A new clinical trial investigating the effectiveness of ultrasound (US) assistance for administering intrathecal nusinersen in patients with scoliosis due to spinal muscular atrophy (SMA) is set to be published soon this year.

The antisense nucleotide nusinersen is a US Food and Drug administration approved treatment for all SMA subtypes and is considered the current standard of care for SMA. It is administered intrathecally through a lumbar puncture; however, the procedure can be challenging in patients with scoliosis secondary to their condition. These patients often require spinal instrumentation to prevent worsening deformities.

US guidance has been proposed as a useful tool for treatment administration, associated with a higher success rate, reduced needed attempts, and reduced risk of adverse events.


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The authors aim to assess the efficacy of the US as well as that of the landmark-based technique through a retrospective study based on a historical cohort of 51 patients.

All participants included had a genetically confirmed diagnosis of SMA and received treatment with nusinersen. Exclusion criteria included a history of congenital coagulopathy, current localized infection, and increased intracranial pressure.

The technique used on each patient varied according to the anesthesiologist’s preference. The position of the procedure varied between seated and lateral decubitus, and the drug was administered through either the L4-L5 or L3-4 intervertebral space.

Furthermore, the primary outcome measures of the study include the number of successful nusinersen administrations, the number of needle insertions through the skin surface, procedure time (in minutes), the number of successful treatment administrations in less than 4 attempts, and adverse effects during the 73 hours following the procedure.

Post-dural puncture headache (PDPH) and low back pain (LBP) are among the most commonly reported adverse effects associated with the procedure.

The study started on December 16, 2022 and is expected to be completed by January 30, 2023.

Reference

Ultrasound-assisted or landmark-based intrathecal administration of nusinersen in adult patients with Spinal Muscular Atrophy (The EchoSpin Study) (EchoSpin). ClinicalTrials.gov. Accessed January 3, 2022.