Living With Chronic Disease

Families perceive chronic illnesses in a variety of ways. Some can integrate their illness into their daily routine, whereas for others, it may be an insurmountable roadblock to normal life. Families should be allowed to define their own roles with the clinical team and responsibilities in a way that conforms to their beliefs. Roles within the family unit will evolve as the patients’ clinical condition changes and must be flexible.

The clinical environment, parenting, family disruptions, and coping were identified as major challenges in a study of children with chronic kidney disease. Parents felt traumatized seeing their children undergo invasive procedures and were overwhelmed with being both parents and caregivers. Caring for their child caused both spousal and sibling neglect, which led to family tension. Parents needed support and relied on their healthcare providers and other parents to help them cope.

All families are challenged by their child’s needs, and chronic illness has a profound impact on the well-being of all family members. Daily routines become more complex and may be centered around the needs of the chronically ill person. Encouraging families to articulate their goals and concerns may provide key information on how to intervene more thoughtfully and respectfully with families.

Preparing the Child for Transplant

The most common indication for pediatric liver transplant worldwide is biliary atresia, and the majority of these patients are transplanted as infants or young toddlers. Therefore their capacity to understand what is happening is limited by virtue of their age. It is important to create a familiar and comfortable atmosphere in the hospital and allow family members to be with them as much as possible.

Preschool-age children may benefit from some teaching about their condition; remember to use simple words and explanations. When discussing an upcoming transplant, consider the child’s age and maturity level. Children are more likely than adults to believe their illness is a punishment caused by their wrongdoing; assure them they are not at fault. The transplant team should answer any questions truthfully but should not overload the patient with too many details. Simple pictures explaining what will happen may also be helpful. Adolescents in particular tend to rely on their own social networks, and they should be encouraged to talk about their upcoming surgery with their peers for support. When appropriate, classmates and friends can write/visit to maintain communication. It is important that patients are informed of support and resources in their language and geographical area. Electronic resources are easily often accessible, and the transplant center should steer patients toward reliable information that is relevant to their disease. Because of the rarity of these diseases, online social networks have created an opportunity to allow families worldwide to connect and share information with each other.

Referral

Referral to transplant indicates a shift in the level of care. Families with children who have chronic liver disease may have already suffered psychosocial and cognitive sequelae. They are accustomed to coping with a life-threatening disease and may view transplantation as a chance for a fresh beginning or the end of attempts to manage a progressive and chronic medical condition. Children who develop acute liver failure have to cope with the other end of the spectrum. Previously healthy, these families and their children must navigate a complex medical system that often requires urgent and invasive medical interventions. They may have to proceed with treatments that they do not fully understand and readjust expectations of their future. Because the majority of LT are performed in children with chronic illness, it is thought that families will have some level of familiarity with the medical system. However, the need for surgery, meeting new providers, and requirements for listing, including insurance approvals, may cause significant stress on an already stressed family system.

Despite an increase in the number of solid-organ transplants performed, the majority of patients are unaware of all the complexities of the transplant process. There are little data on how to initially refer a patient for transplantation as a treatment alternative and even less data on the parental/patient reaction to such news. Lang et al. interviewed parents of children who were referred for lung transplantation for cystic fibrosis. The parents recommended discussing transplant before a medical crisis point, providing as much information in a gradual manner and finally providing written handouts because it was difficult to remember all the information provided.

Transplant teams usually follow a well-defined script when delivering information regarding the transplant process to patients and families. They should be clear that LT is an intervention that extends life but is not a cure. It transforms the patient from a terminal illness, not to perfect health but to a health state requiring lifelong follow-up and medication. This concept may be more familiar for patients who are already in a chronic disease state, but for patients with acute presentations, the idea of extending life may be novel. Long-term risks must be stated at the outset, such as infections, organ rejection, PTLD, and complications from taking immunosuppression.

The transplant team must be aware of the different stressors and should ideally individualize their approach based on the unique needs of each family. The social and psychological impact on the child and family will vary based on disease type, chronicity, individual and family functioning, and outcomes from previous therapies. The transplant team must make ongoing assessments of family stress and provide resources to families to combat or adapt to new situations.

Psychological Aspects

Children with chronic illness have increased physical, developmental, behavioral, and emotional needs. Patients and parents constantly worry about their disease, medications, and appointments. This often leads to increased parental involvement and for a longer duration. Once the patient has been listed for transplant, this process may intensify, leading to increased distress.

In a study from Turkey, parents of 38 patients listed for LT completed the Hamilton Depression Rating Scale, Hamilton Anxiety Rating Scale, and Clinical Global Impression Scale. Two-thirds were diagnosed with a clinical psychiatric disease, with 18% reporting depression and 47% an anxiety disorder, both of which are above average for the general Turkish population.

