Priority Areas for Research

The Institute of Medicine (IOM) report on improving palliative and end-of-life care for children and their families highlighted the lack of research and systematic data on which to base their recommendations.¹ The authors identified a critical need for research in all aspects of pediatric palliative and end-of-life care and recommended focusing research activities on “the effectiveness of: clinical interventions including symptom management; methods for improving communication and decision-making; innovative arrangements for delivering, coordinating, and evaluating care, including interdisciplinary care teams and quality improvement strategies, and different approaches to bereavement care.”¹

Others also have worked to identify research priorities. The four top questions identified from a Delphi survey with pediatric palliative care researchers and clinicians in Canada were: What matters most for patients and parents receiving pediatric palliative care? What are the bereavement needs of families in pediatric palliative care? What are the best practice standards in pain and symptom management? What are effective strategies to alleviate suffering at the end of life?² In pediatric oncology, the areas of palliative and end-of-life care have been identified as research priorities. Recommendations include a focus on: decision making and communication, characteristics of death and profiles of bereaved family members and health professionals, trajectories of dying, a comparison between the care provided and the care that was desired by families, financial cost of death after cancer, and outcomes from symptom management and bereavement support.^3,⁴

The identified priority areas are broad enough to leave opportunities for exploring a wide range of specific research questions. Experience suggests that researchers need to identify the questions that keep them up at night. The questions need to be exciting and interesting enough to researchers so that they will keep going despite the inevitable challenges. Given the current lack of research in the field, it is likely that initial studies are needed to develop appropriate indicators or measures and to more fully understand the experiences and needs of a particular population. Initial studies should be completed before the most burning questions can be answered with designs such as randomized controlled trials (RCT) of interventions.

It has historically been challenging to obtain funding for pediatric palliative care research, partly because the major government funding agencies have agendas and limited resources that may not be supportive of this type of research. In Canada, the government recognized the importance of creating a health agenda that incorporated palliative care into the spectrum of care provided to its citizens. Therefore, in 2004 Canada took a leadership role in funding palliative care research through the CIHR, which is one of the three major research funding agencies in the country. Palliative care was identified as a priority and a targeted competition was held to create interdisciplinary teams (IDT) to facilitate the development of programs of research. Our own team was funded as a result of this competition and we remain the largest funded team in pediatric palliative care in Canada. However, we work in collaboration with colleagues from across the country who also may have their own more local teams. Subsequent funding for our work was obtained through operating fund competitions, but no further resources have been allocated specifically for palliative care let alone for pediatric palliative care. In the United States, the seminal work of the IOM on Approaching Death, published in 1997, explicated gaps in scientific knowledge. In response, the director of the National Institutes of Health (NIH) designated the National Institute for Nursing Research (NINR) to serve as the lead Institute at NIH for end-of-life research. To coordinate this effort, NINR has established the Office of Research on End-of-Life Science and Palliative Care, Investigator Training, and Education (OEPC) and offered numerous Program Announcements (PA) that solicit research pertaining to end-of-life care. The one PA that specifically addressed pediatrics (Improving Care for Dying Children and Their Families – PA 04-57) is inactive although pediatric proposals can be submitted in response to other PAs.

Methodological Challenges

When considering the development of a study in the area of pediatric palliative care, researchers need to be aware of potential impediments to their success. Some of the methodological challenges include research design, outcome measures, recruitment and sampling, and research teams. While ethical challenges are alluded to in this section, they will be discussed more fully later in the chapter.

Research design

Though the gold standard for research remains the RCT with the highest level of evidence being systematic reviews of multiple RCTs, primarily qualitative designs and descriptive methods may be more appropriate to address the research priorities and knowledge in pediatric palliative care.¹ Further, RCTs are difficult to conduct in both adult and pediatric palliative care. The heterogeneity of diseases, illness trajectories, and ages of participants are difficult to accommodate in a design that requires a homogeneous group of participants and testing of simple standardized interventions.⁵ Bensink et al.⁶ made two attempts to use an RCT to test videophones as a support for families receiving pediatric palliative care, but all families approached refused to participate. When the research team changed the design to an acceptability study, without randomization or any measures to be completed by the family, they had a participation rate of more than 90%. These researchers suggested that a clinical trial is overwhelming for families when initiated at the transition to palliative care. Kane, Hellsten, and Coldsmith⁷ discussed the interpersonal relationships at the heart of palliative care and the need for understanding complex interactions among humans in order to identify the social and spiritual interventions needed to address human suffering. It may be that these interactions are not amenable to the quantified measurement and standardization needed for an RCT. Levels of evidence that may be more relevant for palliative care include quasi-experimental studies, qualitative studies, and consensus opinions of palliative care experts.⁸

