On March 9-10, the Institute of Medicine’s (IOM’s) National Cancer Policy Forum (NCPF) and the American Cancer Society (ACS) sponsored a workshop on Comprehensive Cancer Care for Children and their Families to discuss recent trends in comprehensive cancer care for pediatric patients – and their families.
During the two-day event, pediatric oncologists and palliative care clinicians shared research from the pediatric oncology landscape, while patients, survivors, and families offered their unique perspectives on the experiences of living with a cancer diagnosis.
“There was a strong focus on survivorship,” said Richard A. Larson, MD, professor of medicine at the Pritzker School of Medicine in Chicago, an NCPF member who attended on behalf of ASH. “While there has been considerable success treating many childhood cancers – especially leukemia, lymphoma, and neuroblastoma – there are still issues with minimizing the toxicity of the drugs used and their effects on children’s growth and development, both physical and psychosocial.”
ASH Clinical News spoke with Dr. Larson and other presenters and attendees about the findings from the two-day workshop, including the need for education, improvements in palliative and psychosocial care, and potential policy actions to improve care and outcomes.
Underscoring the Differences Between Adult and Pediatric Cancer Care
One of the core issues differentiating pediatric cancer care from adult cancer care is drug side effects, Dr. Larson noted. For example, as Smita Bhatia, MD, from City of Hope in Duarte, California, discussed in a presentation, certain cancer drugs carry cardiotoxic effects. Pediatric cancer patients may be particularly susceptible to these effects, and the effects be much longer-lasting in children than in adults. While researchers have identified a genotype polymorphism that indicates which patients are more likely to experience anthracycline-induced cardiotoxicity with doxorubicin and which subsets are likely to be cured with less intensive dosing, there is still a great knowledge gap about how patients experience these adverse effects.
“As oncology investigators, we tend to look at the bottom line, asking how many patients survive their cancers,” Dr. Larson said. “We’re willing to accept a fair bit of toxicity to get someone to that bottom line. But we have relatively little information from patients, themselves, as they are going through these sometimes complicated – and certainly uncomfortable – treatments.”
“The knowledge gap extends to many consequential questions that may ultimately affect outcomes,” Dr. Larson continued. “Is the patient able to continue attending school? Is he or she being ostracized? Has he or she lost friends? Often, these types of patient-reported outcomes are not included in the design of pediatric oncology clinical trials,” Dr. Larson said.
Integrating Palliative Care Throughout the Disease Course
One issue common across cancer care – whether adult or pediatric – is the access to and use of palliative care. In moderating the session on “Integrating Pediatric Palliative Care: Ensuring Child and Family Well-being along the Continuum,” Chris Feudtner, MD, PhD, MPH, from Children’s Hospital of Philadelphia, chose “courage” as an opening theme.
“It takes a little bit of courage to talk about and, more importantly, to actually engage in some of the problems that pediatric patients with cancer and their families encounter,” he said. For both providers and researchers, he continued, “it takes a certain level of courage to go into that room where a patient is suffering and may be afraid to even find out what is going on.”
The “hard” quantitative side of research needs to be brought to bear more thoroughly toward the psychosocial side of pediatric cancer palliative care, Dr. Feudtner said. Equally important is the need for program funding to build “vibrant pediatric palliative care programs at all centers with good business plans underneath.”
Communication is also a major component of palliative care, as are the relationships being built between the provider and the child, the provider and the parents, and the parent and the child. “This is tough stuff,” Dr. Feudtner commented, “but if done right, it becomes the fabric of resilience that is so important to long-term outcomes.”
There have been many strides in psychosocial care over the past decades, when cancer conversations were shrouded in secrecy, according to Lori Weiner, PhD, from the National Cancer Institute, who walked attendees through the development of palliative psychosocial care in this setting, and outlined areas where progress is still forthcoming.
Children and families at all stages of the cancer trajectory experience distress and suffering from “family life disruptions, rigorous treatments, unpredictable setbacks, repeated invasive procedures, and burdensome symptoms.”¹ However, many barriers to effective palliative pediatric care prevent children and families from taking full advantage of these services, Dr. Weiner added. These barriers include: ineffective communications between health-care providers and families, misalignments between patient/family needs and available resources, and the lack of formal education in palliative and end-of-life care for children.
The changes being implemented in adult cancer care have yet to trickle down to pediatric care. For instance, Dr. Weiner said, a simple terminology change in adult settings (from “palliative care” to “supportive care”) has led to earlier and more frequent use of services.²
Addressing misconceptions about palliative care and integrating it into the entire disease course, from diagnosis onward, would also benefit patients and caregivers.
