Evolution of bulbar function in spinal muscular atrophy type 1 treated with nusinersen.
Harriet Weststrate, Georgia Stimpson, Lily Thomas and 6 others
PMID 35103306WHAT IT FOUND
In 24 children with SMA type 1 treated with nusinersen, swallowing and feeding worsened over 24 months: more needed tube feeding, while motor scores improved.
Bulbar care must stay active even when movement improves.
Key findings
01Median p-FOIS bulbar scores fell from 3 at baseline to 1 at 6 months and 2 at 12 and 24 months.
02Tube feeding increased from 14 patients at baseline to 20 of the 24 patients at 24 months.
03Median CHOP INTEND motor scores across available observations rose from 32 at baseline to 42 at 24 months, even though p-FOIS scores declined.
STILL TO COME
How it was doneWhat they foundWhat it means for PTsWhat it means for SLPs
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What it does not show
Single centre and small: 24 children from a single hospital. Retrospective record review and audit, not a randomised trial. No untreated or natural history comparison group, so treatment effect cannot be separated from disease progression. Motor score data were missing for many patients: CHOP INTEND was available for 14 patients at baseline and 18 at 24 months. Videofluoroscopy was available for only eight patients, at variable time points after treatment, with no follow-up studies. No presymptomatic treated children were included, so results do not apply to children treated before symptom onset. The p-FOIS adaptation and validation were reported separately and need larger studies to confirm use in SMA.
Declared interests
An author reported speaker and consulting fees from Biogen, Novartis Gene Therapies (AveXis), and Roche, and principal investigator roles in SMA studies sponsored by Novartis Gene Therapies and Roche. The supplied text lists non-US government research support, but does not state that a company designed or wrote the study.
The easy way to misread this
Do not conclude that nusinersen worsened bulbar function. This study had no untreated comparison group, so it cannot show whether disease progression, other management, or nusinersen explained the changes.