Tarbell and Koscmach investigated the quality of life and stress in the parents of children before and after liver and intestinal transplantation using the Brief Symptom Inventory. Some 41 mothers and 20 fathers were included, and increased psychological symptoms were reported in 31 parents. Fathers were found to have greater stress than mothers.

In another study, 34 mothers and 22 fathers were evaluated pretransplant through the Brief Symptom Inventory, Coping Strategies Inventory, and Multidimensional Scale of Perceived Social Support. Some 21% of parents reported significant distress, with mothers using more coping strategies than fathers. Mothers with poor psychological functioning also tended to have less social support.

In addition to the transplant team, the pediatrician is an essential element in ensuring a family’s well-being. They often care for other children in the family and may notice adverse effects in these family members of which the transplant team is unaware. The family environment may be reflected in children’s behavior and the parents’ stress of having both healthy and ill children. A nurturing home environment, where family members support each other, is characterized by high medical adherence. Even the parents’ spiritual state is consequential, as it indirectly affects the health of the child in addition to their own health.

Identifying factors associated with, and understanding the level of, parental and child stress is vital so that an effective intervention can be made. Risk factors may include frequent hospitalizations, procedure anxiety, lifestyle changes, adherence difficulties, missed schooling, treatment decision making, and anticipatory grief. Early recognition of psychosocial risk factors in families and early intervention can prevent or reduce psychological distress. This is an essential element of improving outcomes for children and families.

Waitlist

As a transplant physician, the waitlist period is often the most difficult part of the transplant process. Both for the team and the family, there is uncertainty about the outcome of both the waiting period and the transplant itself. The unpredictability greatly challenges the coping ability of the family. It has been compared with “supreme torture” and “living with a time bomb.” This is a difficult area to study because patients and families are very heterogenous, particularly with regard to coping skills. We do know that parents today report many of the same feelings and concerns as their peers did 30 years ago at the beginning of the transplant era.

Active and ongoing assessment of the transplant candidate during the waiting period may detect neurocognitive changes before they add to the complexity of care. One study found a quarter of children awaiting heart transplantation met criteria for psychiatric disease including depression, anxiety, phobia, and adjustment reactions.

Feelings of guilt are common in parents because a cadaveric organ requires the death of a donor, possibly another child of a similar age. Adolescents may feel guilty as a result of benefiting from another person’s death, whereas younger children may worry about having a stranger’s organs inside of them. Children may suffer from low self-esteem or depression if a suitable donor cannot be found or time on the waitlist is more prolonged than expected. In the case of living donor LT (LDLT), they may also suffer from guilt if there is graft loss or the donor suffers a complication. Another worry is whether the disease was preventable, the result of something that occurred during pregnancy, or punishment from a higher power. There is a desire for a high pediatric end-stage liver disease (PELD) score to expedite transplant but wanting to keep their child healthy. These feelings may manifest as social isolation to avoid contagious germs, which, in turn, aggravates feelings of loneliness and loss of social support. Parents may also overindulge and infantilize their child to compensate for what the child is going through, which risks the balance with their other children or at times their spouse or significant other.

There have been conflicting results from studies looking at stress and coping in transplant families. A study of LT families found greater stress before transplant, which decreased over time, leading to increased coping and decreased uncertainty. A second study in solid organ and stem cell transplants found increases in stress in the first 6 months post-transplant. These contradictory results may be a result of the different demographics studied but also demonstrate the need to evaluate parental distress and assess variables that may explain variances in parental adjustment. Lower socioeconomic status, fewer coping resources and the use of avoidant coping strategies, family conflict, previous child and parent illness-related distress, and premorbid child-internalizing behavior problems are predictors of poor parental adjustment in solid organ transplant. Other factors such as ethnicity and culture have not been as well studied or have not been found to be significant.

Parental distress, as well as having negative consequences to the individual, also impacts child well-being by increasing risks for depression and leading to poorer medication adherence. In a study of heart transplant recipients whose parents were classified as high risk by a psychiatrist, high-risk patients were 3.4 times more likely to have rejection, 3.1 times more likely to be inpatient, and 2.9 times more likely to have low immunosuppression trough levels compared with controls. Family adaptation was negatively affected by higher perceptions of stress, fewer coping skills, and increased family strain. Additionally, there may be a loss of social support and increased isolation when families have to relocate to be nearer to the transplant center. This also leads to disruption in family life and may cause financial pressures because of job loss or decreased wages from missed work.

Studies on the adjustment of siblings during the pretransplant phase are limited, although there is an inevitable disruption in the entire family associated with transplantation. In families of children awaiting cardiothoracic transplantation, levels of emotional and behavioral problems were similar to those seen in their siblings awaiting transplant. Parents in this study indicated that they tend to focus on their ill child, spending little quality time with their other children, which is likely to be a contributory factor to the behavioral issues in siblings. Similar rates of behavior problems were identified in siblings of liver transplant candidates, with their mothers reporting significantly higher levels of personal strain than mothers of siblings who did not have behavior problems.

