SMA TREATMENTS: P.348Intrathecal administration of nusinersen in children: usefulness of neuroimaging in patients with abnormal spinal anatomy

Abstract

Nusinersen has to be repeatedly administered intrathecally in patients with spinal muscular atrophy (SMA). The development of scoliosis is a complication associated with these patients and may limit the administration of treatment. Describe our experience in the administration of intrathecal medication in patients with SMA. We propose a strategy that allows the intrathecal approach in patients with severe scoliosis. Prospective observational study. We collected demographic data related to the disease and the intrathecal administration procedure in patients with SMA treated with nusinersen from March 2018 to March 2019. The patients were classified into 2 groups: simple procedure (standard lumbar puncture) and complex procedure (the intrathecal approach that may require guidance by computer tomography (CT). The procedure was considered complex in patients with scoliosis (curve with more than 50 degrees in spine x-ray) or patients with a history of scoliosis surgery. A total of 96 intrathecal treatments of nusinersen were performed in 21 patients. Two patients with SMA type 1, 15 patients with SMA type 2 and 4 patients with SMA type 3. Twelve were boys and the age of onset of treatment was between 6 months and 16 years. A standard lumbar puncture was performed in 14 patients (67procedures). Seven patients required guidance by CT in 22 procedures. The technique was performed under sedation in all cases. The frequency of adverse effect around the procedure was 10,4% in the first dose and decreasing in the following ones. Most of related events were mild. The most common complications were headache and back pain. With the protocol used the administration of nusinersen was possible in all cases. The procedure was safe and feasibility, even in those with significant scoliosis. The use of imaging techniques eases the administration of intrathecal drugs in patients with spinal deformity.