New Delivery System for Nusinersen Safe in SMA New Delivery System for Nusinersen Safe in SMA
A new subcutaneous intrathecal catheter system may be a safe and relatively well-tolerated alternative to lumbar puncture for delivering nusinersen to adults and children with spinal muscular atrophy.Medscape Medical News
Spinal muscular atrophy (SMA) is among the most common monogenic causes of spinal motor neuron(SMN) degeneration with severely affected cases having a median survival of less than 2 years of age. Nusinersen (Spinraza) is a novel antisense oligonucleotide administered intrathecally for treatment of SMA. Most of these patients have scoliosis often treated by posterior fusion from T2 to sacrum making lumbar puncture challenging. Building on our prior experience in intrathecal catheter placement, we describe our initial experience with an image-guided approach to implantation of intrathecal catheters and subcutaneous ports in ...
This report outlines our approach and experience using transforaminal LP (TFLP) in patients with spinal muscular atrophy (SMA) with a 100% success rate. We discuss its utility in other patients with difficult access and compare TFLP with other techniques to access the intrathecal space.
SUMMARY: Patients with spinal muscular atrophy often have complete interlaminar osseous fusion, precluding lumbar puncture via the standard interlaminar approach. Recently, we have developed a new coaxial curved-needle variation of fluoroscopy-guided transforaminal lumbar puncture for intrathecal injections in this patient population. Between October 2017 and November 2018, fifty-nine consecutive transforaminal lumbar punctures using this technique were performed in 12 patients with spinal muscular atrophy for intrathecal nusinersen injection, with a 100% technical success rate and no C1–2 punctures required. One maj...
Conclusion: Our data suggest that a regular examination of routine CSF parameters in patients in which intrathecal ASOs are administered is important to obtain information on possible side effects and to gain further insights into intrathecal processes.
Conclusions: In SMA type 2 and 3 patients undergoing radiological imaging-assisted injections, the effective dose and DAP decreased during therapy with nusinersen. The mean effective dose in patients with spondylodesis was higher than that in patients without spondylodesis. Dosimetry should be monitored carefully in order to detect and prevent unnecessary radiation exposure.
ConclusionsNusinersen demonstrated a favorable safety profile in children with symptomatic infantile- and later-onset SMA. Most reported AEs and serious AEs were consistent with the nature and frequency of events typically seen with SMA or in the context of lumbar puncture procedures.RegistrationNCT01494701, NCT01703988, NCT01839656, NCT02193074, NCT02292537, NCT01780246, NCT02052791.
Discussion: Routine CSF cytology performed by experienced personnel represents an important and feasible tool for safety monitoring during treatment with intrathecally administered therapeutics. Analysis of leukocyte subpopulations did not raise safety concerns during nusinersen therapy. The potential significance of the unique phagocytic cells for disease course and treatment response needs to be further elucidated in the future.
Abstract Nusinersen (Spinraza) is a US Food and Drug Administration-approved intrathecal medication for the treatment of spinal muscular atrophy (SMA). Adult patients with SMA often undergo thoracolumbar fusion to treat neurogenic scoliosis, preventing thecal access. The authors report a laminotomy technique and the ease of intrathecal access in three SMA patients with prior thoracolumbar fusions.Patients were positioned in the lateral decubitus position or prone. Lumbar laminotomy was performed below the conus, between the lateral longitudinal rods, to preserve mechanical stability. Fluoroscopy provided real-time...
To assess the safety and efficacy of transforaminal lumbar puncture (LP) for the injection of Nusinersen (Spinraza ®) in the subset of patients who had extensive spinal fusion and/or scoliosis which precluded the standard interlaminar approach.
Spiranza (nusinersen) is the only treatment currently available for spinal muscular atrophy (SMA) and is delivered intrathecally. In children and adults with long disease duration is usual the presence of spinal deformities and previous spinal surgery that precludes the use of standard techniques of lumbar puncture. In clinical practice, it is becoming even more frequent the need of new ways of accessing the intrathecal space, especially when a repetitive administration route is needed. To present the complications and benefits of lumbar catheter placement for Spiranza administration in a patient with previous spinal surgery.