed with rare diseases.²⁸

Mortality and Prognosis

Over the past century, child mortality has been declining for both children suffering from trauma and from CCCs. Examining US mortality data from 1979 to 2006, the rates of death attributed to CCCs as well as injury, sudden infant death syndrome, and other causes declined substantially, with the corresponding numbers of deaths also declining, but less so since 2000.

One published study focused on the interval between 1979 and 1997, when the annual death rate due to non-cancer and cancer-related CCCs declined for almost every age group (from a 7.1% decline for infants with cancer to a 49.9% decline for 1- to 4-year-olds dying from non-cancer CCCs). The one exception to this decline was the mortality attributed to non-cancer CCCs among 20- to 24-year-olds, for whom the rate of death actually increased by 11.6% over this 18-year interval, which may be due to the longer life spans of children with CCCs dying at later ages, such as those with cystic fibrosis and Duchesne’s muscular dystrophy.²⁹ For this reason, studies of pediatric deaths and palliative care need to include the experiences of young adults, perhaps into their 20s or 30s, who are living with these congenital or childhood-onset conditions (Figures 2.2 and 2.3).

Patient Symptoms and Quality of Life

The management of physical and psychological symptoms is of utmost importance in PPC. Most research in this area has been conducted with bereaved parents of children with cancer, beginning with Wolfe and colleagues who found a high degree of suffering and lack of treatments offered.²¹ Subsequent studies have replicated these findings in multiple countries. In one such study, 96 bereaved Australian parents of children with cancer reported that, in the last month of life, 84% of children suffered from at least one physical symptom (pain, fatigue, poor appetite, constipation, dyspnea, nausea/vomiting, diarrhea, and seizures), with pain, fatigue, and poor appetite as the most frequent complaints.³⁰ Nearly half (43%) of children in this study suffered from three or more of these symptoms. Parents also reported that 42% of children had been “more than a little sad,” 38% experienced “little or no fun,” and 21% were “often afraid.”

In a Swedish study of 449 bereaved parents of children with cancer, physical fatigue was the most frequently reported symptom (86%) that had a moderate or high impact on the child’s well-being. Reduced mobility (76%), pain (73%), and decreased appetite (71%) were also major concerns.³¹ Parents were also more likely to report anxiety in children older than 9 years of age than in younger children. Children also suffered from difficulties in swallowing, depression, reduced mobility, impaired speech, swelling, disturbed sleep due to anxiety, and urinary problems. Problems with breathing during the last day of life, and with loss of motor function in the last week of life, were also reported in a study of 28 bereaved parents whose children had been enrolled in a pediatric hospice program in St. Louis.³² A study of 65 parents (of 52 children) reported similar symptoms, which did not differ according to patient gender, disease, or location of death.³³ Bereaved parents of 48 children who died of cancer in another study reported that their children suffered from anxiety, which was not treated effectively.³⁴

More recently, additional studies of children with cancer have characterized symptoms during therapy and near the end of life. A study in 2010, for example, found that 96% of parents reported that their child with cancer reported fatigue in the last month of life, and 50% reported significant suffering from this fatigue. Of these children, only 13% received any fatigue-related therapy, and only 3% of children received effective treatment for fatigue.³⁵ Outcomes from the PediQUEST trial identified the following prevalence of symptoms reported primarily by children (or their parent when the child was too young or unable) being treated for advanced cancer: pain (48%), fatigue (46%), drowsiness (39%), and irritability (37%). Reports of pain within the final 12 weeks of life increased to 62%.³⁶ A subsequent analysis of data from the PediQUEST trial found that children from low-income families were also more likely to report pain (64% vs. 42%). Other measures of psychological distress and QOL did not reach statistical significance, but point estimates of all measures suggested worse outcomes for children from low-income families.^37,38,39,40 Much remains to be done in improving the treatment of symptoms in this population.

