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Intrathecal Delivery of Antisense Oligonucleotides in the Rat Central Nervous System
Published on: October 29, 2019
Real-Time Ultrasound-Guided Intrathecal Delivery of Nusinersen in Adult Patients With Spinal Muscular Atrophy and
Xijian Ke1, Yinzhu Wu2, Rui Yang3
1Department of Anesthesiology and Pain Medicine, Hubei Key Laboratory of Geriatric Anesthesia and Perioperative Brain Health, and Wuhan Clinical Research Center for Geriatric Anesthesia, Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology, Wuhan, P.R. China.
Background:
Spinal muscular atrophy ( SMA ) is a rare genetic neuromuscular disorder characterized by progressive muscle atrophy and weakness. Nusinersen, the only US Food and Drug Administration-approved antisense oligonucleotide specifically for SMA management, is administered intrathecally. However, a substantial proportion of adult patients with SMA develop severe scoliosis, posing significant technical challenges for traditional lumbar puncture procedures. Real-time ultrasound guidance offers a potential solution for intrathecal nusinersen administration in these challenging cases.
Objectives:
We sought to evaluate the technical feasibility and safety profile of real-time, ultrasound-guided intrathecal delivery of nusinersen in adult patients with SMA and complex spinal anatomy, including those with scoliosis or vertebral hardware.
Study Design:
Retrospective chart review.
Setting:
This study was conducted at a single medical center.
Methods:
The data were retrospectively collected from the medical records Of 26 adult patients with SMA who had challenging intrathecal access (scoliosis or vertebral hardware) and who underwent real-time ultrasound-guided intrathecal nusinersen administration. Real-time ultrasound-guided lumbar puncture was performed using either the paramedian sagittal oblique view translaminar approach or the coronal view transforaminal approach. Procedure time, technical success, and adverse events were noted.
Results:
A total of 151 real-time, ultrasound-guided lumbar punctures were performed. All procedures were technically successful and well-tolerated. The mean procedure time for the paramedian sagittal oblique view translaminar approach was 10.5 ± 1.7 minutes for moderate scoliosis and 20.7 ± 9.3 minutes for severe scoliosis. The mean procedure time for the transforaminal approach, used when the paramedian sagittal oblique view translaminar approach was not feasible, was 22.5 ± 6.1 minutes. No severe adverse events were observed.
Limitations:
This was a retrospective, single-center study with a relatively small sample size. Generalizability may be limited.
Conclusions:
Real-time ultrasound-guided intrathecal administration of nusinersen is feasible and appears safe in adult patients with SMA and complex spinal anatomies. Further prospective, multi-center clinical trials are warranted to validate these findings and evaluate long-term safety and efficacy in a larger patient cohort.
