{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,1,27]],"date-time":"2026-01-27T08:22:00Z","timestamp":1769502120360,"version":"3.49.0"},"reference-count":21,"publisher":"Georg Thieme Verlag KG","issue":"03","content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Neuropediatrics"],"published-print":{"date-parts":[[2021,6]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:p>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.<\/jats:p>","DOI":"10.1055\/s-0040-1718916","type":"journal-article","created":{"date-parts":[[2020,12,5]],"date-time":"2020-12-05T01:37:35Z","timestamp":1607132255000},"page":"179-185","source":"Crossref","is-referenced-by-count":10,"title":["Intrathecal Administration of Nusinersen in Pediatric SMA Patients with and without Spine Deformities: Experiences and Challenges over 3 Years in a Single Center"],"prefix":"10.1055","volume":"52","author":[{"given":"Jessika","family":"Johannsen","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, University Medical Center Hamburg-Eppendorf, Hamburg, Germany"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Deike","family":"Weiss","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, University Medical Center Hamburg-Eppendorf, Hamburg, Germany"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Friderike","family":"Schlenker","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, University Medical Center Hamburg-Eppendorf, Hamburg, Germany"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Michael","family":"Groth","sequence":"additional","affiliation":[{"name":"Department of Pediatric Radiology, University Medical Center Hamburg-Eppendorf, Hamburg, Germany"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Jonas","family":"Denecke","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, University Medical Center Hamburg-Eppendorf, Hamburg, Germany"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"194","published-online":{"date-parts":[[2020,12,4]]},"reference":[{"issue":"01","key":"ref1","doi-asserted-by":"crossref","first-page":"155","DOI":"10.1016\/0092-8674(95)90460-3","article-title":"Identification and characterization of a spinal muscular atrophy-determining gene","volume":"80","author":"S Lefebvre","year":"1995","journal-title":"Cell"},{"issue":"20","key":"ref3","doi-asserted-by":"crossref","first-page":"923","DOI":"10.1212\/WNL.0000000000006502","article-title":"Evidence in focus: nusinersen use in spinal muscular atrophy: report of the Guideline Development, Dissemination, and Implementation Subcommittee of the American Academy of Neurology","volume":"91","author":"D Michelson","year":"2018","journal-title":"Neurology"},{"issue":"18","key":"ref4","doi-asserted-by":"crossref","first-page":"1723","DOI":"10.1056\/NEJMoa1702752","article-title":"Nusinersen versus sham control in infantile-onset spinal muscular atrophy","volume":"377","author":"R S Finkel","year":"2017","journal-title":"N Engl J Med"},{"issue":"07","key":"ref5","doi-asserted-by":"crossref","first-page":"625","DOI":"10.1056\/NEJMoa1710504","article-title":"Nusinersen versus sham control in later-onset spinal muscular atrophy","volume":"378","author":"E Mercuri","year":"2018","journal-title":"N Engl J Med"},{"issue":"03","key":"ref6","doi-asserted-by":"crossref","first-page":"307","DOI":"10.3233\/JND-190403","article-title":"AVXS-101 (Onasemnogene Abeparvovec) for SMA1: comparative study with a prospective natural history cohort","volume":"6","author":"S A Al-Zaidy","year":"2019","journal-title":"J Neuromuscul Dis"},{"key":"ref7","first-page":"6","article-title":"AVXS-101 gene-replacement therapy (GRT) for spinal muscular atrophy type 1 (SMA1): pivotal phase 3 study (STR1VE)","volume":"P1","author":"J W 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Wurster","year":"2019","journal-title":"J Neurol"},{"issue":"05","key":"ref11","doi-asserted-by":"crossref","first-page":"986","DOI":"10.3174\/ajnr.A5596","article-title":"Transforaminal lumbar puncture: an alternative technique in patients with challenging access","volume":"39","author":"D R Nascene","year":"2018","journal-title":"AJNR Am J Neuroradiol"},{"issue":"07","key":"ref12","doi-asserted-by":"crossref","first-page":"e620","DOI":"10.1212\/WNL.0000000000006006","article-title":"Cervical puncture to deliver nusinersen in patients with spinal muscular atrophy","volume":"91","author":"A Veerapandiyan","year":"2018","journal-title":"Neurology"},{"issue":"01","key":"ref13","doi-asserted-by":"crossref","first-page":"136","DOI":"10.1007\/s00247-018-4240-7","article-title":"Ultrasound-guided cervical puncture for nusinersen administration in adolescents","volume":"49","author":"C B Ortiz","year":"2019","journal-title":"Pediatr Radiol"},{"key":"ref14","doi-asserted-by":"crossref","first-page":"464","DOI":"10.1212\/WNL.90.15_supplement.P4.464","article-title":"Use of Ommaya reservoir with a thoracic spinal catheter for intrathecal delivery of nusinersen in a patient with spinal muscular atrophy type 2","volume":"90","author":"A Lakhotia","year":"2018","journal-title":"Neurology"},{"issue":"10","key":"ref15","doi-asserted-by":"crossref","first-page":"e610","DOI":"10.1097\/BPO.0000000000001247","article-title":"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","volume":"38","author":"K A Strauss","year":"2018","journal-title":"J Pediatr Orthop"},{"issue":"03","key":"ref16","doi-asserted-by":"crossref","first-page":"392","DOI":"10.1007\/s00247-017-4031-6","article-title":"Transforaminal intrathecal delivery of nusinersen using cone-beam computed tomography for children with spinal muscular atrophy and extensive surgical instrumentation: early results of technical success and safety","volume":"48","author":"J J Weaver","year":"2018","journal-title":"Pediatr Radiol"},{"key":"ref17","first-page":"1756286419887616","article-title":"Intrathecal nusinersen administration in adult spinal muscular atrophy patients with complex spinal anatomy","volume":"13","author":"I Cordts","year":"2020","journal-title":"Ther Adv Neurol Disorder"},{"key":"ref18","doi-asserted-by":"publisher","DOI":"10.5152\/dir.2020.19607","article-title":"Nusinersen injections in adults and children with spinal muscular atrophy: a single-center experience","author":"C \u00d6z\u00fctemiz","year":"2020","journal-title":"Diagn Interv Radiol"},{"key":"ref19","doi-asserted-by":"crossref","first-page":"1166","DOI":"10.3389\/fneur.2019.01166","article-title":"Clinical implication of dosimetry of computed tomography-and fluoroscopy-guided intrathecal therapy with nusinersen in adult patients with 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