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Cervical puncture to deliver nusinersen in patients with spinal muscular atrophy.
Neurology 2018; 91(7):e620-e624Neur

Abstract

OBJECTIVE

To report our experience delivering intrathecal nusinersen through cervical puncture in patients with spinal muscular atrophy (SMA) with no lumbar access.

BACKGROUND

SMA is a neuromuscular disorder characterized by profound muscle weakness, atrophy, and paralysis due to degeneration of the anterior horn cells. Nusinersen, the first Food and Drug Administration-approved treatment for SMA, is administered intrathecally via lumbar puncture; however, many patients with SMA have scoliosis or solid spinal fusion with hardware that makes lumbar access impossible. Studies in primates have demonstrated better spinal cord tissue concentration with intrathecal injections than with intracerebral ventricular injections. Therefore we have used C1/C2 puncture as an alternative to administer nusinersen.

METHOD

Retrospective chart review.

RESULTS

Intrathecal nusinersen via cervical puncture was given to 3 patients who had thoracic and lumbosacral spinal fusion: a 12-year-old girl with type 1 SMA and 2 17-year-old girls with type 2 SMA. Cervical puncture was performed without deep sedation under fluoroscopic guidance using a 25-G or a 24-G Whitacre needle in the posterior aspect of C1-C2 interspace and full dose of nusinersen (12 mg/5 mL) was injected after visualizing free CSF flow. Patients completed their 4 loading doses and first maintenance dose of nusinersen, and 15 procedures were successful and well-tolerated.

CONCLUSION

Cervical puncture is a feasible alternative delivery route to administer intrathecal nusinersen in patients with longstanding SMA and spine anatomy precluding lumbar access when done by providers with expertise in this procedure.

Authors+Show Affiliations

From the Department of Neurology, Division of Neuromuscular Medicine (A.V., R.P., K.E., E. Collins, E. Ciafaloni), Department of Imaging Sciences, Division of Diagnostic and Interventional Neuroradiology (S.D., I.Y., P.-L.W.), and Department of Neurology, Division of Child Neurology (J.K.), University of Rochester Medical Center, NY.From the Department of Neurology, Division of Neuromuscular Medicine (A.V., R.P., K.E., E. Collins, E. Ciafaloni), Department of Imaging Sciences, Division of Diagnostic and Interventional Neuroradiology (S.D., I.Y., P.-L.W.), and Department of Neurology, Division of Child Neurology (J.K.), University of Rochester Medical Center, NY.From the Department of Neurology, Division of Neuromuscular Medicine (A.V., R.P., K.E., E. Collins, E. Ciafaloni), Department of Imaging Sciences, Division of Diagnostic and Interventional Neuroradiology (S.D., I.Y., P.-L.W.), and Department of Neurology, Division of Child Neurology (J.K.), University of Rochester Medical Center, NY.From the Department of Neurology, Division of Neuromuscular Medicine (A.V., R.P., K.E., E. Collins, E. Ciafaloni), Department of Imaging Sciences, Division of Diagnostic and Interventional Neuroradiology (S.D., I.Y., P.-L.W.), and Department of Neurology, Division of Child Neurology (J.K.), University of Rochester Medical Center, NY.From the Department of Neurology, Division of Neuromuscular Medicine (A.V., R.P., K.E., E. Collins, E. Ciafaloni), Department of Imaging Sciences, Division of Diagnostic and Interventional Neuroradiology (S.D., I.Y., P.-L.W.), and Department of Neurology, Division of Child Neurology (J.K.), University of Rochester Medical Center, NY.From the Department of Neurology, Division of Neuromuscular Medicine (A.V., R.P., K.E., E. Collins, E. Ciafaloni), Department of Imaging Sciences, Division of Diagnostic and Interventional Neuroradiology (S.D., I.Y., P.-L.W.), and Department of Neurology, Division of Child Neurology (J.K.), University of Rochester Medical Center, NY.From the Department of Neurology, Division of Neuromuscular Medicine (A.V., R.P., K.E., E. Collins, E. Ciafaloni), Department of Imaging Sciences, Division of Diagnostic and Interventional Neuroradiology (S.D., I.Y., P.-L.W.), and Department of Neurology, Division of Child Neurology (J.K.), University of Rochester Medical Center, NY.From the Department of Neurology, Division of Neuromuscular Medicine (A.V., R.P., K.E., E. Collins, E. Ciafaloni), Department of Imaging Sciences, Division of Diagnostic and Interventional Neuroradiology (S.D., I.Y., P.-L.W.), and Department of Neurology, Division of Child Neurology (J.K.), University of Rochester Medical Center, NY.From the Department of Neurology, Division of Neuromuscular Medicine (A.V., R.P., K.E., E. Collins, E. Ciafaloni), Department of Imaging Sciences, Division of Diagnostic and Interventional Neuroradiology (S.D., I.Y., P.-L.W.), and Department of Neurology, Division of Child Neurology (J.K.), University of Rochester Medical Center, NY. emma_ciafaloni@urmc.rochester.edu.

