Nusinersen for Patients With Spinal Muscular Atrophy: 1415 Doses via an Interdisciplinary Institutional Approach.

Humans Injections, Spinal Lipopolysaccharides / therapeutic use Muscular Atrophy, Spinal / diagnostic imaging Oligonucleotides Retrospective Studies
Antisense oligonucleotide Cerebrospinal fluid Lumbar puncture Nusinersen Spinal muscular atrophy

Journal

Pediatric neurology
ISSN: 1873-5150
Titre abrégé: Pediatr Neurol
Pays: United States
ID NLM: 8508183

Informations de publication

Date de publication:
07 2022
Historique:
received: 13 01 2022
revised: 15 04 2022
accepted: 19 04 2022
pubmed: 1 6 2022
medline: 22 6 2022
entrez: 31 5 2022
Statut: ppublish

Résumé

Spinal deformity and prior spinal fusion pose technical challenges to lumbar puncture (LP) for nusinersen administration for patients with spinal muscular atrophy (SMA). In this retrospective study over two study phases, we evaluated (1) factors associated with difficult LP or unscheduled requirement for image guidance and (2) effectiveness of a triage pathway for selective use of image guidance and nonstandard techniques, particularly for patients with spinal instrumentation/fusion to the sacrum. With institutional review board approval, electronic health records, imaging, and administrative databases were analyzed for patients receiving nusinersen from January 2012 through September 2021. Descriptive statistics and univariate analyses were used. From January 2012 to March 2018 (phase 1), among 82 patients with SMA, 461 of 464 (99.4%) LP attempts were successful. Univariate analyses associated difficulty with prior spinal instrumentation, higher body mass index, and severity of the spinal deformity. Based on this experience, starting in April 2018 (phase 2), 125 patients were triaged selectively for ultrasound, fluoroscopy, or Dyna computed tomography. Patients with spinal instrumentation/fusion to the sacrum were treated primarily via intrathecal ports (137 doses) or transforaminal LP (55 doses). From April 2018 through September 2021, 704 of 709 (99.3%) LPs were successful. In total from January 2012 to September 2021, 1415 doses were administered. Over 50% of LPs were performed by neurology nurse practitioners without image guidance. Safety outcomes were excellent. A stratified approach resulted in successful intrathecal nusinersen delivery and efficient resource allocation for patients with SMA, with or without complex spinal anatomy.

Sections du résumé

BACKGROUND
Spinal deformity and prior spinal fusion pose technical challenges to lumbar puncture (LP) for nusinersen administration for patients with spinal muscular atrophy (SMA). In this retrospective study over two study phases, we evaluated (1) factors associated with difficult LP or unscheduled requirement for image guidance and (2) effectiveness of a triage pathway for selective use of image guidance and nonstandard techniques, particularly for patients with spinal instrumentation/fusion to the sacrum.
METHODS
With institutional review board approval, electronic health records, imaging, and administrative databases were analyzed for patients receiving nusinersen from January 2012 through September 2021. Descriptive statistics and univariate analyses were used.
RESULTS
From January 2012 to March 2018 (phase 1), among 82 patients with SMA, 461 of 464 (99.4%) LP attempts were successful. Univariate analyses associated difficulty with prior spinal instrumentation, higher body mass index, and severity of the spinal deformity. Based on this experience, starting in April 2018 (phase 2), 125 patients were triaged selectively for ultrasound, fluoroscopy, or Dyna computed tomography. Patients with spinal instrumentation/fusion to the sacrum were treated primarily via intrathecal ports (137 doses) or transforaminal LP (55 doses). From April 2018 through September 2021, 704 of 709 (99.3%) LPs were successful. In total from January 2012 to September 2021, 1415 doses were administered. Over 50% of LPs were performed by neurology nurse practitioners without image guidance. Safety outcomes were excellent.
CONCLUSIONS
A stratified approach resulted in successful intrathecal nusinersen delivery and efficient resource allocation for patients with SMA, with or without complex spinal anatomy.

Identifiants

DOI: 10.1016/j.pediatrneurol.2022.04.008 PMID: 35636280
pubmed: 35636280
pii: S0887-8994(22)00060-1
doi: 10.1016/j.pediatrneurol.2022.04.008
pii:
doi:

Substances chimiques

Lipopolysaccharides 0
Oligonucleotides 0
nusinersen 5Z9SP3X666

Types de publication

Journal Article Research Support, Non-U.S. Gov't

Langues

eng

Sous-ensembles de citation

IM

Pagination

33-40

Informations de copyright

Copyright © 2022. Published by Elsevier Inc.

