FV 871. Pediatric Palliative Care of Duchenne Muscular Dystrophy in Germany

Background: Pediatric palliative care (PPC) care for children and adolescents with life-limiting illnesses (LLIs) and their families aims to improve their quality of life. Duchenne muscular dystrophy (DMD) is a rare, life-limiting neuromuscular disease. The international standardized care guidelines “Diagnosis and management of Duchenne muscular dystrophy” from 2010, supplemented in 2018, includes “palliative care” of patients with DMD.

Objective: The aim of this study to analyze the (1) structural implementation of general and specialized PC, the needs of patients with DMD in Germany and (2) the development of a “tool” for rapid analysis of demands and care in other neurological LLI for getting a differentiated, meaningful and reliable database for the optimization of supply.

Methods: A mixed-method design of qualitative and quantitative research methods was applied. First, patients and/or their families were interviewed. Based on this, a questionnaire was developed that was available online from November 2017 to February 2018. The patients (inclusion criterion from 10 years = early nonambulatory stage) were primarily contacted via the DMD patient registry at the Friedrich Baur Institute, LMU Munich. Microdata of case-related hospital statistics (diagnosis-related groups, DRG) were also used for the analysis of inpatient care.

Results: The qualitative analysis (n = 9 patients, mean age 20.8 years) revealed needs for multiprofessional counseling, care, transition, and the concern to discuss the end of life within the family or with doctors. The quantitative phase sample included n = 150 patients (mean age 19.8 years, 79% living with their parents). The medical care was provided to 45% by pediatricians, to 37% by general practitioners; 27% of the patients had not received a recommendation for multiprofessional care within specialized structures such as sociopediatric centers or neuromuscular centers. Difficulties of transition were evident in general and specialized care: 18% of adult patients were cared for by pediatricians, and 53% of patients over the age of 21 years did not receive specialized multiprofessional care. Fifty-five per cent of respondents had experienced elective hospitalizations in the past 2 years and 32% had unplanned hospital admissions. The DRG statistics showed an increase in muscular dystrophy diagnoses between 2005 and 2013, especially in the age group of 20 to 29 years.

Patients reported the following symptoms: pain (57% of all respondents), fatigue (43%), depressed mood (30%), constipation (25%), and dyspnea (7%). According to the patients, 10 to 53% of the symptoms did not get specific treatment. Eighty-one per cent of respondents did not make use of hospice and palliative care services. Regarding their condition, the majority of patients felt well informed and involved. According to the doctor–patient interview, communication on the subject of “death and dying” was reported as “rare.”

Conclusion: The online questionnaire that we developed was able to comprehensively record the care situation and the needs of patients with DMD. Data analysis revealed deficits in symptom control, care within specialized care structures, transition, and communication about dying and death. The strengths of palliative care providers are in the areas of multiprofessional symptom control and treatment, outreach coordination, counseling, communication for target therapy, and dying and death. Specialized structures of PPC could compensate deficits or close gaps. Complex symptom conditions and hospitalization could provide starting points for the inclusion of specialist palliative care providers. For the future, it will be important to develop an integrative neuropediatric PC together with all the different parties involved.