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sick child in palliative care hospital bed

Children’s National Research Institute receives NIH grant for palliative care study

sick child in palliative care hospital bed

A new NIH grant will support the first study that examines palliative care needs in pediatric rare disease community.

The National Institute of Health (NIH) has awarded $500,875 to the Children’s National Research Institute (CNRI), the academic arm of Children’s National Hospital, to support a new study examining the palliative care needs of children living with rare genetic diseases.

This is the first study of families of children with genetic and metabolic conditions, termed collectively as rare diseases, that is designed to intervene to support the well-being of family caregivers and create advance care plans for future medical decision making. In the United States, a rare disease is defined as a particular condition affecting fewer than 200,000 people. Pediatric patients with rare diseases experience high mortality rates, with 30 percent not living to see their fifth birthday.

“Children with ultra-rare or complex rare disorders are routinely excluded from research studies because of their conditions, creating a significant health disparity. Surveys show that families of children with rare diseases are adversely impacted by lack of easy access to peer and psychological support,” says Maureen Lyon, Ph.D., Clinical Health Psychologist and Professor of Pediatrics at the CNRI and principal investigator on the project. “This study will examine the palliative care needs of family caregivers of children with rare genetic disorders and advance care planning intervention, which will ultimately help facilitate discussions about future medical care choices that families are likely to be asked to make for their child.”

Although greatly needed, there are few empirically validated interventions to address these issues Currently, there is only one intervention described for families of children with rare diseases — a Swedish residential, competence program — which focuses on active coping. However, this intervention does not address pediatric advance care planning, a critical aspect of palliative care.

Lyon adds that the major benefit of this proposed project will be filling the gap in knowledge about what family caregivers of medically fragile children with rare diseases want with respect to palliative care. In the United States, these families are expected to provide a level of care that, until a few decades ago, was reserved for hospitals.

Maureen E Lyon

Maureen Lyon, Ph.D., Clinical Health Psychologist and Professor of Pediatrics at the CNRI and principal investigator on the project.

“Our hope is that this study will provide a structured model for facilitating family decisions about end-of-life care, for those families who do not have the good fortune to have children who have the capacity to share in decision-making,” Lyon says.

In addition to bridging the knowledge gap regarding palliative care in rare disease patients, the study will also help inform current clinical, ethical and policy discussions, as well as the legal issues in a variety of areas, such as the debate surrounding advocacy, particularly for those children with impairments in physical function.

“We look forward to the results of this study,” said Marshall Summar, M.D., director of the Rare Disease Institute and division chief, Genetics and Metabolism at Children’s National Hospital. “As a leader in rare disease care, we continually examine how we can improve care and support for our patient families at our clinic and want to share our findings with others engaged in caring for rare disease patients. Because rare diseases can be life limiting in some cases, we need to learn all we can about how best to care and support a patient and family as they prepare for a potential transition to palliative care.”

All research at Children’s National Hospital is conducted through the CNRI, including translational, clinical and community studies. The CNRI also oversees the educational activities and academic affairs of the hospital and the Department of Pediatrics at the George Washington University School of Medicine and Health Sciences, frequently partnering with many other research institutions regionally and nationally. CNRI conducts and promotes translational and clinical medical research and education programs within Children’s National Hospital that lead to improved understanding, prevention, treatment and care of childhood diseases.

doctor and patient filling out paperwork

How advance care planning can improve life in a pandemic and beyond

doctor and patient filling out paperwork

New research, published in AIDS and Behavior, shows the effectiveness of an Advance Care Planning model developed through participatory research with adolescents in improving palliative care among adult people living with HIV (PLWH).

Since the beginning of the COVID-19 pandemic, there has been a dramatic increase in advance care planning (ACP) and the creation of advance directives, also known as living wills, in the United States. New research, published in AIDS and Behavior, shows the effectiveness of an ACP model developed through participatory research with adolescents in improving palliative care among adult people living with HIV (PLWH).

