Nusinersen by subcutaneous intrathecal catheter for symptomatic spinal muscular atrophy patients with complex spine anatomy

2021 ◽  
Author(s):  
Vincent J. Carson ◽  
Millie Young ◽  
Karlla W. Brigatti ◽  
Donna L. Robinson ◽  
Robert M. Reed ◽  
...  
Keyword(s):  
Spinal Muscular Atrophy ◽  
Muscular Atrophy ◽  
Intrathecal Catheter

scholarly journals P.154 Subcutaneous intrathecal catheter and port implants for administration of Nusinersen in patients with Spinal Muscular Atrophy

2021 ◽  
Vol 48 (s3) ◽  
pp. S64-S64
Author(s):  
A Bokeris ◽  
D Mcneely ◽  
c restrepo ◽  
J Sheriko
Keyword(s):  
Spinal Cord ◽  
Spinal Muscular Atrophy ◽  
Motor Neurons ◽  
Muscular Atrophy ◽  
Medication Administration ◽  
Intrathecal Baclofen ◽  
Outpatient Setting ◽  
Intrathecal Catheter ◽  
Spine Deformities ◽  
Health Canada

Background: Until recently, no effective treatment was available for spinal muscular atrophy (SMA). In 2017, Health Canada approved intrathecal Nusinersen a medication that prevents degeneration of the motor neurons in the spinal cord. The administration is intrathecally most commonly via lumbar puncture (LP) to have a direct effect on the motor neurons of the spinal cord. Many older patients with SMA and concomitant spinal deformities present technical challenges to access the thecal sac. Different routes have been described for delivery of the medication whoever these techniques may require sedation, are associated with radiation exposure, and demand experience personnel. Methods: A new surgical technique has been proposed to overcome these obstacles by combining two Health Canada approved devices: 1) an intrathecal catheter designed for intrathecal baclofen pumps and 2) an implantable subcutaneous port designed for intravascular medication administration Results: We describe the technical nuances and outline the clinical outcomes of six patients with complex spine deformities who have undergone such an implant for administration of Nusinersen. Conclusions: We discuss the benefits of the procedure which includes: 1) administration in the outpatient setting without sedation, 2) avoidance of costly imaging and experienced personnel, and 3) placement of the catheter in the cervicothoracic junction.

Multicenter Experience with Nusinersen Application via an Intrathecal Port and Catheter System in Spinal Muscular Atrophy

2020 ◽  
Vol 51 (06) ◽  
pp. 401-406
Author(s):  
Marina Flotats-Bastardas ◽  
Andreas Hahn ◽  
Oliver Schwartz ◽  
Steffan Linsler ◽  
Sascha Meyer ◽  
...  
Keyword(s):  
Spinal Muscular Atrophy ◽  
Muscular Atrophy ◽  
Survival Motor Neuron ◽  
Study Cohort ◽  
Intrathecal Catheter ◽  
Survival Motor Neuron Protein ◽  
Severe Scoliosis ◽  
Port Implantation ◽  
Catheter System

AbstractNusinersen, an antisense oligonucleotide enhancing the production of the survival motor neuron protein, is approved for the treatment of spinal muscular atrophy (SMA) but requires repetitive lumbar punctures. Application via a subcutaneous port connected to a permanent intrathecal catheter has been proposed as an alternative for patients with severe scoliosis, spinal fusion, or comorbidities, rendering serial interlaminar punctures complicated and risky. Since experience with this technique is sparse and follow-up data are lacking, we assessed feasibility, safety, and tolerability of this approach in eight patients with SMA II/SMA III receiving Nusinersen in a multicenter study. Median age at port implantation was 21 years (range: 10–30 years), and median follow-up time thereafter was 19 months (range: 7–24 months). Leakage of the port catheter occurred in two patients, promptly resolving after resuturing. No further complications such as infection, dislocation, kinking, or obstruction of the port were noted in any of the patients. These findings suggest that application via an intrathecal port and catheter system represents a safe and feasible option for Nusinersen treatment in subjects with SMA. However, to detect rare adverse events longer term follow-up in a larger study cohort is warranted.

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