Researchers have focused less on QOL in the face of serious illness. The PediQUEST study demonstrated strong associations between distressing symptoms and QOL, ⁴¹ but few other studies have evaluated QOL in PPC. A key barrier is the lack of instruments specifically developed for use in this population and the evidence that existing generalized instruments may not be suitable for this population of children.^42,43 Currently available instruments tend to focus primarily on physical functioning as a key component of QOL, yet children with cancer have highlighted the importance of social functioning throughout the illness experience.⁴⁴ Health professionals have suggested additional domains of QOL, such as living in the present moment and experiencing pleasure, which may be important in the assessment of QOL particularly in advanced illness.⁴⁵

Family Experience

When a child has a CCC, the entire family is affected. Furthermore, the ability of the family to adapt to their child’s condition can affect (negatively or positively) the experience of the child. For example, the structure and membership of a family, as well as patterns of family organization and function, can influence the care of children with CCCs.⁴⁶ Family structure shapes how tasks are divided and roles are assigned in caring for the child. One parent might assume primary physical care for the patient, while the other parent or other family members manage care of the household and siblings. Structure also dictates how families respond to and regulate their intense emotional reactions to a child’s illness, which influence daily symptom management, soothing the ill child, and making critical end-of-life decisions. Family structure critically affects how care is performed and is in turn affected by the family’s finances, their social and cultural backgrounds or community, and their religious and spiritual beliefs and practices. Additionally, several aspects of care can affect how parents cope with their child’s illness and/or death. To varying degrees, the healthcare literature provides useful data and knowledge about each of these topics.

Family Structure

Parents of children with serious illness report negative effects in many aspects of their lives including family life, finances, social life, and physical health.^47,48 Social support may serve as a protective factor for some families.^49,50

Families vary in how they function, and some families may be characterized by high conflict levels, poor cohesion, poor communication, and a tendency to reject healthcare.^51,52 Even within a family, parents may differ in how they experience and cope with a child’s illness.^53,54 Such families may be considered “difficult” families by clinical staff, especially if there is a history of limited family support, inconsistent caregivers, and multiple living environments.⁵⁵

Some research initially suggested that two-parent married households of children receiving palliative care who died were at higher risk for divorce⁵⁶, but later research found that these parents were not necessarily at greater risk for divorce.⁵⁷ In fact, some may experience positive benefits, such as feeling closer as a family and supporting each other, which offset the stresses.⁵⁸ One study of families of children diagnosed with cancer found that, while many families went through a period of difficulty and stress in terms of marital, parent–child, and sibling conflict, the majority of families were able to weather the storm and maintain good family relationships.⁵⁹ Caregiver- and family-focused interventions may help families cope with the stress of caring for a child with a life-threatening illness.^60,61,62

When parents are divorced, remarried, or have never been married, complex relationships (spouses, ex-spouses, step-children, step-siblings, other extended family) and a history of conflict may undermine communication and support efforts by clinicians,⁶³ especially when the nonresidential parent has a different opinion about the child’s care than that of the custodial parent.⁶⁴ Having multiple or discordant decision-makers within the family can contribute to conflicts both within the family and with clinical staff.⁶⁵

Single-parent households have been less frequently studied,⁶⁶ but some evidence suggests that single mothers of children with serious illness may have higher stress-related symptoms, their children might have higher hospitalization rates, the parent may engage in fewer health-maintaining behaviors, and the parent may feel more isolated.^67,68 Other studies on children with disabilities and children being treated with cancer suggest that single parents had greater difficulty with finances⁶⁹ but also were more flexible in adapting to new family roles and arrangements.⁷⁰ Children in the foster care system with serious illness may have unique needs because of a history of prior trauma and abuse.⁷¹ These prior traumas could impact the child’s coping responses and exacerbate psychological and social insults from their disease process. In these scenarios, psychologists and social workers provide critical skills and guidance to PPC teams.

Social and cultural characteristics of families can also affect family functioning. Numerous studies indicate that racial and ethnic minorities may have difficulties with poverty, less trust and poorer communication with the medical profession, and different belief systems and family structures.^{72,73,74,75,76} Religion and spirituality are very important to many families, but the impact of these beliefs is not uniform or prescriptive.^77,78,79 Some families appreciate spiritual and religious support from clinicians but prefer that such support be initiated by the family based on their needs.^80,81 Discussing religion and spirituality with families is considered an advanced communication skill that not all clinicians are comfortable with.^82,83

