Designing and Implementing a Longitudinal Study of Children with Neurological, Genetic, or Metabolic Conditions: \u3cem\u3eCharting the Territory\u3c/em\u3e

Background: Children with progressive metabolic, neurological, or chromosomal conditions and their families anticipate an unknown lifespan, endure unstable and often painful symptoms, and cope with erratic emotional and spiritual crises as the condition progresses along an uncertain trajectory towards death. Much is known about the genetics and pathophysiology of these diseases, but very little has been documented about the trajectory of symptoms for children with these conditions or the associated experience of their families. A longitudinal study design will help to close this gap in knowledge. Methods/Design: Charting the Territory is a longitudinal descriptive, correlational study currently underway with children 0–19 years who are diagnosed with progressive neurological, metabolic, or chromosomal conditions and their families. The purpose of the study is to determine and document the clinical progression of the condition and the associated bio psychosocial spiritual experiences of these parents and siblings age 7–18 years. Approximately 300 families, both newly diagnosed children and those with established conditions, are being recruited in six Canadian cities. Children and their families are being followed for a minimum of 18 months, depending on when they enroll in the study. Family data collection will continue after the child’s death if the child dies during the study period. Data collection includes monthly parental assessment of the child’s symptoms; an annual functional assessment of the child; and completion of established instruments every 6 months by parents to assess family functioning, marital satisfaction, health status, anxiety, depression, stress, burden, grief, spirituality, and growth, and by siblings to assess coping and health. Impact of participation on parents is assessed after 1 year and at the end of the study. Chart reviews are conducted at enrollment and at the conclusion of the study or at the time of the child’s death. Discussion: Knowledge developed from this study will provide some of the first ever detailed descriptions of the clinical symptom trajectory of these non-curable progressive conditions and the bio-psychosocial spiritual aspects for families, from diagnosis through bereavement. Information about developing and implementing this study may be useful to other researchers who are interested in designing a longitudinal study

Up against the System: A Case Study of Young Adult Perspectives Transitioning from Pediatric Palliative Care

Advances in pediatric care have not provided the interdisciplinary support services required by those young adults with pediatric life-threatening conditions (pedLTCs) who live beyond childhood but have limited expectations to live past early adulthood. These young adults, the first generation to live into adulthood, face multiple challenges transitioning from a plethora of pediatric palliative services to scant adult health services. In a case study, using an innovative bulletin board focus group, we describe the complex interplay of the health, education, and social service sectors in this transition. Our descriptions include system deficits and strengths and the young adults' resilience and coping strategies to overcome those deficits and move forward with their lives. Young adults with pedLTC need knowledgeable providers, coordinated and accessible services, being respected and valued, and services and supports that promote independence. We recommend implementation of multidisciplinary solutions that are focused on young adult priorities to ensure seamless access to resources to support these young adults' health, educational, vocational, and social goals. The input and voice of young adults in the development of these services are imperative to ensure that multisystem services support their needs and life goals

The boy who refused an IV: a case report of subcutaneous clodronate for bone pain in a child with Ewing Sarcoma

BACKGROUND: Bone pain in malignancy can be challenging to treat. Bisphosphonates have been found to be useful in adults with bone pain, but there are no reports of their use in children for this indication. In pediatric palliative medicine there are hurdles in translating knowledge gained primarily in adult studies into application in children. Obstacles exist in initially determining whether the evidence supports using a drug in children, and once a drug is chosen, then determining the optimal route of delivery. There is very little data to guide pediatric practitioners in this situation. CASE PRESENTATION: A 9 year old boy with disseminated Ewing Sarcoma presented with extremity pain not responsive to a combination of opiates, gabapentin and non-steroidal anti-inflammatory drugs. Clodronate, a bisphosphonate, was added to the regimen to treat bone pain. It was given subcutaneously every 4 weeks with a good response and no side effects. CONCLUSION: This case report describes the use of a bisphosphonate, clodronate, given subcutaneously to a child with Ewing sarcoma with effective relief of bone pain. It describes how the care team encountered the challenges inherent in translating adult therapy into a pediatric regimen. Furthermore the report details how a regimen was developed to address this child's concerns regarding medication administration. Further effort needs to be made at finding solutions to address the lack of good evidence for pediatric palliative therapies

Protocol: Evaluating the impact of a nation-wide train-the-trainer educational initiative to enhance the quality of palliative care for children with cancer