Auteurs

Elena Madan (E)

Department of Anesthesiology, Critical Care & Pain Medicine, Boston Children's Hospital and Harvard Medical School, Boston, Massachusetts.

Sabrina Carrié (S)

Department of Anesthesiology, Critical Care & Pain Medicine, Boston Children's Hospital and Harvard Medical School, Boston, Massachusetts.

Carolina Donado (C)

Department of Anesthesiology, Critical Care & Pain Medicine, Boston Children's Hospital and Harvard Medical School, Boston, Massachusetts.

Kimberly Lobo (K)

Department of Anesthesiology, Critical Care & Pain Medicine, Boston Children's Hospital and Harvard Medical School, Boston, Massachusetts.

Michelle Souris (M)

Department of Neurology, Boston Children's Hospital and Harvard Medical School, Boston, Massachusetts.

Regina Laine (R)

Department of Neurology, Boston Children's Hospital and Harvard Medical School, Boston, Massachusetts.

Elizabeth Beers (E)

Department of Neurology, Boston Children's Hospital and Harvard Medical School, Boston, Massachusetts.

Laura Cornelissen (L)

Department of Anesthesiology, Critical Care & Pain Medicine, Boston Children's Hospital and Harvard Medical School, Boston, Massachusetts.

Basil T Darras (BT)

Department of Neurology, Boston Children's Hospital and Harvard Medical School, Boston, Massachusetts.

Anjali Koka (A)

Department of Anesthesiology, Critical Care & Pain Medicine, Boston Children's Hospital and Harvard Medical School, Boston, Massachusetts.

Bobbie Riley (B)

Department of Anesthesiology, Critical Care & Pain Medicine, Boston Children's Hospital and Harvard Medical School, Boston, Massachusetts.

Pradeep Dinakar (P)

Department of Anesthesiology, Critical Care & Pain Medicine, Boston Children's Hospital and Harvard Medical School, Boston, Massachusetts.

Scellig Stone (S)

Department of Neurosurgery, Boston Children's Hospital and Harvard Medical School, Boston, Massachusetts.

Brian Snyder (B)

Department of Orthopaedic Surgery, Boston Children's Hospital and Harvard Medical School, Boston, Massachusetts.

Robert J Graham (RJ)

Department of Anesthesiology, Critical Care & Pain Medicine, Boston Children's Hospital and Harvard Medical School, Boston, Massachusetts.

Horacio Padua (H)

Department of Radiology, Boston Children's Hospital and Harvard Medical School, Boston, Massachusetts.

Navil Sethna (N)

Department of Anesthesiology, Critical Care & Pain Medicine, Boston Children's Hospital and Harvard Medical School, Boston, Massachusetts.

Charles Berde (C)

Department of Anesthesiology, Critical Care & Pain Medicine, Boston Children's Hospital and Harvard Medical School, Boston, Massachusetts. Electronic address: charles.berde@childrens.harvard.edu.

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Classifications MeSH

questionsmedicales.fr › Eucaryotes › Animaux › Chordés › Vertébrés › Mammifères › Eutheria › Primates › Haplorhini › Catarrhini › Hominidae › Humains › Nusinersen for Patients With Spinal Muscular...
questionsmedicales.fr › Thérapeutique › Traitement médicamenteux › Voies d'administration de substances chimiques et des médicaments › Injections › Injections rachidiennes › Nusinersen for Patients With Spinal Muscular...
questionsmedicales.fr › Facteurs biologiques › Toxines biologiques › Toxines bactériennes › Endotoxines › Lipopolysaccharides › Nusinersen for Patients With Spinal Muscular...
questionsmedicales.fr › Maladies du système nerveux › Maladies neuromusculaires › Maladies du motoneurone › Amyotrophie spinale › Nusinersen for Patients With Spinal Muscular...
questionsmedicales.fr › Acides nucléiques, nucléotides et nucléosides › Nucléotides › Polynucléotides › Oligonucléotides › Nusinersen for Patients With Spinal Muscular...
questionsmedicales.fr › Environnement et santé publique › Santé publique › Méthodes épidémiologiques › Caractéristiques des études épidémiologiques › Études épidémiologiques › Études de cohortes › Études rétrospectives › Nusinersen for Patients With Spinal Muscular...