Parents’ perspectives on nusinersen treatment for children with spinal muscular atrophy

Aim To gain insight into parents’ perspectives about their decision‐making process concerning nusinersen treatment for their child, including perceived needs and concerns, and to explore factors that influence this process. Method This was an exploratory qualitative interview study among parents of children with spinal muscular atrophy types 1 to 3. Data were analysed using inductive thematic analysis. Results Nineteen parents of 16 children representing 13 families participated. A wide variety of perspectives was reported ranging from a biomedical approach, which focused on battling the disease, to a holistic approach, which aimed for a good quality of life for their child. The most important factors that helped parents to decide were honest and neutral communication with their physician and access to available information. Interpretation It is important physicians understand that there are different perspectives influencing the decision‐making process. Physicians should create an environment that allows parents to accept or reject treatment by communicating honestly and openly with them and by discussing both options extensively. Clear information about pros and cons, recent developments in research, and the experiences of other parents should be made available to enable parents to make an informed decision. What this paper adds Parents perceived different needs and concerns about nusinersen treatment, which emphasized individual differences.Parents’ perspectives varied from battling the disease to preserving quality of life.Life expectancy, stopping deterioration, and improving quality of life were the perceived benefits of nusinersen treatment.Open communication about the pros and cons of treatment with clinicians facilitated decision‐making.Clear and honest information facilitated the alignment of values and goals.
What this paper adds Parents perceived different needs and concerns about nusinersen treatment, which emphasized individual differences.Parents’ perspectives varied from battling the disease to preserving quality of life.Life expectancy, stopping deterioration, and improving quality of life were the perceived benefits of nusinersen treatment.Open communication about the pros and cons of treatment with clinicians facilitated decision‐making.Clear and honest information facilitated the alignment of values and goals. This article's abstract has been translated into Spanish and Portuguese. Follow the links from the abstract to view the translations.