Abstract
Aims: The unpredictability and life-limiting nature of chronic, pediatric neuromuscular disorders raise significant physical, psychosocial, spiritual, and social difficulties for affected individuals and their families, and create special challenges for the healthcare team. Drawing on the principles of pediatric palliative care (PPC), we explored these challenges in the context of Duchenne Muscular Dystrophy and Spinal Muscular Atrophy-Type 1 and identified opportunities for and barriers to the integration of PPC worldwide. Methods: We convened an international interdisciplinary consortium of 53 clinicians, families, and advocates from seven countries. Facilitated small groups explored the key aspects of palliative care integration into the two diseases. Results: Participants identified four transition points in the disease trajectories (i.e., peri-diagnostic, treatment decision making, life-threatening events, and end of life) and opportunities for PPC at each phase. Interpretation: Patients, families, and clinicians can openly discuss their challenges and create a shared vision of quality care for children with NMDs, and that opportunities exist to integrate PPC at each point of transition in the progression of NMDs.
Highlights
Spinal Muscular Atrophy Type I (SMA-1) and Duchenne Muscular Dystrophy (DMD) are the two most common inherited pediatric lifethreatening neuromuscular disorders (NMDs) worldwide [1,2]
Advances in respiratory and cardiac management [4,5] allow DMD patients to live into their 20’s, but their burden of disease is high, commensurate with the amount of weakness, and they o en undergo major operations or require extended hospitalizations for routine childhood illnesses [6]. In both SMA-1 and DMD, the ability of children to comprehend their illness depends on their stage of development and their parents’ con dence in addressing their needs
Many are not for several reasons: the broader notion of palliative care is not included in their education and training; formal collaboration with palliative care specialists has been inadequate to date; and systems of care reinforce hierarchical rather than relational models of interaction-a key feature of palliative care. Reaching this new horizon requires better understanding of the progression and critical decision points of NMDs, and training for clinicians to raise their awareness and create new knowledge and skills they can apply to the integration of pediatric palliative care (PPC) practices [34,35]
Summary
To advance palliative care integration, we formed an international consortium of patients and families a ected by NMDs and an interdisciplinary group of clinicians who care for these families. We had three goals: identify transitions in disease progression; explore the challenges facing patients, families, and clinicians during these transitions; and identify opportunities for integrating PPC into the care of children a ected by these conditions. Selection criteria for team members included leadership experience, commitment to improving care for children with NMDs, and willingness to explore palliative care integration and new models of engagement (Table 2). E rest of the summit involved group discussions led by interdisciplinary professionals who were experienced facilitators. Based on their experience and expertise, participants were assigned to six groups, three focused on SMA-1, three on DMD. All six groups addressed elements of palliative care, with one group from each disease focused on (1) communication and decision making, (2) ethical and cultural challenges, and (3) pain/su ering and spiritual care
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