Assessment of the relationship between disease progression and goals of care by individuals with Duchenne muscular dystrophy and their caregivers

Abstract

Introduction/Aims Most patients with Duchenne muscular dystrophy (DMD) in the US are diagnosed at about age 5 years. Some adolescents and young adults (AYAs) with DMD are now living into their fourth decade, yet AYAs and caregivers are frequently unprepared to address changes in goals of care due to disease progression. The hypothesis‐generating research question was how AYAs with DMD and their caregivers understand the relationship between physical changes and the need to change goals of care. Methods Grounded theory design using data from N = 30 semi‐structured interviews (n = 13 AYA; n = 17 caregivers) from two sites. Results AYAs with DMD frequently defer considering and/or reconsidering goals of care based on (1) delays in diagnosis; (2) gradual, rather than episodic, disease progression; and (3) orientation to living in the present. Desire for autonomy motivates advance care planning and end‐of‐life treatment preferences for some. Discussion Routine inquiry into AYA and caregiver goals for living may normalize goals of care conversations, maximizing patients' ability to process information, reflect on preferences, and articulate wishes. Discussing present‐day goals and abilities may invite conversation about future preferences. Framing conversations in terms of AYA autonomy may increase motivation to engage in such discussions.

Palliative care in Duchenne muscular dystrophy: Goals of care discussions and beyond

“…The study also unraveled the mistrust in the health care system, as described by the caregivers, and fewer provider led goals of care discussions 9 . It is reasonable to consider that this could related in part to the timing of goals of care discussions, the introduction to the palliative care team, consistency of the service, and discussions throughout the care of the individual.…”

“…It is important to note that only 44% of the participants in the study by Grossoehme et al used palliative care services in spite of the strong representation of palliative care team at their centers 9 . Studies have shown that providers′ awareness, discomfort, and uncertainty can influence the discussions around goals of care and advance care planning 3,9 . Physicians caring for adult patients generally defer end‐of‐life conversations with their patients due to several reasons, including inadequate preparation and training for facilitating difficult conversations, personal discomfort with dying and death, medicolegal concerns, and fear of causing pain and bearing bad news.…”

“…Grossoehme et al 9 interviewed 13 individuals with DMD, aged 11 to 33 years, at different disease stages together with 17 caregivers. Their findings revealed that the adolescents and young adults with DMD were unlikely to have explicit goals of care discussions with their providers and generally deferred such evaluation because of delays in diagnoses and a gradual disease progression.…”

“…Some confounders to discussing goals of care and advance care planning in children with DMD could be the high emotional impact of such decisions, involvement of multiple stakeholders such as parents, families, and school in the care of children, and the inability of some patients to consent 10 . Grossoehme et al included males with DMD aged 11 years and older 9 ; however, differences, if any, in responses between younger and older age groups, were not examined. One could envision changes in goals of care over time as the disease progresses and the need to tailor discussions based on such needs.…”

“…The patient‐family‐provider relationship is critical to initiate and continue conversations regarding goals of care and advance care planning. It is important to note that only 44% of the participants in the study by Grossoehme et al used palliative care services in spite of the strong representation of palliative care team at their centers 9 . Studies have shown that providers′ awareness, discomfort, and uncertainty can influence the discussions around goals of care and advance care planning 3,9 .…”