It may be challenging to determine the proportion of parental psychosocial morbidity related to chronic illness versus what is related to being on the waitlist. However, it is clear that waiting for a transplant presents a unique psychosocial challenge for the entire family, and family-centered interventions, as well as psychosocial support, remain of paramount importance.

Financial Stress

Although private or government insurance programs may provide the bulk of payments for patients receiving solid organ transplantation, not all costs may be covered, and insurance coverage can vary widely according to country. Other well-studied models of pediatric chronic disease, such as cancer, have identified financial hardships as a consequence of therapy. In addition, pediatric studies have shown that poverty is correlated with negative health outcomes.

LT recipients face frequent admissions for medical complications both before and after transplant. This can lead to work disruptions for parents and compound psychological and financial stress. Nonmedical costs include lodging near the center for families who may live a distance away, food, transportation to the transplant center for subsequent visits, childcare, and lost wages. Contemplating these costs, along with caring for a sick child, is a daunting task. Financial counseling should be provided at the start of the transplant journey to help prepare families for this possibility. Bona and colleagues surveyed 86 parents of children with advanced cancer and found 94% had some work disruptions, and in 42%, a parent quit their job. Families with the lowest incomes were disproportionately affected, and 15% of the nonpoor became poor because of their child’s illness. The highest-risk families were lower income whose children were admitted for extended periods of time and lived far from the treating center.

In a second study, Bona et al. surveyed 45 families of pediatric patients with stem cell transplants to describe family-reported poverty, as well as the impact on family income and clinical outcomes. As a result of work disruptions, 20% of families reported losing more than 40% of their annual income, and low-income families experienced the greatest impact compared with wealthier families (39% vs. 7%; P = .02 ). A quarter of families had difficulty paying bills because of medical costs, and 38% of families also reported some material hardship during the post-transplantation period ( Fig. 7.1 ). Coping strategies included selling personal property, taking out a loan or mortgage, or incurring credit card debt (22%). Cost shifting, through reduced college or retirement fund saving and allowing life insurance to lapse, was more prevalent in lower-income families. This study also uniquely looked at clinical outcomes and found children from low-income families and those who reported material hardship were more likely to experience graft-versus-host disease within 180 days of transplantation compared with their wealthier counterparts. In a cohort that was 100% covered by health insurance before transplant, the financial burdens reported were all above and beyond costs covered by insurance.

Family material hardship by FPL. This shows the percent of the cohort reporting each of the measured concrete resource needs, stratified by FPL. Comparison of proportions by FPL is denoted with significant P values.

FPL: Federal Poverty Level (From Bona K, London WB, Guo D, et al. Prevalence and impact of financial hardship among New England pediatric stem cell transplantation families. Biol Blood Marrow Transplant. 2015;21(2):312-318.)

Medical complexity can also cause financial hardships, even in wealthier families. In a study comparing families of medically complex children (CMC) with those with asthma, families of CMC were 2.6 times more likely to report failure to pay housing costs and 2.9 times less likely to expect help from family/friends despite having a higher socioeconomic status.

Because there are many similarities between cancer patients and LT recipients, such as prolonged inpatient admissions, therapy far from home at referral centers, and the need for full-time parental caretaking secondary to isolation precautions, it is likely our patients face a similar burden. The global presence of social media has allowed some families to successfully use Facebook or GoFundMe to help raise funds for transplantation. For others, several organizations exist to help navigate costs of transplantation, and an excellent directory of such resources can be accessed at https://transplantliving.org/financing-a-transplant/financial-resources-directory/

Misconceptions

It is important to quickly address any misconceptions surrounding transplantation, donation, and time on the waitlist. Some of these may come from the media or are propagated in transplant forums on social media. Families on the waitlist face great uncertainty, and thus information regarding their child’s clinical condition, change in PELD score, and need for follow-up must be as consistent and transparent as possible.

Persons desiring to be living donors, even if parents, must undergo a thorough medical and psychosocial evaluation. Between 52% and 64% of living donors are disqualified, most commonly for donor reluctance or fat on liver biopsy. Although families desire the workup to be expeditious, it is a time-intensive process. The transplant team may need to provide “an out” for unwilling donors who have been pressured into donating and must evaluate all other criteria, including the ability to support their own families, motivation, and relationship with the recipient. Education should focus on organ donation as a selfless gift, a gift whose timing the transplant team cannot control.

