Ultrasound-assisted or Landmark-based Intrathecal Administration of Nusinersen in Adult Patients With Spinal Muscular Atrophy (The EchoSpin Study)

Institute of Hospitalization and Scientific Care (IRCCS)

Status: Completed

Conditions: Spinal Puncture Complications; Ultrasonography; Anesthesia, Spinal; Spinal Muscular Atrophy

Treatments: Procedure: Interlaminar ultrasound-assisted intrathecal administration of nusinersen; Procedure: Landmark-based intrathecal administration of nusinersen

Study type: Observational

Funder types: Other

Identifiers

NCT05644899

EchoSpin

Details and patient eligibility

About

Nusinersen (Spinraza, Biogen Inc, Boston, MA), the first treatment approved by FDA and EMA for all Spinal Muscular Atrophy (SMA) subtypes, is an antisense oligonucleotide that is administered intrathecally through a lumbar puncture. This procedure can be challenging in some adults with intermediate and late onset SMA (types II-IV) frequently presenting scoliosis secondary to neuromuscular weakness and often treated with spinal instrumentation to prevent worsening deformities.

In such patients, in order to access the intrathecal space, US guidance and/or assistance have been recently proposed as useful and successful tool. The US guidance and/or assistance have been associated to a high success rate, a reduction of number of attempts and needle passes to obtain a successful anesthesia. A reduced risk of adverse events (AEs), such as post dural puncture headache (PDPH) and low back pain (LBP), and low patient satisfaction often associated with multiple needle punctures was also reported.

Aim of this retrospective study was to report the efficacy, evaluated as rate of the successful procedures and subsequent delivery of nusinersen within the subarachnoid space, the number of attempts, the procedure time and the adverse events (AEs) of interlaminar intrathecal nusinersen administration using either ultrasound assistance or the landmark-based technique in a historical cohort of 51 adult SMA patients.

Enrollment: 51 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• A genetically confirmed diagnosis of SMA
• Treatment with nusinersen

Exclusion criteria

• congenital coagulopathy,
• localized infections,
• increased intracranial pressure.

Trial design

51 participants in 1 patient group

SMA patients

Description:

51 adults with a genetically confirmed diagnosis of SMA (13 SMA type 2, 38 SMA type 3) treated with nusinersen. The technique used was based on the anesthesiologist's preference.

Treatment:

Procedure: Interlaminar ultrasound-assisted intrathecal administration of nusinersen

Procedure: Landmark-based intrathecal administration of nusinersen