Background: There are identified gaps in the care provided to children with cancer based on the self-identified lack of education for health care professionals in pediatric palliative care and in the perceptions of bereaved parents who describe suboptimal care. In order to address these gaps, we will implement and evaluate a national roll-out of Education in Palliative and End-of-Life Care for Pediatrics (EPEC (R)-Pediatrics), using a 'Train-the-Trainer' model.Methods/design: In this study we are using a pre-post-test design and an integrated knowledge translation approach to assess the impact of the educational roll-out in four areas: 1) self-assessed knowledge of health professionals; 2) knowledge dissemination outcomes; 3) practice change outcomes; and 4) quality of palliative care. The quality of palliative care will be assessed using data from three sources: a) parent and child surveys about symptoms, quality of life and care provided; b) health record reviews of deceased patients; and c) bereaved parent surveys about end-of-life and bereavement care. After being trained in EPEC (R)-Pediatrics, 'Master Facilitators' will train 'Regional Teams' affiliated with 16 pediatric oncology programs in Canada. Each team will consist of three to five health professionals representing oncology, palliative care, and the community. Each team member will complete online modules and attend one of two face-to-face conferences, where they will receive training and materials to teach the EPEC (R)-Pediatrics curriculum to 'End-Users' in their region. Regional Teams will also choose a Tailored Implementation of Practice Standards (TIPS) Kit to guide implementation of a quality improvement project in their region; support will be provided via quarterly meetings with Co-Leads and via a listserv and webinars with other teams.Discussion: Through this study we aim to raise the level of pediatric palliative care education amongst health care professionals in Canada. Our study will be a significant step forward in evaluation of the impact of EPEC (R)-Pediatrics both on dissemination outcomes and on care quality at a national level. Based on the anticipated success of our project we hope to expand the EPEC (R)-Pediatrics roll-out to health professionals who care for children with non-oncological life-threatening conditions

Pediatric Palliative Care for Children with Progressive Non-Malignant Diseases

A substantial number of children cared for by pediatric palliative care physicians have progressive non-malignant conditions. Some elements of their care overlap with care for children with cancer while other elements, especially prognosis and trajectory, have nuanced differences. This article reviews the population, physical-emotional and social concerns, and trajectory.Other UBCReviewedFacult

An agenda to develop Pediatric Palliative care programs to serve children with life-threatening and life-limiting conditions in the Gulf Cooperation Council countries

Medical advances have increased the number of children living with life-threatening/life-limiting illnesses worldwide, including in Gulf Cooperation Council (GCC) countries. Pediatric palliative care (PPC) is a relatively young subspecialty that cares for children with life-threatening/life-limiting illnesses and their families. PPC aims to alleviate physical, psychological, and social distress in children with life-threatening/life-limiting illnesses and their families and improve their quality of life. PPC is an essential service that should be implemented in all nations, as it is a human right. Moreover, a core value of PPC services is to alleviate children’s suffering, irrespective of cure availability. Hence, the global consensus on palliative services must be universal and include developing countries with limited resources. While PPC services are growing internationally, the GCC countries have yet to implement these valuable services in the region. This work aims to define the local base information important to facilitating the PPC program. We explored and identified the information vital for establishing a successful program, which was then categorized and mapped into subgroups. In doing so, we outline a roadmap to facilitate the smooth introduction of PPC in GCC countries to benefit the lives of children with life-limiting illnesses

Integrating parent-partners as research team members : Creating the role of Family Liaison

In caring for children with serious illness the clinician’s approach is always family centred. As such our clinical research team has continuously involved families in our studies beyond their role as participants in our research. A Family Advisory Group reviews our project proposals and protocols and participating families provide feedback on their experience after graduating from one of our studies. However, in learning about patient-oriented research strategies through our engagement with the CHILD-BRIGHT network, we have come to realize that in order to fully partner with families, specifically parents of children participating in our research, we need to have an embedded team member who acts as a parent-partner in all our endeavours. Therefore we have created the role of Family Liaison as a fixed part of our team makeup and hired Laesa Kim to fill this position. Laesa’s job goes beyond that of advisor for two important reasons: She holds a paid position within our staff group and her contribution to our program supersedes that which can be expected of volunteer committee members. The Family Liaison is a bridge between researchers and participants, not just in the connections she makes when translating the research objectives and protocols to participants, but also in bringing a parent or patient perspective to every stage of study design and roll out. Along with Laesa’s specific job description comes expectations of rather unique credentials to successfully fill the role. Laesa shares in the lived experience of our participants as the mother of a medically complex child, with years of learning to understand and navigate health care systems. Her experience coupled with a disposition for connecting with health care providers as well as fellow parents of children needing health care while understanding and advocating for the specific needs of this population, positions her perfectly to fill this role. We will describe the value of the Family Liaison within our team as an integral part of our research program while showing the power of investing in patient-oriented research strategies to advance our projects.Medicine, Faculty ofNon UBCPediatrics, Department ofUnreviewedFacultyResearcherOthe