{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,6,26]],"date-time":"2026-06-26T06:41:52Z","timestamp":1782456112857,"version":"3.54.5"},"reference-count":33,"publisher":"Becaris Publishing Limited","issue":"7","content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["J. Comp. Eff. Res."],"published-print":{"date-parts":[[2024,7]]},"abstract":"<jats:p>\n            <jats:bold>Aim:<\/jats:bold>\n            Nusinersen, administered by intrathecal injection at a dose of 12\u00a0mg, is indicated across all ages for the treatment of spinal muscular atrophy (SMA). Evidence on real-world healthcare resource use (HRU) and costs among patients taking nusinersen remains limited. This study aimed to evaluate real-world HRU and costs associated with nusinersen use through US claims databases.\n            <jats:bold>Patients &amp; methods:<\/jats:bold>\n            Using the Merative\u2122 MarketScan\n            <jats:sup>\u00ae<\/jats:sup>\n            Research Databases, patients with SMA receiving nusinersen were identified from commercial (January 2017 to June 2020) and Medicaid claims (January 2017 to December 2019). Those likely to have complete information on the date of nusinersen initiation and continuous enrollment 12\u00a0months pre- and post-index (first record of nusinersen treatment) were retained. Number and costs (US$ 2020) of inpatient admissions and emergency department (ED) visits, unrelated to nusinersen administration, were evaluated for 12\u00a0months pre- and post-nusinersen initiation and stratified by age: pediatric (&lt;18\u00a0years) and adult (\u226518\u00a0years).\n            <jats:bold>Results:<\/jats:bold>\n            Overall, 103 individuals treated with nusinersen were retained: 59 were pediatric (mean age [range]: 9 [1\u201317] years), and 44 were adults (30 [18\u201363] years). Inpatient admissions decreased by 41% for pediatrics and 67% for adults in the 12\u00a0months post-treatment versus the 12\u00a0months pre-treatment. Average inpatient admission costs per patient for the pediatric cohort decreased by 63% ($22,903 vs $8466) and by 79% ($13,997 vs $2899) for the adult cohort when comparing the 12\u00a0months pre-index with the 12\u00a0months post-index period. Total ED visits and ED visit costs decreased by 8% and 35%, respectively, for the overall cohort over the 12-month period pre- and post-index.\n            <jats:bold>Conclusion:<\/jats:bold>\n            Using US claims databases, nusinersen treatment in pediatric and adult patients was associated with reductions in HRU and costs over a 12-month period post-treatment initiation relative to the pre-treatment period.\n          <\/jats:p>","DOI":"10.57264\/cer-2023-0187","type":"journal-article","created":{"date-parts":[[2024,7,4]],"date-time":"2024-07-04T10:45:30Z","timestamp":1720089930000},"source":"Crossref","is-referenced-by-count":4,"title":["Evaluation of inpatient and emergency department healthcare resource utilization and costs pre- and post-nusinersen for the treatment of spinal muscular atrophy using United States claims"],"prefix":"10.57264","volume":"13","author":[{"given":"Cong","family":"Zhu","sequence":"first","affiliation":[{"name":"Biogen, Cambridge, MA 02142, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Craig","family":"Zaidman","sequence":"additional","affiliation":[{"name":"Department of Neurology, Washington University School of Medicine, St. Louis, MO 63110, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Bora","family":"Youn","sequence":"additional","affiliation":[{"name":"Biogen, Cambridge, MA 02142, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Angela D","family":"Paradis","sequence":"additional","affiliation":[{"name":"Biogen, Cambridge, MA 02142, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Stephanie","family":"Raynaud","sequence":"additional","affiliation":[{"name":"Biogen, Cambridge, MA 02142, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Bridget A","family":"Neville","sequence":"additional","affiliation":[{"name":"Biogen, Cambridge, MA 02142, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Nicole B","family":"Johnson","sequence":"additional","affiliation":[{"name":"Biogen, Cambridge, MA 