The Work of Care

Although most children with serious illness die in the hospital, some children with serious illness are cared for and die at home.^84,85 Although this allows families to spend more time together at home, it also requires much work on their part.⁸⁶ This work of care consists of the effort, labor, and various tasks in which parents and other family members engage in to care for the child with a CCC and for the family system. Daily tasks include administering medications, feeding and bathing, providing emotional support, and monitoring any changes in the child’s health.^{87,88,89,90,91} Some parents are responsible for maintaining complex technology such as ventilators, which, in addition to the stress of keeping the ventilator going, may wake parents up in the middle of the night when alarms go off.^92,93 Parents also are responsible for managing their child’s medical care by arranging and bringing their child to appointments and filling out copious amounts of paperwork⁹⁴ while creating an environment in which their child can have as normal a life as possible.⁹⁵ These efforts can occupy a great deal of time.⁹⁶ One study of parents whose children had gastrostomy tubes, compared to parents whose children did not, found parents in the first group devoted more than 7 hours a day to technical and nontechnical care of the child compared to 3 hours in the second group.⁹⁷ How this work is organized, distributed, and completed likely varies across family structures, and some evidence also exists that stronger family function is related to better execution of disease management activities.^98,99,100

Psychological Distress and Coping

Parents of children with serious illness can experience acute emotional distress and high levels of uncertainty about their child’s future.^{101,102,103,104,105} Parents and siblings have been observed to suffer from an array of psychological and physical forms of distress: parents report depression, grief, guilt, and anxiety as well as problems such as insomnia, headaches, and musculoskeletal pain. One study of parents of children with severe cardiac disease, compared to parents of healthy children, found substantial decrements in the parents’ QOL in general and specifically regarding emotions, physical health, and vitality.⁹⁸ Also, posttraumatic stress disorder (PTSD) was first described in survivors of childhood cancer in the late 1980s.^106,107 Subsequent research has documented the presence of PTSD in 10% of mothers of childhood cancer survivors but noted that 95% of mothers met the criteria for one component of PTSD, namely re-experiencing.¹⁰⁸ PTSD symptoms have been noted in other populations including parents of children who received care in critical care settings.¹⁰⁹ Bereaved parents also report symptoms of PTSD long after their child’s death. One study surveyed parents 13 months after their child died in an intensive care unit (ICU), finding that many parents reported symptoms of PTSD (35% of mothers and 24% of fathers) and depression (35% of mothers and 30% of fathers).¹¹⁰ Another study evaluated parents 5 years after their child was diagnosed with cancer, finding that 19% of mothers and 8% of fathers reported symptoms of PTSD. For bereaved parents, 20% of mothers and 35% of fathers reported PTSD symptoms.¹¹¹ In another study of parents 5 years after their child was diagnosed with cancer, 66% of parents reported low peace of mind, regardless of their child’s relapse status. This low peace of mind was associated with persistent worries about relapse, new toxicities, and persistent limitations on their child’s life as a result of the cancer and its treatment.¹¹²

For families of children with CCCs, engagement and effective communication with clinical teams might support coping and adaptations. In a qualitative analysis of focus groups, bereaved parents highlighted the importance of maintaining relationships with the clinical team, the benefits of high-quality communication, and the impact of negative interactions with clinicians on their grief.¹¹³ Furthermore, a study of parents whose children received PPC found a positive association between higher ratings of communication and continuity of care with lower levels of long-term parental grief. Higher reports of the child’s symptom burden, however, were associated with higher levels of long-term grief.¹¹⁴ Some of this grief might be related to the challenges parents face in meeting the needs of everyone in their family. One mixed-methods study found that parents engage in “parenting triage,” a process by which parents make difficult tradeoffs in their perceived responsibilities and obligations to all the children in the family. Parents described the “emotional and relational consequences of agonizing decisions made addressing needs of one family member over needs of another.” These parents felt “torn, guilty, overwhelmed, frustrated, afraid, exhausted, and heartbroken.”¹¹⁵ Another study focused on population-based data on the psychological outcomes of mothers and siblings of children with cancer, finding that mothers and siblings were more likely to seek psychological care than the general population, with the risk being highest among mothers who were younger, of lower socioeconomic status, and of rural residence.¹¹⁶