Health Literacy

Health literacy, defined as “the degree to which individuals have the capacity to obtain, process, and understand basic health information and services needed to make appropriate health decisions,” is important to consider when transplantation information is provided. Almost half of the US population is unable to follow healthcare recommendations or fully understand informed consent for procedures. The implications should be obvious. Transplantation requires a prolonged and complex evaluation as well as a complicated follow-up regimen where deviations may result in severe morbidity. Studies in adult transplant recipients reveal 41% have limited health literacy, and serious gaps in transplant knowledge exist. A qualitative study in eight adolescent kidney transplant recipients suggested that acquiring more knowledge about the transplant experience helped them to cope with transplant-related stressors. These included body image, pain, desire to be normal, and communication breakdowns. Patients varied in the ways they wanted to be educated but were united in the desire to have more information about self-management and preparation for transition to adult healthcare.

Consent and Assent

Similar to most centers in the United States, it is our practice to obtain assent for transplant from patients who are between 12 and 18 years old. Consent is obtained from the parent or legal guardian for all patients up to the age of 18. We advocate an open and honest approach in talking to young children about their illness and disease. In cases where parents do not want to disclose the reality of transplantation, we have successfully employed social workers and psychologists to help them understand the child’s need and right to know. We then work with the family to explain what will happen in an age-appropriate manner. We do ensure the child has an opportunity to ask questions about the process, and parents are sometimes surprised by their level and depth of understanding. The delivery of information in an unhurried, empathetic, and nonthreatening manner has been shown to lower parental stress levels.

Although involving children in decisions relating to their care is well accepted, there remains debate about the appropriate age for certain decisions as well as how to proceed if the adolescent and parent disagree. Here, we enter the arena of competency, autonomy, and their legal implications, which vary by region, culture, religion, and country. When faced with such issues, our practice is to involve an ethics team, who are available to provide an unbiased outside opinion.

Decision Making and Information

Patients and families must understand the reasons for referral for transplantation as well as the risks and alternatives. Studies of parents of heart transplant candidates have identified the significance of understanding the family’s illness narrative and individual and family belief systems and values, including issues of culture and spirituality, irrespective of the illness course that brings the child and family to the point of transplant referral. Once the transplant team understands the family’s journey, they may be able to better tailor their approach to shared decision making and disseminating information. The pitfall of this approach is the possibility of under- or overestimating the capability of the family to absorb critical information. We advocate providing more rather than less information whenever possible and developing center-specific guidelines on a minimum level of information to share.

Transplant Choice

With the commoditization of transplantation, patients and families have much more choice regarding their center compared with two decades ago. Limitations that exist today are usually because of geographical preference, patient acuity, or insurance coverage. The choice of a transplant center has not been studied very well. In certain cases such as acute liver failure, it is often the nearest facility; however, for specific disease types, such as hepatoblastoma, families may seek certain expertise and travel outside their home state.

In a study of over 56,000 data requests from nonhealthcare professionals over 6 years to the Unified Network for Organ Sharing (UNOS) and the Scientific Registry for Transplant Recipients (SRTR), the most common request was about wait times and help in interpreting statistics. Program-specific questions included contact information, interest in multiple listings, how to be waitlisted, and living donor information. Interestingly, a quarter of requests came from within the candidates’ support network, a group who are less likely to have direct contact with the transplant team. Many sought information to help select a program that specialized in transplanting patients with their characteristics.

These results demonstrate the variability in literacy and numeracy of the consumers of these data, and they argue for easier scales. Access to transplant information is a potential source of disparities in access to transplantation.

Transplant Data

Transplantation is one of the most highly regulated fields of medicine. In the United States, Congress passed the National Organ Transplantation Act in 1984, which authorized the Department of Health and Human Services to grant funds, creating the transplant structure that exists today. Providing some knowledge of the infrastructure and institutions governing transplantation may benefit families as they start the transplant journey.

In Europe, Eurotransplant helps facilitate the allocation of organs in Austria, Belgium, Croatia, Germany, Hungary, Luxemburg, the Netherlands, and Slovenia. They have also partnered with transplant organizations in other European countries including the United Kingdom and Spain to share organs and increase transplantation rates. In Latin America, although there is no uniform allocation system, the Sociedad de Trasplante de America Latina y El Caribe (STALYC) has created a registry to track data and provide information to transplant professionals and the public. In Japan, the Japan Organ Transplant Network collects data on all transplant activity, and similar groups exist in almost all countries where transplantation is active.

Today, when we seek any information or data on an unfamiliar subject, our first instinct is to search online. Many families and their extended networks will naturally search for information regarding their transplant center, and in the United States, they may directly query UNOS. The commoditization of transplantation has given patients and families more choice regarding their transplant center compared with two decades ago. Geographic barriers still exist, and there remains debate around the ethics of transplant tourism. In other cases, patient acuity, finances, and insurance coverage will dictate the transplant center. However, for specific disease types such as hepatoblastoma, families may seek certain expertise and travel outside their home state. Access to transplant information is a potential source of disparities in access to transplantation and must be disseminated as widely as possible.