Cost of Care: A Problem without an Age Limit
“In the United States, it’s said that health-related costs are a common cause of bankruptcies,” Dr. Larson said. “It seems that each new drug comes in at a higher price, and the price jump is not fully justified by a greater benefit. Bankrupting a family to obtain an extra two or three weeks at the end of life when the patient is not well hardly seems worth it,” Dr. Larson commented, noting that the targeted cancer therapies come with especially high price tags.
Kira Bona, MD, MPH, from Dana-Farber Cancer Institute, addressed both the impact of socioeconomic status on outcomes and the material hardships wrought by illness and its treatment in her presentation. While race, ethnicity, and socioeconomic status are known to drive cancer incidence and outcomes in adult oncology, data in pediatric oncology are sparse, Dr. Bona said.
Dr. Bona highlighted other gaps limiting our understanding of socioeconomic status on outcomes and material hardships:
- Inferior survival in racial and ethnic minorities is documented but poorly understood.
- Pediatric oncology providers do not routinely consider poverty as a mediator of clinical outcomes and quality of life.
- Researchers do not assess financial hardship as a consequence of therapy on a regular basis.
Pediatric oncology research outcomes are meaningful, she emphasized, only if targetable measures of poverty are identified – such as the six-item U.S. Household Food Security Scale and definitions of housing insecurity. Dr. Bona detailed research supporting that these measures of poverty are targetable: programs addressing food, housing, and energy insecurity improve childhood health, reduce under-nutrition, and reduce hospitalizations.
Other analyses have demonstrated that poverty negatively impacts survival in pediatric cancer: children living in middle and high poverty areas experienced 31 percent and 88 percent higher all-cause mortality, according to a report from the Department of Health and Human Services.3 Federal estimates also show that material hardship increased chances five-fold of being in fair or poor health, with higher rates of hospitalization, injury, and infectious disease.4
Unfortunately, awareness of these social determinants of health is not reflected in research protocols and upfront systematic data collection, making their effects on outcomes opaque. Reliance on non-targetable measures, like zip code as a proxy for income, is inadequate for useful assessment of interventions.
Dr. Bona recommended systematic and standardized collection of socio-demographic variables, including screening for material hardship, and robust testing of their relationship with clinical outcomes. This, she said, can be implemented immediately.
Pediatric-specific oncology research confirms the significant financial burden that childhood cancer treatment places on families; conversely, patients from low-income families have a decreased adherence to oral chemotherapy and decreased overall survival.
According to data from trials from the Dana-Farber Cancer Institute, children in high-poverty areas have lower five-year survival (p=0.02) and are more likely to experience early (<1.5 years) relapse (p=0.009). Further research from trials in the Poverty and Survival DFCI ALL Consortium demonstrated that 20 percent of families experiencing a new cancer diagnosis report material hardship; following six months of chemotherapy, 30 percent reported material hardship, and 25 percent reported annual income losses of >40 percent due to treatment-related work disruptions. In families in which the patient underwent transplant, material hardship was more likely, as was incidence of graft-versus-host disease (risk ratio = 1.9) compared with those without material hardship.
Identifying Training and Workforce Needs
As part of the workshop, pediatric cancer survivors and family members whose children had succumbed to pediatric cancer provided the patient perspective on these issues.
These families had to face the difficult transition from in-hospital aggressive treatment to home hospice care, and the most common concerns were “the lack of support for pain, nutrition, and hydration issues,” Dr. Larson said. “We’ve addressed those in adult hospice care, but not sufficiently in pediatrics.”
While most of the more than 3,000 hospices in the United States consider accepting children, there is a dearth of freestanding pediatric inpatient hospice programs.5 More research into understanding the needs and preferences of patients and families, and into the costs and psychosocial benefits of end-of-life care, is needed.
Joanne Wolfe, MD, MPH, from the Dana-Farber Cancer Institute and chair of the workshop’s Planning Committee, addressed workforce development needs and current gaps in evidence in her presentation.
Contrary to policies of the American Academy of Pediatrics, Dr. Wolfe reported, access to a pediatric palliative care service is available in only 58 percent of Children’s Oncology Group member institutions, and while 69 percent of children’s hospitals have such a program, many are understaffed.6 Pediatric palliative care training opportunities for clinicians, however, are increasing, and evidence that these services improve outcomes – including shorter hospitalizations and fewer emergency department visits – is emerging.
