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Neuropediatrics 2021; 52(03): 179-185
DOI: 10.1055/s-0040-1718916
Original Article

Intrathecal Administration of Nusinersen in Pediatric SMA Patients with and without Spine Deformities: Experiences and Challenges over 3 Years in a Single Center

Jessika Johannsen
1   Department of Pediatrics, University Medical Center Hamburg-Eppendorf, Hamburg, Germany
,
Deike Weiss
1   Department of Pediatrics, University Medical Center Hamburg-Eppendorf, Hamburg, Germany
,
Friderike Schlenker
1   Department of Pediatrics, University Medical Center Hamburg-Eppendorf, Hamburg, Germany
,
Michael Groth
2   Department of Pediatric Radiology, University Medical Center Hamburg-Eppendorf, Hamburg, Germany
,
Jonas Denecke
1   Department of Pediatrics, University Medical Center Hamburg-Eppendorf, Hamburg, Germany
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Abstract

Spinal muscular atrophy (SMA) is a rare neurodegenerative disease leading to progressive muscular atrophy, respiratory failure, and premature death. Secondary thorax and spine deformities are frequent. In July 2017, the antisense oligonucleotide nusinersen (Spinraza) was approved for the recurrent lifelong intrathecal treatment of SMA in Europe. Lumbar punctures are challenging especially in SMA patients with severe spine deformities and after spine surgery. In the light of alternative SMA therapies that are available or are expected to be available soon and which are administered orally or via one-time infusion, an appraisal of the established therapy is significant. Discussion about which therapy is the best for each individual patient will have to include not only the safety and efficacy of data but also the application form and its burden for the patient and the health care system. Therefore, we analyzed our 3-year experiences and challenges with 478 lumbar puncture procedures in 61 pediatric SMA patients with and without spine deformities or instrumentation.

Keywords

spinal muscular atrophy - nusinersen - intrathecal application - spine deformities - fluoroscopy


Publication History

Received: 27 April 2020

Accepted: 02 September 2020

Article published online:
04 December 2020

© 2020. Thieme. All rights reserved.

Georg Thieme Verlag KG
Rüdigerstraße 14, 70469 Stuttgart, Germany

 

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