The impact of a sibling’s death can be long-lasting. Siblings may struggle with fears, traumatic reactions, isolation, difficulties in school and social settings, and the behavior problems that result.¹¹⁷ Nearly all bereaved siblings in one survey-based study reported that their sibling’s death still affected them 12 years later, and half of these respondents reported that this death affected their current educational and career goals.¹¹⁸ When asked for advice in another study, one-third of bereaved siblings said they had needed better medical information when their sibling was alive. Also, these participants recommended that clinicians provide clear guidance to parents about how to engage their other children before and after their child’s death.¹¹⁹

Beyond these findings, few methodologically rigorous studies have examined the impact of a child’s death on family members or the family structure. Much of what is known about parental reactions to a child’s death is based on samples of those who present for psychological treatment; presumably, a much larger percentage of bereaved families experience essentially normal patterns of grief and other mental health consequences from the death of a child without ever presenting for clinical care. Estimates of psychopathology in bereaved parents are therefore likely to be erroneously high.

Financial Concerns

A family’s financial concerns are related to both the work of care and coping. Many parents of children with serious illness report struggling to figure out how to cover the expenses associated with medical care and lost income due to missing work to care for the child.^{37,69,120,121} In particular, three areas of finances—insurance, paid work, and out-of-pocket expenses—are affected by caring for a child with a CCC. Due to the high costs of care, families may have difficulty finding or keeping insurance coverage and may find that certain services are not covered or that caps on spending prevent other services from being used.¹²² Often in families caring for a child with a CCC, one family member, usually the mother, decides to stay at home, forsaking the income and benefits provided by paid work.^123,124 For example, 52% of parents in one study reported a change in their job status due to their child’s illness, and only 52% of parents reported that their current job allowed them sufficient time to care for their child.¹²⁵ Out-of-pocket expenditures are also very high and might further impoverish families.¹²⁶ Although the Family and Medical Leave Act has provided some protections, parents report “difficulties in accessing and using benefits, including lack of knowledge by employers, complexity of rules and processes, and inadequacy of the benefits themselves.”¹²⁷ Furthermore, only about half of Americans are eligible for these benefits, and the protected time off is unpaid, which can exacerbate financial distress.

Communication Studies

Communication is an essential tool for palliative care clinicians, especially in pediatrics. Clinicians must navigate the communication preferences and needs of the child, the parents, and the siblings. In some situations, parents might also request support in communicating with their extended family or social support network.

Although communication is central to the role of palliative care clinicians, research on communication in pediatrics has largely focused on the pediatric oncology population. Additionally, most published studies are descriptive in nature, with limited (if any) studies of interventions to bolster communication outcomes. However, this body of literature continues to grow, and recent psychosocial palliative care standards reinforce the central role of communication in palliative care.^128,129 Here, we briefly highlight key findings and limitations in this communication literature, focusing on communication with parents and communication with children.

Communication with Parents

Most parents of children with life-threatening illnesses report a desire for disclosure of difficult information, such as poor prognosis or future limitations for the child. For example, most parents of children with cancer indicate that they value and prefer transparent disclosure of this information, even if it is upsetting.^130,131 Despite clinicians’ concerns about negative effects of this disclosure, many parents report that such information actually supports their hope despite its upsetting nature because it resolves uncertainty about the future.¹³²

Yet many parents’ communication needs are unmet. A national survey study from Denmark found that 42% of bereaved parents felt that information about their child’s end of life was given too late, and 31% felt they were “deprived of the option to say goodbye to their child in their preferred manner.”¹³³ Even when parents of children with cancer report “high-quality” communication, many still hold inaccurate estimates of prognosis and future limitations.^134,135 In part, these inaccuracies might result from the positive framing of this communication. For example, a study of neonatal ICU (NICU) family conferences found that discussions were optimistically framed and focused on the functional needs of the child rather than QOL for the infant or family. In this study, “most parents left the conference believing their infant’s prognosis to be more optimistic than did clinicians.” ¹³⁶

Communication Across Cultures

Communication among individuals from different social or cultural backgrounds poses additional challenges. For example, parents of color report higher unmet information needs. One study in pediatric oncology found that 87% of parents wanted as much detail as possible about their child’s prognosis, and this preference did not vary by race or ethnicity. However, significantly more physicians believed that black and Hispanic parents were less interested in this information.¹³⁷ Similarly, parents did not vary in their preferred role in decision-making by race or ethnicity. Yet physicians were significantly less likely to accurately predict the preferred role of parents of color (53% for white parents, 23% for black parents, 37% for Hispanic parents, and 43% for Asian/other race parents).¹³⁸ Additionally, parents of color held more active roles in decision-making than they preferred, which has been associated with increased decisional regret in other studies.¹³⁹ These challenges can be further exacerbated when parents speak different languages and clinicians must rely on interpreters.¹⁴⁰ However, most communication research samples have underrepresented people of color and non-English speakers. Moving forward, more research is needed to ensure communication practices are meeting the needs of all families, regardless of race, ethnicity, or culture.