Longitudinal studies in pediatric palliative care have been uncommon, but are useful in expanding the evidence base. Researchers who embark on longitudinal work need to design their studies in ways that will minimize attrition. Though families in pediatric palliative care often participate in research as a way of helping others, even when they find it challenging practically or emotionally,⁹ researchers need to pay attention to keeping families involved in a study over time. A number of effective strategies have been reported in the literature,¹⁰^–¹³ and we are using some of them in our longitudinal study with families where a child has a progressive metabolic, neurological, or chromosomal condition and in our parent caregiver study. Development of a solid relationship with families is crucial. An initial face-to-face visit, telephone contact at prescribed intervals, and letters to thank families for their participation are useful for building and maintaining relationships. Continuity of research assistants or other personnel involved in the study is important and will contribute to the trusting relationship that is needed to help participants feel more connected to and interested in the study over time. Efforts should be made to respect families’ time and to recognize the value of their contribution. Researchers should schedule data collection at the family’s convenience and should regularly express verbal and written appreciation for their participation in the study. In addition, providing a monetary or other gift as a token of appreciation along with a letter thanking them for participation may encourage families to remain in a study, because families feel valued when their time and effort is recognized. Strategies that limit attrition will result in the type of high quality data needed to provide solid, evidence-based care.

Parent Participation

Parents in all of the studies we have undertaken as a team have given us feedback about what they see as being the value of their participation. In some cases this feedback was solicited and in others it was offered by the parents without our invitation. Our parent caregiver study has provided several touching examples. Many of the 273 participants took the time to do more than simply answer a survey. They wrote comments on the forms or sent letters to accompany their returning questionnaires. One mother wrote five pages about her story and added questions that she thought should be asked in the research. Another mother wrote a note about her depression, her partner leaving her, and the need to keep moving forward with a smile for her daughter who did not ask to be born as she was. Other parents have called the toll-free number to give us updates on their situations or thank us for the research. Some of our studies include or are solely comprised of qualitative interviews. Parents have commented on their desire to participate in research in order to make the difficult situations that they have experienced less difficult for someone else. Many parents want to participate in research. They also want the opportunity to decide for themselves if a study is right for them.

Outcome measures

Science can progress no further than the measurement of its key variables.

— ATTRIBUTED TO J. NUNNALLY, 1994

Unfortunately, in pediatric palliative care the most important variables or outcomes to be measured are not well defined, and there are few valid measures. Outcomes usually involve a change in the health status of the individual, but also can include increased knowledge of health conditions, changes in behavior related to health, or patient and family satisfaction with the care received and its outcomes.¹⁴ The expected outcome for the child in pediatric palliative care is death, which cannot be changed. However, one can improve the quality of that death¹⁵ and the family’s satisfaction with the care provided. One also may be able to improve health outcomes for the family including reduction in the incidence and severity of depression, anxiety, post traumatic stress, guilt, and complicated grief.

Research in adult palliative care is more advanced than that of pediatrics; however, that field of research is still relatively recent and similar issues about the difficulty of identifying and measuring important outcomes have been reported. There has been some research in adult palliative care asking patients and family members to identify components of quality care and of a good death.¹⁶^–¹⁸ A number of measures have been developed and used with adult palliative care patients and their families (Mularski et al¹⁹). However, for multiple reasons, including the developmental level of children, the variables and/or outcomes and measures used in research with adults cannot simply be taken and applied to children. For example, one outcome deemed indicative of quality care for adults is that death occurs at home.²⁰ In children, this outcome may not be as indicative of quality end-of-life care. Dussel, Kreicbergs, Hilden et al.²¹ found that the opportunity to plan the location of death was more important to parents than where the death actually occurred. Satisfaction is another outcome that has been identified as a component of quality care.¹⁴ However, satisfaction has not been well conceptualized in the literature and may be influenced by demographic variables.²² In end-of-life care, there is a concern that families have very low levels of expectation and therefore are easily satisfied with care.²³ In pediatric palliative care, there is evidence that parents tend to report high levels of satisfaction even when high levels of distressing symptoms are reported.²⁴ Thus, satisfaction may not be a good indicator of the actual quality of care.

Given that research in adult palliative care is more prevalent than in pediatric palliative care, pediatric researchers can learn from their adult-researching counterparts about possible instruments, methods, and research designs that might be useful. However, this information must be critically examined to see how instruments might need to be altered to fit the pediatric population. Any alterations must be based on clinical expertise and done with an intimate understanding of the population to be studied. This speaks to the need for researchers and clinicians to work together to conduct appropriate research in pediatric palliative care.