02142, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"36897","reference":[{"issue":"1","key":"e_1_3_7_3_2","doi-asserted-by":"crossref","first-page":"52","DOI":"10.1038\/s41572-022-00380-8","article-title":"Spinal muscular atrophy","volume":"8","author":"Mercuri E","year":"2022","unstructured":"Mercuri E, Sumner CJ, Muntoni F, Darras BT, Finkel RS. Spinal muscular atrophy. Nat. Rev. Dis. Primers 8(1), 52 (2022).","journal-title":"Nat. Rev. Dis. Primers"},{"key":"e_1_3_7_4_2","doi-asserted-by":"crossref","first-page":"1057","DOI":"10.1016\/B978-0-323-37101-8.00139-9","volume-title":"Swaiman's Pediatric Neurology (6th Edition).","author":"Darras BT","year":"2017","unstructured":"Darras BT, Monani UR, De Vivo DC. Genetic disorders affecting the motor neuron: spinal muscular atrophy. In: Swaiman's Pediatric Neurology (6th Edition). Swaiman KF, Ashwal S, Ferriero DMet al. (Eds). Elsevier, The Netherlands, 1057\u20131064 (2017)."},{"issue":"9","key":"e_1_3_7_5_2","doi-asserted-by":"crossref","first-page":"529","DOI":"10.1038\/gt.2017.52","article-title":"The clinical landscape for SMA in a new therapeutic era","volume":"24","author":"Talbot K","year":"2017","unstructured":"Talbot K, Tizzano EF. The clinical landscape for SMA in a new therapeutic era. Gene Ther. 24(9), 529\u2013533 (2017).","journal-title":"Gene Ther."},{"issue":"9","key":"e_1_3_7_6_2","doi-asserted-by":"crossref","first-page":"810","DOI":"10.1212\/WNL.0000000000000741","article-title":"Observational study of spinal muscular atrophy type I and implications for clinical trials","volume":"83","author":"Finkel RS","year":"2014","unstructured":"Finkel RS, McDermott MP, Kaufmann P Observational study of spinal muscular atrophy type I and implications for clinical trials. Neurology 83(9), 810\u2013817 (2014).","journal-title":"Neurology"},{"issue":"4","key":"e_1_3_7_7_2","doi-asserted-by":"crossref","first-page":"473","DOI":"10.1007\/s40265-017-0711-7","article-title":"Nusinersen: first global approval","volume":"77","author":"Hoy SM","year":"2017","unstructured":"Hoy SM. Nusinersen: first global approval. Drugs 77(4), 473\u2013479 (2017).","journal-title":"Drugs"},{"key":"e_1_3_7_8_2","doi-asserted-by":"crossref","first-page":"175628561875450","DOI":"10.1177\/1756285618754501","article-title":"Advances in spinal muscular atrophy therapeutics","volume":"11","author":"Parente V","year":"2018","unstructured":"Parente V, Corti S. Advances in spinal muscular atrophy therapeutics. Ther. Adv. Neurol. Disord. 11, 1756285618754501 (2018).","journal-title":"Ther. Adv. Neurol. Disord."},{"key":"e_1_3_7_9_2","unstructured":"Food and Drug Administration. Spinraza prescribing information. Available at: https:\/\/www.accessdata.fda.gov\/drugsatfda_docs\/label\/2023\/209531s011lbl.pdf (Accessed: 8 August 2023)."},{"key":"e_1_3_7_10_2","unstructured":"European Medicines Agency. Spinraza summary of product characteristics. 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Engl. J. Med. 377(18), 1723\u20131732 (2017).","journal-title":"N. Engl. J. Med."},{"issue":"11","key":"e_1_3_7_14_2","doi-asserted-by":"crossref","first-page":"842","DOI":"10.1016\/j.nmd.2019.09.007","article-title":"Nusinersen initiated in infants during the presymptomatic stage of spinal muscular atrophy: interim efficacy and safety results from the Phase II NURTURE study","volume":"29","author":"De Vivo DC","year":"2019","unstructured":"De Vivo DC, Bertini E, Swoboda KJ Nusinersen initiated in infants during the presymptomatic stage of spinal muscular atrophy: interim efficacy and safety results from the Phase II NURTURE study. Neuromuscul. Disord. 29(11), 842\u2013856 (2019).","journal-title":"Neuromuscul. 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Muscle Nerve 68(2), 157\u2013170 (2023).","journal-title":"Muscle Nerve"},{"issue":"21","key":"e_1_3_7_18_2","doi-asserted-by":"crossref","first-page":"e2492","DOI":"10.1212\/WNL.0000000000007527","article-title":"Nusinersen in later-onset spinal muscular atrophy: long-term results from the phase 1\/2 studies","volume":"92","author":"Darras BT","year":"2019","unstructured":"Darras BT, Chiriboga CA, Iannaccone ST Nusinersen in later-onset spinal muscular atrophy: long-term results from the phase 1\/2 studies. 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