Clinical trial NCT05644899: Ultrasound-assisted or Landmark-based Intrathecal Administration of Nusinersen in Adult Patients With Spinal Muscular Atrophy (The EchoSpin Study)
Official title: Interlaminar Ultrasound-assisted or Landmark-based Intrathecal Administration of Nusinersen in Adult Patients With Spinal Muscular Atrophy: a Retrospective Analysis of More Than 500 Consecutive Administrations
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Anesthesia, Spinal
Spinal Puncture Complications
Spinal Muscular Atrophy
Ultrasonography
Lead sponsor: Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Sponsor class: OTHER
First posted: 2022-12-09
Enrollment: 51
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: RETROSPECTIVE
Sampling method: PROBABILITY_SAMPLE
Study population: 51 adults with a genetically confirmed diagnosis of SMA (13 SMA type 2, 38 SMA type 3) consecutively treated with nusinersen intrathecal administration between April 2018 and August 2022 in "Policlinico A. Gemelli" in Rome.

Brief summary: 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.


Eligibility criteria: Inclusion Criteria:

* A genetically confirmed diagnosis of SMA
* Treatment with nusinersen

Exclusion Criteria:

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

Primary outcomes:
measure: Number of successful administrations
timeFrame: During the procedure
measure: Number of attempts
timeFrame: During the procedure
measure: Procedure time
timeFrame: During the procedure
measure: Number of technical success procedures
timeFrame: During the procedure
measure: Adverse events (AEs)
timeFrame: In the first 72 hours after the procedure
Secondary outcomes:
Other outcomes:
Locations:
city: Roma
country: Italy
facility: Fondazione Policlinico Gemelli
state: RM
Study officials:
affiliation: Fondazione Policlinico A. Gemelli
name: Bruno A Zanfini
role: PRINCIPAL_INVESTIGATOR
References: