Neuropediatrics 2019; 50(S 02): S1-S55
DOI: 10.1055/s-0039-1698258
Poster Presentations
Poster Area GNP Neuromuscular Diseases/Varia 2
Georg Thieme Verlag KG Stuttgart · New York

SMArtCARE - Real-world-data Collection of Patients with Spinal Muscular Atrophy

Astrid Pechmann
1   Universitätsklinikum Freiburg, Zentrum für Kinder- und Jugendmedizin, Klinik für Neuropädiatrie und Muskelerkrankungen, Freiburg, Germany
,
Günther Bernert
2   G.v. Preyer’sches Kinderspital, Neuropädiatrie, Wien,Germany
,
Ulrike Schara
3   Universitätsklinikum Duisburg-Essen, Neuropädiatrie, Essen,Germany
,
Inge Schwersenz
4   Deutsche Gesellschaft für Muskelkranke, -, Freiburg,Germany
,
Maggie C. Walter
5   Friedrich-Baur-Institut, Klinik für Neurologie, München, Germany
,
Hanns Lochmüller
1   Universitätsklinikum Freiburg, Zentrum für Kinder- und Jugendmedizin, Klinik für Neuropädiatrie und Muskelerkrankungen, Freiburg, Germany
,
Janbernd Kirschner
6   Universitätsklinikum Bonn, Neuropädiatrie, Bonn,Germany
› Author Affiliations
Further Information

Publication History

Publication Date:
11 September 2019 (online)

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    Based upon a better understanding of the molecular genetics of spinal muscular atrophy (SMA), targeted treatments for SMA have been developed in preclinical and clinical studies. Nusinersen is the first drug to be approved in the United States and in Europe since 2016 and 2017 for the treatment of SMA. Furthermore, the natural history of SMA changed over the last decade due to changes in care. The approval of nusinersen for all types of SMA is based on well-controlled clinical trial data of a small subgroup of pediatric SMA patients. Post-market surveillance and data capture are crucial to evaluate outcomes and the effect of treatment after prolonged treatment periods and in a broader spectrum of patients. Systems are required to monitor treated and untreated SMA patients in a real-life environment to optimize treatment and care, and to provide outcome data to regulators, payers, and the SMA community.

    SMArtCARE aims to collect longitudinal data on all available SMA patients independent of their actual treatment regime as disease specific SMA registry. For this purpose, we provide an online platform for SMA patients seen by health-care providers. Data are collected during routine patient visits as real-world-outcome data and items for data collection are aligned with the international consensus for SMA registries. Data analysis is carried out independent of commercial partners. More than 50 neuromuscular centers in Germany, Austria, and Switzerland have already confirmed their participation in SMArtCARE.

    Prospective monitoring of all SMA patients will lead to a better understanding of the natural history of SMA and the influence of different drug treatments. This is crucial to improve care of SMA patients. Furthermore, SMArtCARE strives to establish a research network among neuromuscular centers to share experience with SMA patients and to collaborate in research projects.


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    No conflict of interest has been declared by the author(s).