Given that there is no single instrument encompassing all of the dimensions of care in pediatric palliative care, researchers must choose among several to measure the specific domains of interest. Those domains will include tools that are specific to areas such as pain and other symptoms, the child’s emotional well-being, family issues, and spiritual support. There is no single instrument in these fields that answers all questions. For example, in studying families one may want to know about marriage and relationship stability, personal growth, family stress and functioning, or the health of individuals.

The Team for Research with Adolescents and Children in Palliation and Grief (TRAC-PG) at Toronto’s Hospital for Sick Children (SickKids) has compiled a list of instruments that some of its members have used with children, adolescents, and adults in pediatric palliative and end-of-life care research. This list is available on TRAC-PG’s website at www.tracpg.ca/index.pho/researchers/instruments_measures_scales, but caution should be used when reviewing these instruments for future research. As previously noted, few measures have been specifically validated for use in pediatric palliative care. Many of the measures have also been used with parents in the months or years after their child’s death.

Recruitment and sampling

There are several challenges in identifying, accessing, and recruiting a sample to take part in a study related to pediatric palliative care. The number of children who die each year is small relative to the number of adults. However, small numbers at any one center may make it difficult to access a large enough sample to fit with particular research designs. As well, many children have rare illnesses where there may be very few in one country or around the world, let alone within a particular institution. Two possible methods to increase the size of the accessible population are to use disease groupings rather than single diseases or to conduct multicenter research.

Disease Groupings

The Association for Children with Life Threatening or Terminal Conditions and their Families (ACT) and the Royal College of Paediatrics and Child Health identified four distinct groups, or quadrants, of conditions (Table 10-1) seen in children who may not live until adulthood and who require palliative care.²⁵ Though there is some fluidity regarding which quadrant a condition fits into, conditions in a quadrant are similar to each other in terms of the expected trajectory and time course of the illness. Therefore, when planning a study, rather than only including children with a particular neurodegenerative condition, it may be possible, depending on the type of research questions being asked, to increase the available population by including children with any condition that fits into Quadrant 3.

TABLE 10-1 Association for Children with Life-Threatening or Terminal Conditions and Their Families and the Royal College of Paediatrics and Child Health Quadrants of Conditions That May Require Palliative Care

Quadrant 1	Quadrant 2
Conditions that can be cured but have a possibility of death (e.g., cancer)	Conditions requiring intensive medical therapy but are ultimately terminal (e.g., cystic fibrosis, HIV/AIDS)

Quadrant 3	Quadrant 4
Conditions that have no cure but whose symptoms can be managed (e.g., neurodegenerative, metabolic disease)	Severe neurological impairments where complications may lead to early death (e.g., anoxic brain injury)

Multicenter Research

Recruiting participants through a number of centers is another way to increase the size of the research population and to broaden the racial, ethnic, and geographic diversity of the sample.²⁶ However, multicenter research may raise other challenges. The need for ethical approval at each site may add an additional six months or more to the time required to start a study.²⁷ Additional associated costs must be factored in to grant proposals. A great deal of negotiation may be needed to ensure that recruitment strategies at each site will meet the requirements of local ethics boards, but are similar enough to each other to not threaten the validity of the study findings.

Participatory action research (PAR) is one method that may help to overcome some recruitment challenges. PAR involves extensive collaboration between the researchers and those affected by the issue that is being studied.^28,²⁹ In pediatric palliative care research, PAR has been used to some extent with both clinicians and families. For example, one study involved collaboration between a group of researchers and a group of clinicians who were providing hospice care. This collaboration included design and implementation of a project to evaluate the care provided by the clinical team. Trust between the two groups and active involvement of the clinicians in all aspects of the research facilitated recruitment of families as participants, as well as increased the uptake of study findings by the clinician group.³⁰ In a similar project designed to evaluate a hospice program, the research team used PAR with the families. This collaboration ensured project objectives and methods used for data collection were sensitive to family needs and issues and were meaningful to participants.³¹

The positive outcomes from such studies suggest that using PAR with clinicians and families in pediatric palliative care may assist with some of the challenges to recruitment. Even if all of the principles of PAR are not followed, some evidence that a research protocol has been developed with direct input of families may be helpful in receiving ethics approval as well as funding for pediatric palliative care studies. We have found that including families’ input early as we design our studies, for example, asking families about the suitability of the potential instruments or piloting the accrual process with families, has contributed to an increased success rate for our funding proposals. It has also smoothed the process of conducting research, from receiving funding to ethical approval to recruitment.

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