Experiences and the psychosocial situation of parental caregivers of children with spinal muscular atrophy against the background of new treatment options: a qualitative interview study.

Background: Spinal muscular atrophy is a rare neurodegenerative disorder in children which leads untreated to muscle wasting, respiratory impairments, and a shortened life expectancy. Parents as primary caregivers are often physically and psychologically burdened. In recent years, new and promising treatment options have been approved, but it remains unclear if they have an impact on the psychosocial situation of affected families.

Objectives: The aim of this study was to explore the views and experiences of parents as informal caregivers of children with SMA in the course of the disease against the background of new treatment options (Spinraza® or Zolgensma®).

Methods: We conducted qualitative interviews with 27 parents of children with SMA treated with Spinraza® and Zolgensma® from April to September 2020. The analysis was done using thematic analysis and reported according to the COREQ criteria.

Results: The data analysis resulted in three main themes: a) caregiver burden and negative consequences for families, b) resources and protective aspects, c) psychosocial care needs. The results are discussed against the background of new treatment options and previous models of supportive care needs. Parental caregivers of affected children face multiple burdens in different stages of the child's disease progression.

Conclusion: Although new treatment options for SMA showed observable effects for most parents, the main caregiver burden and reported symptoms were attributable to the overburdening care tasks. To unburden families, more screening for unmet needs, family-centered help services, professional caregivers, childcare, and sufficient financial support are needed.