Ultrasound‐assisted and landmark‐based nusinersen delivery in spinal muscular atrophy adults: A retrospective analysis

Author:

Zanfini Bruno Antonio1ORCID,Patanella Agata Katia2,Vassalli Francesco3,Catarci Stefano1,Pane Marika45,Frassanito Luciano1,Biancone Matteo1,Di Muro Mariangela1ORCID,Bravetti Chiara4,Mercuri Eugenio Maria45,Sabatelli Mario2345,Draisci Gaetano12345

Affiliation:

1. Department of Emergency, Anesthesiological and Reanimation Sciences Fondazione Policlinico Universitario Agostino Gemelli IRCCS Rome Italy

2. Centro Clinico NeMO Adulti Fondazione Policlinico Universitario Agostino Gemelli IRCCS Rome Italy

3. Obstetric Anesthesia, Department of Critical Care and Perinatal Medicine Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) Istituto Giannina Gaslini Genoa Italy

4. Centro Clinico NeMO Pediatrico Fondazione Policlinico Universitario A Gemelli IRCCS Rome Italy

5. Università Cattolica del Sacro Cuore Rome Italy

Abstract

AbstractIntroduction/PurposeNusinersen, the first treatment approved for all spinal muscular atrophy (SMA) types, is administered intrathecally through lumbar puncture. We used ultrasound assistance or a landmark‐based technique to access the lumbar intrathecal space in adult SMA patients. This study aimed to evaluate the technical success and adverse events (AEs) in such patients using either technique over a long observation period.MethodsFifty‐one adult patients received 507 consecutive interlaminar nusinersen administrations. Patients presented with both ‘uncomplicated spines’ or ‘complicated spines’; two patients had previous back surgery. Technical success and AEs were recorded using either technique. A generalised linear mixed model was applied to evaluate predictors of technical success and complications.ResultsAn overall success rate of 99.6%, with only two procedures failing to reach the intrathecal space, and an overall optimal procedure rate of 90.3% have been reported. A total of 455 procedures (89.7%) were uneventfully performed. One (0.2%) case of severe AE (puncture of a bulky abdominal annexal cyst) was recorded. Twenty‐seven episodes (5.3%) of post‐dural puncture headache (PDPH) and 24 episodes (4.7%) of radicular or back pain, both successfully treated with medical therapy, have also been reported. Technical success was significantly associated with ‘complicated spines’ (P = 0.022) and the use of ultrasound assistance (P = 0.01), and the use of ultrasound was the only independent predictor of uncomplicated procedures (P = 0.007).DiscussionIn adult patients with SMA both landmark‐based and ultrasound‐assisted techniques are safe and effective even in the long term. The use of assistance is associated with technical success and can predict uncomplicated procedures.ConclusionOur results support the use of ultrasonography in order to improve the success and reduce the burden of nusinersen intrathecal administration.

Publisher

Wiley

Reference42 articles.

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4. Spinal muscular atrophy

5. Preliminary Safety and Tolerability of a Novel Subcutaneous Intrathecal Catheter System for Repeated Outpatient Dosing of Nusinersen to Children and Adults With Spinal Muscular Atrophy

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