Communication with Children

Communicating with children who have CCCs can challenge clinical teams for many reasons. First, some children with CCCs have limited cognitive capacity, which can impede communication efforts. Additionally, children will undergo substantial physical, developmental, and social changes as they grow. In each stage of development, children have different concerns, capacities, and preferences for involvement in their care.¹⁴¹ Parents and clinicians can also feel a sense of duty to protect the child from any harms of communication.¹⁴² Given these complexities, it is unsurprising that most communication literature has focused on adolescents and young adults (AYA) rather than young children. As such, we will focus this section on AYA studies.

Similar to studies of parents, most AYA patients with life-threatening illnesses value information about their treatment, prognosis, and future limitations. One oncology study, for example, found that 85–96% of AYA patients reported that prognostic information was very or extremely important throughout the first year after diagnosis of cancer. Additionally, 76–85% of patients reported that a numeric estimate of prognosis was very or extremely important.¹⁴³ Another study of 10- to 18-year-old patients with cancer found a widespread desire for more communication about QOL, pain and symptom management, and prognosis.¹⁴⁴ However, many of these communication needs are unmet. For example, one study found that AYA patients’ involvement in communication about their relapsed cancer was limited to responding to questions and participating in the history and physical examination.¹⁴⁵ Recent research highlights the importance of communicating directly with the AYA patient as there is not always strong congruence between patients’ reported needs and their families’ understanding of these needs.¹⁴⁶ Despite this desire for information, AYA patients need the information tailored to their social and cognitive development. Otherwise, clinicians might be viewed as “aloof, patronizing or cold.”¹⁴⁷

Other studies have explored the preferred role of AYA patients in decision-making. Again, nearly all of these studies have focused on cancer populations. One study, for example, found that 58% of AYA patients with cancer wanted to share in decision-making with their physician, and 51% preferred their parents to have limited involvement.¹⁴⁸ Another study found that AYA patients with advanced cancer or HIV infection “want to be able to choose and record (1) the kind of medical treatment they want and do not want, (2) how they would like to be cared for, (3) information for their family and friends to know, and (4) how they would like to be remembered.” ¹⁴⁹ Such goals can be supported by advanced care planning guides,¹⁵⁰ and many AYA patients have the desire and capacity to engage in such meaningful deliberation.¹⁵¹ However, only a minority of AYA patients with life-threatening illnesses utilize such advanced care planning tools.¹⁵² Much work is needed to better understand how to communicate effectively with children of all ages who have life-threatening illnesses, especially illnesses beyond cancer.

Limitations in the Communication Literature

The literature on communication in PPC is growing, but still has several limitations. First, nearly all studies have been observational or descriptive, with limited development of communication interventions. In a 2019 review article of communication interventions in oncology, no studies specifically targeted communication in pediatric oncology.¹⁵³ Although a similar review article does not exist for communication interventions in PPC, we suspect similar limitations exist.

Some studies have attempted to determine the effect of specialist PPC consultation on improving communication with parents, but these studies tend to be methodologically flawed. For example, a systematic review from 2019 explored the impact of specialist PPC consultation on communication experiences for parents. The authors concluded that studies suggest some positive benefit, but they rated 58% of studies as poor quality. The poor quality of these studies limits the utility of these results.¹⁵⁴

Another challenge in communication research is the dearth of reliable measures of communication effectiveness. For example, the previously mentioned oncology review article found that 88 communication intervention studies used 188 different outcome measures for communication, with 156 of these measures used only by single studies.¹⁵³ Without consistent and theory-driven outcome measures that are used across studies, comparison of results will remain a challenge.

