Healthcare utilisation in children with SMA type 1 treated with nusinersen: a single centre retrospective review.

ABSTRACT:

Background

Nusinersen has been used to treat spinal muscular atrophy type 1 (SMA1) in the UK since 2017. While initial trials showed neuromuscular benefit from treating SMA1, there is little information on the respiratory effects of nusinersen. We aimed to look at the respiratory care, hospital utilisation and associated costs in newly treated SMA1.

Methods

We reviewed the medical records of all children within the West Midlands with SMA1 treated with nusinersen at Royal Stoke University Hospital. Baseline demographics and hospital admission data were collected including: the reason for admission, total hospital days, days of critical care, days intubated, discharge diagnosis, doses of nusinersen and treatment complications.

Results

11 children (six girls) received nu

SUBMITTER: Ali I 

PROVIDER: S-EPMC6937078 | biostudies-literature | 2019

REPOSITORIES: biostudies-literature