Parents' perspectives on nusinersen treatment for children with spinal muscular atrophy.

Abstract

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.

• Authors: Mette van Kruijsbergen, Carin D Schröder, Marjolijn Ketelaar, W Ludo van der Pol, Inge Cuppen, Annette van der Geest, Fay-Lynn Asselman, Maarten J Fischer, Johanna M A Visser-Meily, Marijke C Kars
• Journal: Developmental medicine and child neurology (Dev Med Child Neurol) Vol. 63 Issue 7 Pg. 816-823 (07 2021) ISSN: 1469-8749 [Electronic] England
• PMID: 33550591 (Publication Type: Journal Article, Research Support, Non-U.S. Gov't)
• Copyright: © 2021 The Authors. Developmental Medicine & Child Neurology published by John Wiley & Sons Ltd on behalf of Mac Keith Press.

Chemical References

• Oligonucleotides
• nusinersen

Topics

• Child
• Child, Preschool
• Databases, Factual
• Decision Making
• Female
• Humans
• Male
• Muscular Atrophy, Spinal (drug therapy)
• Oligonucleotides (therapeutic use)
• Parents
• Quality of Life
• Treatment Outcome