Interventions and Outcomes

Most research in PPC to date has described patient characteristics, symptom burdens, and communication practices, among other topics. These descriptive studies have created the foundation for PPC research, but intervention studies are necessary to improve the care of children with CCCs. In recent years, the number of intervention studies in PPC has started to grow. Next, we describe the current state of studies on pharmacological and nonpharmacological symptom management. Similar to the need for interventions, this field also needs robust and reliable outcome measures to test and monitor the effectiveness of PPC interventions. Here, we also review the current literature on outcome measures in PPC.

Palliative Care Services

Several studies (including randomized control trials and observational designs) have examined the impact of receiving palliative care services, essentially as a bundle of different interventions, on patient and family outcomes. Patients receiving PPC experience reduced suffering, receive fewer medications and procedures, and are more likely to have medical orders setting limits on care received.^155,156 Early integration of PPC has been found to be both feasible and beneficial for families.^157,158,159 Other researchers have shown the benefits of providing PPC in outpatient and home settings.^160,161,162

Interdisciplinary, Team-Based Care

Providing palliative care to children with CCCs requires collaborative, interdisciplinary care. No single clinician or profession can provide appropriate care and attention to the child’s psychological, social, spiritual, emotional, and medical needs. Despite a growing body of evidence on team science,^{163,164,165,166} little work in PPC has focused on the functioning of interdisciplinary teams. In one study of parents whose children had refractory cancer, only 47% of parents described their medical team’s teamwork as excellent.¹⁶⁷ Other studies have explored the role of teamwork in communication, identifying several barriers to inclusion of nurses in difficult conversations.^168,169 Interdisciplinarity is the hallmark of effective PPC, and future studies must further explore the functioning of teams and develop data-driven best practices.

Pharmacologic Treatments

Medications are one of the mainstays of modern medicine. Polypharmacy is usually considered a problematic phenomenon among geriatric patients, but children admitted to children’s hospitals are typically exposed to five different medications (median count) during their hospitalization, with 10% of the patients exposed to 14 or more medications. By the time pediatric patients have been hospitalized for a month or more, the 10% receiving the most medication have been exposed to 40 or more medications.¹⁷⁰

The drugs used to treat children who are receiving palliative or hospice care have not yet been well-described. In a cohort of 834 pediatric patients enrolled in hospices across the United States, the most commonly used class of drug was opioids (72% of all patients), followed by hypnotics (60%), laxatives and other medications for the bowels (50%), antacids and other medications for the stomach (41%), and anti-epileptic drugs (38%) (unpublished data). In the United Kingdom, a small study found that 21 children being treated by hospice services received a median of 10 medications, ranging from 3 to 23. The most commonly used drugs were anti-epileptics (27%), analgesics (16%), laxatives (8%), anti-reflux agents (6%), muscle relaxants (6%), and anti-cholinergic agents (5%).¹⁷¹

The use of specific medications appears to vary substantially across hospitals and hospices. For instance, in a study of 626 children’s and general hospitals (which include many NICUs) in the United States, among terminal hospitalizations the exposure to opioids varied across hospitals from 0.7% to 99.1%, with the interquartile range extending from 35.3% to 59.5%.¹⁷² Studies of more specific pharmacologic interventions in PPC have been rare. Most pertinent studies have focused on children with cancer. For example, some groups have studied topical and systemic treatments for peripheral neuropathy,¹⁷³ phantom limb pain,¹⁷⁴ pain with needle insertion,¹⁷⁵ and opioid-refractory cancer-related pain.¹⁷⁶

Nonpharmacologic Treatments

Medications are only one of many possible interventions used in PPC to ameliorate symptoms or improve QOL. Much less is known about the epidemiology of other interventions, such as a child’s exposure to complementary and alternative medical (CAM) practices. In one study of 92 parents of children with cancer in the United States, 49% reported that their children used at least one type of CAM and 20% used herbal remedies, homeopathy, or vitamins in the 2 months preceding the survey,¹⁷⁷ which suggests that, if any of these children died, some may have continued to receive CAM treatments at the end of life.

