Neuropediatrics
DOI: 10.1055/s-0040-1715481
Original Article

Multicenter Experience with Nusinersen Application via an Intrathecal Port and Catheter System in Spinal Muscular Atrophy

1  Department of Neuropediatrics, University Children's Hospital of Saarland, Homburg, Germany
,
Andreas Hahn
2  Department of Child Neurology, University Hospital Giessen, Giessen, Germany
,
Oliver Schwartz
3  Department of Neuropediatrics, Münster University Hospital, Münster, Germany
,
Steffan Linsler
4  Department of Neurosurgery, University Children's Hospital of Saarland, Homburg, Germany
,
Sascha Meyer
1  Department of Neuropediatrics, University Children's Hospital of Saarland, Homburg, Germany
,
Malgorzata Kolodziej
5  Department of Neurosurgery, University Hospital Giessen, Giessen, Germany
,
Cornelia Koehler
6  Department of Neuropediatrics, University Hospitals of the Ruhr University of Bochum, Bochum, Germany
› Author Affiliations

Abstract

Nusinersen, an antisense oligonucleotide enhancing the production of the survival motor neuron protein, is approved for the treatment of spinal muscular atrophy (SMA) but requires repetitive lumbar punctures. Application via a subcutaneous port connected to a permanent intrathecal catheter has been proposed as an alternative for patients with severe scoliosis, spinal fusion, or comorbidities, rendering serial interlaminar punctures complicated and risky. Since experience with this technique is sparse and follow-up data are lacking, we assessed feasibility, safety, and tolerability of this approach in eight patients with SMA II/SMA III receiving Nusinersen in a multicenter study. Median age at port implantation was 21 years (range: 10–30 years), and median follow-up time thereafter was 19 months (range: 7–24 months). Leakage of the port catheter occurred in two patients, promptly resolving after resuturing. No further complications such as infection, dislocation, kinking, or obstruction of the port were noted in any of the patients. These findings suggest that application via an intrathecal port and catheter system represents a safe and feasible option for Nusinersen treatment in subjects with SMA. However, to detect rare adverse events longer term follow-up in a larger study cohort is warranted.



Publication History

Received: 17 January 2020

Accepted: 22 April 2020

Publication Date:
22 October 2020 (online)

Georg Thieme Verlag KG
Stuttgart · New York