These findings demonstrate that ACP positively contributes to the palliative care of adult PLWH by relieving suffering and maximizing quality of life. The intervention was based on the FAmily CEntered (FACE) Advance Care Model, which was developed and tested by principal investigator Maureen E. Lyon, Ph.D., and her colleagues.

Dr. Lyon’s team used this model successfully with adolescents living with HIV as part of five-year, five-site trial that included Children’s National Hospital. The trial was co-funded by the National Institutes of Health and National Institute of Nursing Research. The success of that study was parlayed into a new five-year study testing a slightly modified ACP intervention in adults, with Children’s National serving as the coordinating center. “The adolescents showed us the way,” says Dr. Lyon.

The paper details the findings of a longitudinal, two arm, randomized controlled clinical trial examining whether an ACP intervention aimed at adult PLWH and their families correlated with higher congruence in treatment preferences, as well as higher congruence over time. Patient-surrogate dyads were randomized to an ACP intervention arm or an active control arm at a 2:1 ratio (86 intervention dyads and 43 control dyads at 18-month follow up), due to prior demonstrated benefit of ACP.

The ACP intervention consisted of two 60-minute, patient-focused sessions. During session 1, Respecting Choices Next Steps® ACP Conversation, both patients and their surrogate decision-makers focused on the patients’ understanding of HIV, experience of symptoms, fears, hopes and worries. Next, a patient’s treatment preferences were explored via the Statement of Treatment Preferences (SoTP), which became a part of the patient’s electronic health record (EHR). Surrogates were questioned on their comprehension and willingness to comply with the patient’s wishes. Session 1 was acknowledged as the beginning of a conversation, and continued conversation between the dyad was encouraged.

Session 2, Five Wishes©, involved a facilitator guiding the dyad through a Five Wishes© advance directive. Session 2 resulted in legal documentation of a patient’s preferences in five specific areas: The patient’s preferred health care decision-maker, the kind of medical treatment the patient wants, how comfortable the patient wants to be, how the patient wants people to treat him/her and what the patient wants loved ones to know. The patient, surrogate and treating physicians all received a copy, and a copy was also submitted to the patient’s EHR.

Dyads in the control arm participated in two 60-minute sessions entitled Developmental or Relationship History (excluding any medical questions) and Nutrition & Exercise.

The researchers then assessed treatment preference congruence for each patient-surrogate dyad by presenting them with five different hypothetical scenarios. After the first session, congruence across all scenarios was significantly higher among ACP intervention dyads compared to control dyads. ACP patients were also significantly more likely to give their surrogates leeway in treatment decision making compared to control patients.

Compared to control dyads, ACP dyads were significantly more likely to maintain High → High congruence transition and significantly less likely to experience Low → Low congruence transition as measured from immediately post-intervention to 12-months post-intervention. The only two cases of Low → High congruence transition occurred in the intervention arm. Of note, ACP surrogates accurately reported on changes in patient preferences over one year, showing the positive impact of early conversation on longitudinal congruence.

Dr. Lyon hopes these results will encourage people to talk to their loved ones as soon as possible about ACP, not only during the current pandemic but into the future. “People can use what’s happening in the news as a trigger to begin these conversations,” she says. “The 1990 Patient Self-Determination Act (PSDA) encourages persons of all ages– including children and their parents– to decide the type and extent of medical care they want to accept or refuse if they become unable to make those decisions due to illness. Our research shows conversations matter.”

The original research paper, “Effect of FAmily CEntered (FACE®)Advance Care Planning on Longitudinal Congruence in End-of-Life Treatment Preferences: A Randomized Clinical Trial,” was recently published in AIDS and Behavior. Dr. Maureen E. Lyon, Ph.D., FABPP, of the Center for Translational Research/Children’s Research Institute, was the principal investigator of the trial and a co-senior of the paper.

Telemedicine

A rare prescription: Providing children with palliative care

Telemedicine

A pilot program at Children’s National enabled parents of children with extremely rare diseases to receive in-person or virtual health consultations with a trained provider.

