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Open Access 01-12-2019 | Spinal Muscular Atrophy | Research

SMArtCARE - A platform to collect real-life outcome data of patients with spinal muscular atrophy

Authors: Astrid Pechmann, Kirsten König, Günther Bernert, Kristina Schachtrup, Ulrike Schara, David Schorling, Inge Schwersenz, Sabine Stein, Adrian Tassoni, Sibylle Vogt, Maggie C. Walter, Hanns Lochmüller, Janbernd Kirschner

Published in: Orphanet Journal of Rare Diseases | Issue 1/2019

Abstract

Background

Survival and quality of life for patients affected by spinal muscular atrophy (SMA) are thought to have improved over the last decade due to changes in care. In addition, targeted treatments for SMA have been developed based on a better understanding of the molecular pathology. In 2016 and 2017, nusinersen was the first drug to be approved for treatment of all types of SMA in the United States and in Europe based on well-controlled clinical trials in a small subgroup of pediatric SMA 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.

Methods

Within SMArtCARE, we conduct a prospective, multicenter non-randomized registration and outcome study. SMArtCARE collects 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 in Germany, Austria and Switzerland. All data are collected during routine patient visits. Items for data collection are aligned with the international consensus for SMA registries. Data analysis is carried out independent of commercial partners.

Conclusion

A prospective monitoring of all SMA patients will lead to a better understanding of the natural history of SMA and the influence of drug treatment. This is crucial to improve the care of SMA patients. Further, we will establish a network for neuromuscular centers to share experience with SMA patients and to promote research projects on SMA.

Trial registration

German Clinical Trials Register (“Deutsches Register klinischer Studien”) DRKS00012699. Registered 09 August 2018. https://www.drks.de/drks_web/navigate.do?navigationId=trial.HTML&TRIAL_ID=DRKS00012699.

Available only for authorised users

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Title: SMArtCARE - A platform to collect real-life outcome data of patients with spinal muscular atrophy
Authors: Astrid Pechmann
Kirsten König
Günther Bernert
Kristina Schachtrup
Ulrike Schara
David Schorling
Inge Schwersenz
Sabine Stein
Adrian Tassoni
Sibylle Vogt
Maggie C. Walter
Hanns Lochmüller
Janbernd Kirschner
Publication date: 01-12-2019
Publisher: BioMed Central
Keyword: Spinal Muscular Atrophy
Published in: Orphanet Journal of Rare Diseases / Issue 1/2019
Electronic ISSN: 1750-1172
DOI: https://doi.org/10.1186/s13023-019-0998-4

ACC 2024 Congress

Springer Medicine

SMArtCARE - A platform to collect real-life outcome data of patients with spinal muscular atrophy

Abstract

Background

Methods

Conclusion

Trial registration

ACC 2024 Congress

Springer Medicine

Abstract

Background

Methods

Conclusion

Trial registration

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