The team from the London Neuromuscular Center studied the evolution of oral food intake capacities measured by the Pediatric Functional Oral Intake Scale (p-FOIS), in 24 children with spinal muscular atrophy linked to SMN1 ( SMA) type 1, on nusinersen for 2 years, from a median age of 11 months (1 month to 7.5 years). The cohort consisted of 3 children with SMA types 1A, 9 types 1b and 12 types 1c.
- Three children had a gastrostomy and 11 a nasogastric tube at the start of treatment with nusinersen.
- If the median CHOP INTEND functional score increased by 10 points after 1 and 2 years (42 vs 32) of treatment with nusinersen, the median p-FOIS score decreased from 3 to 2 at the same time, indicating the majority share feeding by gastrostomy.
- The median age for placement of a gastrostomy was 8 months (0 to 2 years and 2 months).
- Only four children did not need it: they were the least severe forms of SMA type 1 (SMA type 1c).
Larger prospective studies will explore the impact of long-term nusinersen treatment on swallowing and oral food intake abilities of children with SMA 1.