At the same time, the NIH funds very few pediatric palliative care grants – underscoring the need to build the evidence base supporting funding for research and mentorships in this important area. Dr. Wolfe cited the Children’s Oncology Group’s initiative to reduce overall morbidity and mortality by decreasing acute and delayed treatment-related toxicities in all children with cancer.7 She also noted the Pediatric Palliative Care Research Network’s objectives to foster interdisciplinary research, increase the pool of highly qualified investigators, enable evidence-based pediatric palliative care practices, and promote a culture of inquiry in collaboration with patients and families.
The pediatric palliative care field can be moved forward, Dr. Wolfe concluded, by researching the following questions:
- What are the most effective communication techniques for improving patient and family outcomes?
- How can we best help patients and families make decisions for care that are centered on their personal values?
- How can we most effectively train clinicians (both younger and more established clinicians) in these techniques?
The need for training in pediatric palliative care is particularly great in the area of bereavement. Dr. Wolfe offered this example: When families receive a pediatric cancer diagnosis and are told that 80 percent of children with that diagnosis survive, the parents hear that their child has a 20 percent chance of dying and experience tremendous uncertainty.
“Bereavement care begins from the moment of diagnosis and continues past the lifespan of the child,” Dr. Wolfe said. “While all interdisciplinary clinicians who care for children with cancer should have basic pediatric palliative care knowledge, skills, behaviors, and attitudes, most physicians learn primarily through trial and error, and 71 percent of training programs lack a curriculum for pediatric palliative care.8 Nurses, as well, need specific training.”
While prolonged grief, isolation, behavioral health concerns, and economic and health decline persisting for years after a loss of a child are frequent, Dr. Weiner stated, many families do not seek or cannot find available support services.9 Dr. Weiner recommended the creation and implementation of financially sustainable continuity-of-care models extending for the whole family, at least through the second year post-loss. She asked, as well, for rigorous prospective research and quality improvement projects encompassing education and training, interdisciplinary care initiatives and interventions, advanced care planning models, and formulation and implementation of standards.
Finally, she said, “We must work collaboratively. If we work in our individual silos, we will get absolutely nowhere.” Also, she added, because there will never be enough resources to meet health-care needs, priorities need to be set.
“A top priority of the ACS,” Rebecca Kirch, JD, the ACS Director of Quality of Life and Survivorship told ASH Clinical News, “is to put quality of life on equal par with curative treatment in our strategic planning for both clinical and basic research.”
The common theme of the workshop: we need better – and earlier – education for families and providers. She added, “This workshop made it crystal clear that we have a long way to go in preserving quality of life and avoiding preventable suffering for both children and their families.” –By Walter Alexander
- Remke SS, Schermer MM. Team collaboration in pediatric palliative care. J Soc Work End Life Palliat Care. 2012;8(4):286-96.
- Maciasz RM, Arnold RM, Chu E, et al. Does it matter what you call it? A randomized trial of language used to describe palliative care services. Support Cancer Care. 2013;21(12):3411-9.
- Singh GK. Child mortality in the United States, 1935-2007: Large racial and socioeconomic disparities have persisted over time. A 75th Anniversary Publication. Health Resources and Services Administration, Maternal and Child Health Bureau. Rockville, MD: U.S. Department of Health and Human Services; 2010.
- Yoo JP, Slack KS, Holl JL. Material hardship and the physical health of school-aged children in low-income households. Am J Public Healht. 2009;99(5):829-36.
- Kassam A, Skiadaresis J, Alexander S, Wolfe J. Parent and clinician preferences for location of end-of-life care: home, hospital, or freestanding hospice? Pediatr Blood Cancer. 2014;61(5):859-64.
- Feudtner C, Womer J, Augustin R, et al. Pediatric palliative care programs in children’s hospitals: a cross-sectional national survey. Pediatrics. 2013;132(6):1063-70.
- Sung L, Zaoutis T, Ullrich NJ, et al. Children’s Oncology Group’s 2013 blueprint for research: cancer control and supportive care. Pediatr Blood Cancer. 2013;60(6):1027-30.
- Roth M, Wang D, Kim M, Moody K. An assessment of the current state of palliative care education in pediatric hematology/oncology fellowship training. Pediatr Blood Cancer. 2009;53(4):647-51.
- Rosenberg AR, Baker KS, Syrjala K, Wolfe J. Systematic review of psychosocial morbidities among bereaved parents of children with cancer. Pediatr Blood Cancer. 2012;58(4):503-12.