Pediatric advance care planning (pACP) and making complex  medical decisions is especially difficult for parents of children with extremely rare diseases. Imagine if your child is the only person in the world with a rare disease that may limit basic functions: eating, breathing, walking and talking. Now, imagine you are presented with two scenarios: Experiment with a new drug to see if it improves your child’s conditions or plan for near-future, end-of-life care.

While these types of difficult decisions for parents of children with rare diseases are common, a new counseling model, based on a four-session pilot program conducted at Children’s National, aims to ease this process by providing parents with a comprehensive support plan.

On Oct. 15 and 16, Maureen Lyon, Ph.D., a clinical psychologist at Children’s National and a professor of pediatrics at the George Washington University School of Medicine and Health Sciences, will present “Living on the Precipice: The Journey of Children with Rare Diseases and Their Families” at a poster session at the National Organization for Rare Disorders’ Rare Disease and Orphan Products Breakthrough Summit at the Marriott Wardman Park in Washington.

Dr. Lyon will highlight key take-home points she observed during the pilot program:

  • Background: Eight families were recruited for the pilot program and seven enrolled. Six completed the four-session program, which was spread out over two months.
    • All parents were mothers, but two fathers joined for the goal-planning care conversation sessions. Some families brought their children to visits.
    • Five parents were married and two were single.
    • Four families identified as Caucasian, three families identified as African American, and one family identified as American Indian or Alaska Native.
  • Visits: About half of the families – three – attended the sessions at Children’s National. Four used the telemedicine option. A research nurse, clinical psychologist and advanced practice nurse participated in the 60- to 90-minute sessions.
  • Plans: The families discussed basic palliative care needs, such as comprehensive care coordination, which is highly individualized, before discussing their goals of care. After their needs and goals were discussed, the families created advance care plans to guide them during a medical crisis.
  • Results: Out of the six parents who completed the study, the mean positive caregiver appraisal score increased from 4.5. To 4.7, mean family well-being increased from 3.9 to 4.1, and the mean score for meaning and peace increased from 21.4 to 23.3. The scores were calculated by using the Carer Support Needs Assessment Tool (CSNAT) during the assessment and with modified protocols to assess quality of life and caregiver appraisal after the intervention.
Maureen Lyon

“The goal of palliative care is to optimize quality of life for children with life-threatening illnesses and their families by anticipating, preventing and treating suffering in all its forms,” explains Maureen Lyon, Ph.D. “This is delivered throughout illness and addresses physical, intellectual, emotional, social and spiritual needs.”

“These sessions increased a family’s sense of overall well-being,” says Jessica Thompkins, B.S.N., R.N., C.P.N., a research nurse coordinator with the FAmily CEntered Advanced Care Planning Team (FACE) and a co-author of the poster. “The families felt better just by knowing that they had time scheduled each week to connect with a trained medical provider to discuss a range for options they need as a caregiver, from everyday care at home to long-term health care planning at the hospital.”

The top-rated support need identified by all parents, according to the survey: “Knowing what to expect in the future when caring for their children.”

“The goal of palliative care is to optimize quality of life for children with life-threatening illnesses and their families by anticipating, preventing and treating suffering in all its forms,” says Dr. Lyon. “This is delivered throughout illness and addresses physical, intellectual, emotional, social and spiritual needs.”

The researchers would like to use this pilot to partner with other medical centers to create an evidence-based template to support the palliative care needs of family caregivers who have children with life-limiting rare diseases. Their goal is to improve a family caregiver’s quality of life, over time, and increase the completion and documentation of advance care plans for children of all ethnic and racial groups.

Rare diseases are defined as a disease that affects fewer than 200,000 people in the U.S. Extremely rare diseases, including those observed in this pilot, may affect just one or a few people in the world.

The rare disease pilot program is based on previous pACP models with adolescent HIV and pediatric cancer populations.

Additional poster authors include Jichuan Wang, Ph.D., Karen Fratantoni, M.D., M.P.H., Kate Detwiler, Ph.D., Yao Cheng, M.S., and Marshall Summar, M.D.