Pub Type(s)

Journal Article

Language

eng

PubMed ID

30006410

Citation

Veerapandiyan, Aravindhan, et al. "Cervical Puncture to Deliver Nusinersen in Patients With Spinal Muscular Atrophy." Neurology, vol. 91, no. 7, 2018, pp. e620-e624.
Veerapandiyan A, Pal R, D'Ambrosio S, et al. Cervical puncture to deliver nusinersen in patients with spinal muscular atrophy. Neurology. 2018;91(7):e620-e624.
Veerapandiyan, A., Pal, R., D'Ambrosio, S., Young, I., Eichinger, K., Collins, E., ... Ciafaloni, E. (2018). Cervical puncture to deliver nusinersen in patients with spinal muscular atrophy. Neurology, 91(7), pp. e620-e624. doi:10.1212/WNL.0000000000006006.
Veerapandiyan A, et al. Cervical Puncture to Deliver Nusinersen in Patients With Spinal Muscular Atrophy. Neurology. 2018 08 14;91(7):e620-e624. PubMed PMID: 30006410.
* Article titles in AMA citation format should be in sentence-case
TY - JOUR T1 - Cervical puncture to deliver nusinersen in patients with spinal muscular atrophy. AU - Veerapandiyan,Aravindhan, AU - Pal,Ria, AU - D'Ambrosio,Stephen, AU - Young,Iris, AU - Eichinger,Katy, AU - Collins,Erin, AU - Westesson,Per-Lennart, AU - Kwon,Jennifer, AU - Ciafaloni,Emma, Y1 - 2018/07/13/ PY - 2018/02/22/received PY - 2018/05/14/accepted PY - 2018/7/15/pubmed PY - 2019/8/16/medline PY - 2018/7/15/entrez SP - e620 EP - e624 JF - Neurology JO - Neurology VL - 91 IS - 7 N2 - OBJECTIVE: To report our experience delivering intrathecal nusinersen through cervical puncture in patients with spinal muscular atrophy (SMA) with no lumbar access. BACKGROUND: SMA is a neuromuscular disorder characterized by profound muscle weakness, atrophy, and paralysis due to degeneration of the anterior horn cells. Nusinersen, the first Food and Drug Administration-approved treatment for SMA, is administered intrathecally via lumbar puncture; however, many patients with SMA have scoliosis or solid spinal fusion with hardware that makes lumbar access impossible. Studies in primates have demonstrated better spinal cord tissue concentration with intrathecal injections than with intracerebral ventricular injections. Therefore we have used C1/C2 puncture as an alternative to administer nusinersen. METHOD: Retrospective chart review. RESULTS: Intrathecal nusinersen via cervical puncture was given to 3 patients who had thoracic and lumbosacral spinal fusion: a 12-year-old girl with type 1 SMA and 2 17-year-old girls with type 2 SMA. Cervical puncture was performed without deep sedation under fluoroscopic guidance using a 25-G or a 24-G Whitacre needle in the posterior aspect of C1-C2 interspace and full dose of nusinersen (12 mg/5 mL) was injected after visualizing free CSF flow. Patients completed their 4 loading doses and first maintenance dose of nusinersen, and 15 procedures were successful and well-tolerated. CONCLUSION: Cervical puncture is a feasible alternative delivery route to administer intrathecal nusinersen in patients with longstanding SMA and spine anatomy precluding lumbar access when done by providers with expertise in this procedure. SN - 1526-632X UR - https://www.unboundmedicine.com/medline/citation/30006410/Cervical_puncture_to_deliver_nusinersen_in_patients_with_spinal_muscular_atrophy_ L2 - http://www.neurology.org/cgi/pmidlookup?view=long&pmid=30006410 DB - PRIME DP - Unbound Medicine ER -