A small number of clinical trials from other medical disciplines have evaluated nonpharmacological approaches to pain and symptom management. Some of these interventions have focused on psychological interventions to improve symptom management. For example, trials have tested cognitive-behavioral therapy¹⁷⁸ and yoga¹⁷⁹ as adjuvant treatments for pain for children with sickle cell disease. Others have studied the use of guided imagery to decrease pain and anxiety for children undergoing surgery.¹⁸⁰ Additionally, the PRISM study evaluated the impact of a brief intervention targeting stress management, goal-setting, cognitive reframing, and meaning-making on supporting resilience for children with cancer or diabetes mellitus type 1.^181,182 Similar interventions should be developed and evaluated in PPC in the future.

Outcome Measures

To ensure that interventions and PPC services are benefiting patients, researchers and clinical teams need reliable and appropriate outcome measures. Currently, researchers and clinicians have not identified consensus definitions or measures of value or effectiveness. This deficiency presents a major barrier to ensuring the quality and efficacy of specialized PPC. For example, a 2019 scoping review of outcome measures in PPC identified 46 studies that compared specialized PPC with usual care. Of these studies, one was a randomized controlled trial. The most commonly used indicators were location of death, length of stay in hospital, and number of hospital admissions. Overall, these studies used 82 different indicators, and only 22 of these indicators were defined the same way in at least two studies.¹⁸³ Another group performed a systematic review of instruments to measure outcomes in PPC. They found 15 articles that utilized quantitative measures, and 9 of these measures were estimated to have at least moderate quality. Only 5 of these measures included patient-reported outcomes. Also, the authors noted, “We had no access to the developmental process of the instruments used to present the underlying concepts that were underpinning the constructs.”¹⁸⁴

Multilevel System of Care

Children with CCCs receive care in a broad range of settings, spanning from the child’s home to ICUs, with each level of this complex overall system of care playing important roles for these children and their families.

PPC in the home is intended to increase the QOL of patients and families, and some, if not a majority, families prefer being at home to care for the child.⁸⁸ From 1989 to 1993, only 12.3% of children with CCCs died in the home; from 1999 to 2003, 17.7% of children with CCCs died in the home. In the same period, hospital deaths declined and deaths at all other sites and care institutions remained comparably stable.¹⁸⁵ This pattern of a rising proportion of pediatric deaths attributed to CCCs occurring at home is traceable to 1979, shortly after the first study demonstrated that families could support dying children in their homes.¹⁸⁶ Over the same time periods, stark differences in the proportion of deaths that occurred at home are evident and persistent among patients classified on their death certificates as white, Hispanic, or black.¹⁸⁵

Home care usually involves the partnering of skilled nurses with the parents, who are able to share knowledge and divide tasks related to the work of care.¹⁸⁷ Issues to be considered in home care include the work of care, impact on the family, the likelihood of adverse events, and cost.^{88,188,189,190,191} In a study of parents of 48 children who died from cancer, 88% of parents chose “at home” as the most appropriate location of death in hindsight, but only 41% of parents provided palliative care in the home and 48% of children died at home.³⁴

Hospice Care

The term “hospice” has different meanings in different contexts. In the United States, hospice care is defined operationally by insurance coverage benefits and is provided by dedicated hospice agencies and their staff; care is almost always in a patient’ home or in a specific hospice facility or hospital. In Canada, hospice typically refers to a free-standing building. In the United Kingdom, hospice care can refer to either palliative care in general or to the buildings where respite care and end-of-life care are provided for children.

A study of Florida Medicaid pediatric patients who died revealed that only 11% received any hospice services, but those who did were more likely to be non-Hispanic whites than non-Hispanic black and have chronic conditions and longer Medicaid enrollment than non-hospice users.¹⁹² In a study of pediatric oncologists, physicians with access to hospice that accepted patients receiving chemotherapy had more patients die outside the hospital, but not all hospices admit patients actively receiving chemotherapy.¹⁹³ A broader survey of pediatric medical providers experienced in end-of-life care reported that 86% of responders thought hospice was beneficial, especially in the provision of nonmedical services.¹⁹⁴

Hospitals

While there is a trend of increasing hospice and home-based PPC, pediatric death still occurs mostly in the hospital setting. From 1989 to 2003, 80.1% of childhood deaths resulting from CCCs occurred in the hospital (down from 85.7% in 1989 to 2003), with considerable variation in the location of death by CCC category.¹⁸⁵ The same study also demonstrated that infants and children from racial and ethnic minorities were more likely to die in the hospital than were older children and nonminority children.

