Abstract
BackgroundLife extension by medical interventions and health-related quality of life (HRQOL) are sometimes conflicting aspects of medical care. Long-term ventilation in children with neuromuscular disease is a well-established life-extending procedure and often at the center of this conflict. HRQOL and the mental health of affected children and their families become even more important in respect to emerging therapies in neuromuscular diseases with longer life-expectancy of treated patients and considerable costs of medical treatment.MethodsWe performed a questionnaire survey in a total of forty-three families of children with neuromuscular disease treated in the University Medical Center Hamburg-Eppendorf and the Children’s Hospital Altona. We evaluated self- and proxy-reported HRQOL and mental health outcomes of affected children and their parents using validated and age-appropriate instruments.ResultsCompared to normative data, children with neuromuscular diseases and their families experienced a lower HRQOL and mental health. However, there was no additional negative influence on the overall HRQOL by ventilator use.ConclusionsAs ventilator use was not responsible for the reduction of HRQOL and mental health our data contributes an important aspect to the discussion about life-prolonging procedures, in particular mechanical ventilation, in severly disabled patients.
Highlights
Life extension by medical interventions and health-related quality of life (HRQOL) are sometimes conflicting aspects of medical care
The purpose of our study was to investigate health-related quality of life and mental health outcomes of affected children and their families enclosing the aspect of ventilation
The following demographic information was collected by self-reported questionnaires and electronic health records: age of children and parents, gender of children, occupational status of parents, highest level of education of parents, family income, type of neuromuscular disorder, type and duration of mechanical ventilation
Summary
Life extension by medical interventions and health-related quality of life (HRQOL) are sometimes conflicting aspects of medical care. In recent years a growing number of new therapies in the field of neuromuscular diseases have emerged modifying the disease’s course and enhancing the life-expectancy of affected children [4,5,6] In this context two aspects of mechanical ventilation in those patients become more important: 1. Focusing on the quality of life considering that new therapies might extend life expectancy but still do not reverse the course of the disease to a significant extent One example in this discussion is the initiation of mechanical ventilation in patients with spinal muscular atrophy type 1 in the face of recently approved disease-modifying treatments such as nusinersen and/or Onasemnogene abeparvovec [4,5,6,7]. HRQOL of technology-dependent and/or physically disabled children were reported to be significantly underestimated by caregivers [8, 9]
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