Improved upper limb function in non-ambulant children with SMA type 2 and 3 during nusinersen treatment: a prospective 3-years SMArtCARE registry study
Rattachement africain : de, at, ca. Niveau de preuve : code pays fourni par la source.
Le résumé fourni par la source
BACKGROUND: The development and approval of disease modifying treatments have dramatically changed disease progression in patients with spinal muscular atrophy (SMA). Nusinersen was approved in Europe in 2017 for the treatment of SMA patients irrespective of age and disease severity. Most data on therapeutic efficacy are available for the infantile-onset SMA. For patients with SMA type 2 and type 3, there is still a lack of sufficient evidence and long-term experience for nusinersen treatment. Here, we report data from the SMArtCARE registry of non-ambulant children with SMA type 2 and typen 3 under nusinersen treatment with a follow-up period of up to 38 months. METHODS: SMArtCARE is a disease-specific registry with data on patients with SMA irrespective of age, treatment regime or disease severity. Data are collected during routine patient visits as real-world outcome data. This analysis included all non-ambulant patients with SMA type 2 or 3 below 18 years of age before initiation of treatment. Primary outcomes were changes in motor function evaluated with the Hammersmith Functional Motor Scale Expanded (HFMSE) and the Revised Upper Limb Module (RULM). RESULTS: Data from 256 non-ambulant, pediatric patients with SMA were included in the data analysis. Improvements in motor function were more prominent in upper limb: 32.4% of patients experienced clinically meaningful improvements in RULM and 24.6% in HFMSE. 8.6% of patients gained a new motor milestone, whereas no motor milestones were lost. Only 4.3% of patients showed a clinically meaningful worsening in HFMSE and 1.2% in RULM score. CONCLUSION: Our results demonstrate clinically meaningful improvements or stabilization of disease progression in non-ambulant, pediatric patients with SMA under nusinersen treatment. Changes were most evident in upper limb function and were observed continuously over the follow-up period. Our data confirm clinical trial data, while providing longer follow-up, an increased number of treated patients, and a wider range of age and disease severity.
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Le contrôle bibliographique ouvert
DOI retrouvé dans Crossref DOI retrouvé ; titre concordant.
- Titre Crossref
- Improved upper limb function in non-ambulant children with SMA type 2 and 3 during nusinersen treatment: a prospective 3-years SMArtCARE registry study
- Date Crossref
- 23/10/2022
- Éditeur
- Springer Science and Business Media LLC
- Type
- journal-article
Ce recoupement confirme des métadonnées liées au DOI. Il ne confirme ni la méthode ni les conclusions de l’étude, et il ne compte pas comme une seconde source scientifique indépendante.
Où se fait cette recherche
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University of Freiburg pays non établi dans la noticeUniversité ou école supérieure
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University Medical Center Freiburg pays non établi dans la noticeÉtablissement de santé
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Essen University Hospital pays non établi dans la noticeOrganisme public
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University of Duisburg-Essen Department of Neuropediatrics and Neuromuscular Centre for Children and Adolescents pays non établi dans la noticeUniversité ou école supérieure
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Ludwig-Maximilians-Universität München pays non établi dans la noticeUniversité ou école supérieure
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University of Rostock pays non établi dans la noticeUniversité ou école supérieure
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LMU Klinikum pays non établi dans la noticeÉtablissement de santé
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Innsbruck Medical University pays non établi dans la noticeUniversité ou école supérieure
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Universität Innsbruck pays non établi dans la noticeUniversité ou école supérieure
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Ordensklinikum Linz Barmherzige Schwestern pays non établi dans la noticeÉtablissement de santé
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AGO Austria pays non établi dans la noticeOrganisation à but non lucratif
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Technical University of Munich Department of Neurology pays non établi dans la noticeUniversité ou école supérieure
University of Freiburg, University Medical Center Freiburg et Essen University Hospital, avec 9 autres affiliations.
Une affiliation ne permet pas de déduire la nationalité d’un auteur.