Tasks and interfaces in primary and specialized palliative care for Duchenne muscular dystrophy – A patients’ perspective

Abstract

In spite of the improvements in care and the emergence of disease-modifying treatments, Duchenne muscular dystrophy (DMD) remains a life-limiting disease of adolescence and (young) adulthood. Palliative care approaches and principles should be integrated from the point of diagnosis and implemented throughout the lifespan. A nationwide cross-sectional survey based on a mixed-method-design of qualitative and quantitative research approaches evaluated the structural implementation and perception of palliative care for DMD in Germany. Data analyses revealed that palliative care was predominantly provided at the primary care level by pediatricians, general practitioners and specialized multi-professional outpatient structures. The majority of patients did not utilize the scopes of specialized palliative structures. Simultaneously, insufficiently treated complex symptoms, emergent and elective hospitalizations and barriers in transitioning into adult care presented a considerable burden. A collaborative integrated model with a close cooperation of patients, families and care providers is proposed involving task areas and interfaces complementing primary and specialized palliative care (1) management of complex symptoms, (2) crisis support, (3) intermittent relief of the strain for caregivers, (4) coordination of care, (5) advance care planning and (6) end-of-life care. Specialized palliative care should be used as an "add-on" approach in time of need rather than as a prognosis or disease stage.