Hospitalizations of children with CCCs who subsequently died were characterized by long lengths of stay, mechanical ventilation during their terminal admissions, and death in the ICU. In a study that linked information from death certificates to hospital use records from 1990 to 1996 in Washington state, 458 infants with CCCs who died under a week of age spent 92% of their lives in the hospital, and 286 infants with CCCs who died during the second to twelfth months of life spent 41% of all their lives in the hospital.¹⁹⁵ For children and young adults with CCCs, the median time spent in the hospital in the year preceding death was 18 days, and a third of this group was hospitalized until the last week of life. Across all age groups, rates of hospital use increased closer to the child’s death. Half of hospitalized infants and 19% of children and young adults with CCCs were mechanically ventilated during their terminal admission. Studies from hospitals in a range of countries (the United States, Australia, and the Netherlands) suggest that although a number of deaths take place in the operating room and emergency departments, most pediatric deaths occur in the ICU.^{196,197,198,199} In a study of a UK children’s hospital, infants with congenital malformations and perinatal conditions were most likely to die in the ICU (odds ratio [OR] 2.42; 95% confidence interval [CI ]1.65, 1.35), and older children with cancer were the most likely to die outside the ICU (OR 6.5; 95% CI 4.4, 9.6).²⁰⁰

A more recent Canadian study of 1,620 children evaluated the costs associate with end-of-life care, finding that most children who died in Ontario received end-of-life care in acute care settings. The average cost of healthcare in a child’s last year of life was $78,332 (Canadian), with a median cost of $18,450. Two-thirds of these costs were incurred in acute care settings. The vast majority of children with perinatal/congenital illness (88%) and chronic illness (80%) died in acute care settings. Only 33% of children received any home care during their last year of life.²⁰¹

Over the past decade, hospital-based palliative care services have begun in many pediatric hospitals across the country. An evaluation of the impact of one such interdisciplinary program in Boston documented, after the program’s establishment, an increased frequency of hospice discussions (76% vs. 54%, for an adjusted risk difference of 22%), earlier documentation of do-not-resuscitate orders (18 vs. 12 days), and fewer deaths in the ICU or in other hospitals (decrease of 16%). Parents also reported that their child suffered less pain (adjusted risk difference of 19%) and that they felt more prepared during their child’s last month of life and at the death.²⁰² In another study on the impact of a PPC program in Germany, 69% of families preferred to have their child at home for the death compared to 18% of families before the program was instituted.²⁰³ Yet barriers to consultation persist. A systematic review of specialist PPC initiation in pediatric cancer, for example, found that 55% of pediatric oncology patients received any specialist PPC services prior to their death. The first discussion of PPC and initiation of PPC often occurred shortly before the child’s death.²⁰⁴

Respite and Other Modalities of Care

Beyond hospital and hospice care, other modalities of care might also be useful for seriously ill children. Broadening the definition of the population in need to encompass children with complex or special healthcare needs, respite care provides parents with time off from being in charge of this care. Respite care also has different meanings and purposes attributed to it by different families.²⁰⁵ In a study among caregivers of children with cerebral palsy in Canada, 46% of caregivers reported using respite services in the past year, and 90% of caregivers indicated that respite was beneficial for both the child and family.²⁰⁶ In another study of children with special healthcare needs, researchers demonstrated that a program in an emergency department setting to provide telephone advice and care coordination enhanced parental satisfaction with emergency care.²⁰⁷ Furthermore, respite care through a pediatric hospice can serve as an entry point to palliative care services for families who initially have concerns about seeking these services.²⁰⁸

Future Directions

PPC is now firmly established as a professional medical discipline across multiple countries. The body of research to support this field is growing, but many unanswered questions remain. For guidance, we will refer to the results of a workshop of PPC leaders and researchers, in which they identified seven priorities for future research. These should be prioritized as PPC investigators develop research agendas in the future (Box 2.1).

Related posts:

Quality in Pediatric Palliative Care Program Development The Language of Pediatric Palliative Care Relationships with Children and Ensuring Their Voices in Decision-Making Advanced Heart Disease Children’s Voices: The Experience of Patients and Their Siblings

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