Parents' perspectives on nusinersen treatment for children with spinal muscular atrophy.
Mette van Kruijsbergen, Carin D Schröder, Marjolijn Ketelaar and 7 others
PMID 33550591WHAT IT FOUND
Parents described choosing nusinersen as weighing survival, disease progression, and their child's daily life.
Clear, neutral physician communication and accessible information helped them feel able to accept or decline treatment.
Key findings
01Parents described a spectrum from a biomedical focus on battling the disease and prolonging life to a holistic focus on quality of life.
02Many parents felt refusing treatment did not feel like a choice, while some declined because they feared treatment would prolong a poor quality of life.
03Physicians' clear and neutral communication helped parents feel in control of the treatment decision.
STILL TO COME
How it was doneWhat they foundWhat it means for PTs
Read the rest of this summary
You get three full summaries a month, free, and we do not ask for a card. Search, the TL;DRs and your library stay unlimited either way.
What it does not show
The sample was selective: parents were recruited from a national database and had already decided about nusinersen, so they may have justified their choice. Only parents fluent in Dutch or English were included, so other cultural and language groups were not represented. All parents who declined treatment were parents of children with type 1 SMA; no families of children with type 2 SMA declined in the registry, so perspectives on refusal in other SMA types were limited. Some children had died before or around the interviews, so bereavement may have shaped parents' accounts. Participants may have been more motivated to elaborate on their thoughts than parents who did not respond.
Declared interests
Prinses Beatrix Spierfonds funded the study.
The easy way to misread this
Do not read this as evidence that nusinersen improves survival, function, or quality of life. It reports parents' views about treatment decisions, not measured clinical outcomes.