{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,6,29]],"date-time":"2026-06-29T17:16:02Z","timestamp":1782753362858,"version":"3.54.5"},"reference-count":39,"publisher":"Springer Science and Business Media LLC","issue":"1","license":[{"start":{"date-parts":[[2025,3,27]],"date-time":"2025-03-27T00:00:00Z","timestamp":1743033600000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0"},{"start":{"date-parts":[[2025,3,27]],"date-time":"2025-03-27T00:00:00Z","timestamp":1743033600000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0"}],"content-domain":{"domain":["link.springer.com"],"crossmark-restriction":false},"short-container-title":["BMC Pediatr"],"abstract":"<jats:title>Abstract<\/jats:title>\n          <jats:sec>\n            <jats:title>Background<\/jats:title>\n            <jats:p>Nusinersen was the first approved disease modifying therapy (DMT) for spinal muscular atrophy (SMA). Intrathecal administration of nusinersen enables drug delivery directly to the central nervous system, where the motor neurons are located. Per the package insert, individuals with SMA receive 4 loading doses of nusinersen followed by maintenance doses every 4 months thereafter. The aim of this analysis was to investigate the administration practices of and adherence to nusinersen in Chinese children with SMA.<\/jats:p>\n          <\/jats:sec>\n          <jats:sec>\n            <jats:title>Methods<\/jats:title>\n            <jats:p>Data were analyzed from a longitudinal, multicenter registry enrolling children with 5q-SMA in China. Information on nusinersen administration, including administration date, care setting, use of sedation and general anesthesia, method of administration, and use of imaging guidance before administration, was collected both retrospectively and prospectively. Adherence rate was calculated at dose and participant level. A dose was considered adherent if the inter-dose interval (for dose-level) and interval from the first dose (for participant-level) followed the standard dosing regimen, with a grace period of \u00b1\u20097 days for Dose 2 to 4 and \u00b1\u200928 days thereafter.<\/jats:p>\n          <\/jats:sec>\n          <jats:sec>\n            <jats:title>Results<\/jats:title>\n            <jats:p>A total of 385 participants receiving nusinersen with a total of 2,415 doses were included in the study. The median (interquartile range) number of doses administered per participant was 6 (5\u20137). Over 99% of intrathecal injections were given in an inpatient setting. Only a few (<jats:italic>n<\/jats:italic>\u2009=\u20093, 0.1%) required general anesthesia, while 9% (<jats:italic>n<\/jats:italic>\u2009=\u2009217) of doses were administered under the use of sedation. Interlaminar lumbar puncture (<jats:italic>n<\/jats:italic>\u2009=\u20092,407, 99.7%) was the most common method of administration, followed by cervical puncture (<jats:italic>n<\/jats:italic>\u2009=\u20095, 0.2%) and transforaminal lumbar puncture (<jats:italic>n<\/jats:italic>\u2009=\u20093, 0.1%). Over 90% of injections did not utilize any imaging guidance prior to administration, with ultrasound (<jats:italic>n<\/jats:italic>\u2009=\u2009142, 5.9%) being the most commonly used imaging guidance. The adherence rate was 95.7% (1,943\/2,030) at dose level and 81.0% (312\/385) at participant level. The median inter-dose intervals aligned well with the dosing schedule, with 14 days for Doses 2 and 3, 35 days for Dose 4, and 114\u2013124 days for maintenance doses thereafter.<\/jats:p>\n          <\/jats:sec>\n          <jats:sec>\n            <jats:title>Conclusions<\/jats:title>\n            <jats:p>Findings from the analysis demonstrated high real-world adherence to nusinersen in Chinese children with SMA.<\/jats:p>\n          <\/jats:sec>","DOI":"10.1186\/s12887-024-05290-0","type":"journal-article","created":{"date-parts":[[2025,3,27]],"date-time":"2025-03-27T03:42:15Z","timestamp":1743046935000},"update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":8,"title":["Administration practices of and adherence to nusinersen in children with spinal muscular atrophy: a multicenter disease registry study in China"],"prefix":"10.1186","volume":"25","author":[{"given":"Jing","family":"Peng","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Xiaoli","family":"Yao","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Rong","family":"Luo","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Xiuxia","family":"Wang","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Liwen","family":"Wu","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jianmin","family":"Zhong","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ruifeng","family":"Jin","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Xinguo","family":"Lu","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jianmin","family":"Liang","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Siqi","family":"Hong","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lin","family":"Yang","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Xiaoli","family":"Zhang","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Shanshan","family":"Mao","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Zhe","family":"Tao","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jun","family":"Hu","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Dan","family":"Sun","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Hua","family":"Wang","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Li","family":"Zhang","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yanyan","family":"Xia","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ken","family":"Chen","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yi","family":"Wang","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"297","published-online":{"date-parts":[[2025,3,27]]},"reference":[{"issue":"6","key":"5290_CR1","doi-asserted-by":"crossref","first-page":"409","DOI":"10.1136\/jmg.15.6.409","volume":"15","author":"J Pearn","year":"1978","unstructured":"Pearn J. 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The ethics committees of study sites contributing to this manuscript include: Medical Ethics Committee of Xiangya Hospital Central South University; Clinical Research and Experimental Animal Ethics Committee of The First Affiliated Hospital, Sun Yat-sen University; Clinical Trial Ethics Committee of West China Second University Hospital, Sichuan University; Clinical Trial Ethics Committee of The Second Hospital of Hebei Medical University; Medical Ethics Committee of Hunan Children\u2019s Hospital; Drug and Device Clinical Trial Ethics Committee of Jiangxi Provincial Children\u2019s Hospital; Clinical Trial Ethics Committee of Children\u2019s Hospital Affiliated to Shandong University; Medical Ethics Committee of Shenzhen Children\u2019s Hospital; Ethics Committee of The First Hospital of Jilin University; Ethics Committee of Children\u2019s Hospital of Chongqing Medical University; Medical Ethics Committee of The Second Affiliated Hospital of Xi\u2019an Jiaotong University; Drug Clinical Trial Ethics Committee of The Third Affiliated Hospital of Zhengzhou University; Medical Ethics Committee of Children\u2019s Hospital of Zhejiang University School of Medicine; Drug Clinical Trial Ethics Review Committee of Dalian Women and Children\u2019s Medical Group; Ethics Committee of Fujian Medical University Union Hospital; Ethics Committee of Wuhan Children\u2019s Hospital, Tongji Medical College Huazhong University of Science & Technology; Medical Ethics Committee of Shengjing Hospital of China Medical University; Ethics Committee of Children\u2019s Hospital of Fudan University. The registry was registered on clinicaltrials.gov (NCT05042921).","order":2,"name":"Ethics","group":{"name":"EthicsHeading","label":"Ethics approval and consent to participate"}},{"value":"Not applicable.","order":3,"name":"Ethics","group":{"name":"EthicsHeading","label":"Consent for publication"}},{"value":"L.Z. is an employee of and holder of stock\/stock options in Biogen. Y.X. was previously an employee of Biogen when this manuscript was developed. K.C. is an employee of IQVIA. Other authors report no relevant disclosures.","order":4,"name":"Ethics","group":{"name":"EthicsHeading","label":"Competing interests"}}],"article-number":"239"}}