Treatment with Nusinersen - Challenges Regarding the Indication for Children with SMA Type 1

Research output: Contribution to journalResearch articleContributedpeer-review

Contributors

  • Astrid Pechmann - , University of Freiburg (Author)
  • Matthias Baumann - , Innsbruck Medical University (Author)
  • Günther Bernert - , Kaiser Franz Josef Hospital (Author)
  • Marina Flotats-Bastardas - , Saarland University (Author)
  • Ursula Gruber-Sedlmayr - , Medical University of Graz (Author)
  • Maja Von Der Hagen - , Department of Paediatrics, Division of Neuropediatrics (Author)
  • Oswald Hasselmann - , Children’s Hospital of Eastern Switzerland (Author)
  • Elke Hobbiebrunken - , University of Göttingen (Author)
  • Veronka Horber - , University of Tübingen (Author)
  • Jessika Johannsen - , University of Hamburg (Author)
  • Anna Kellersmann - , Kaiser Franz Josef Hospital (Author)
  • Cornelia Köhler - , Ruhr University Bochum (Author)
  • Arpad Von Moers - , DRK Klinikum Westend (Author)
  • Wolfgang Müller-Felber - , Ludwig Maximilian University of Munich (Author)
  • Barbara Plecko - , Medical University of Graz (Author)
  • Christof Reihle - , Klinikum Stuttgart (Author)
  • Kurt Schlachter - , State Hospital of Bregenz (LKH Bregenz) (Author)
  • Gudrun Schreiber - , Klinikum Kassel GmbH (Author)
  • Oliver Schwartz - , University of Münster (Author)
  • Martin Smitka - , Department of Paediatrics, Division of Neuropediatrics (Author)
  • Elisabeth Steiner - , Klinikum Wels - Grieskirchen GmbH (Author)
  • Corinna Stoltenburg - , Charité – Universitätsmedizin Berlin (Author)
  • Burkhard Stüve - , Childrens Hospital (Author)
  • Manuela Theophil - , DRK Klinikum Westend (Author)
  • Claudia Weiß - , Charité – Universitätsmedizin Berlin (Author)
  • Gert Wiegand - , Asklepios Clinic Hamburg Nord-Heidberg (Author)
  • Ekkehard Wilichowski - , University of Göttingen (Author)
  • Benedikt Winter - , Ulm University (Author)
  • Wolfgang Wittmann - , Kinderzentrum St. Martin (Author)
  • Ulrike Schara - , University of Duisburg-Essen (Author)
  • Janbernd Kirschner - , University of Freiburg, University of Bonn (Author)

Abstract

The natural history of patients with spinal muscular atrophy (SMA) has changed due to advances in standard care and development of targeted treatments. Nusinersen was the first drug approved for the treatment of all SMA patients. The transfer of clinical trial data into a real-life environment is challenging, especially regarding the advice of patients and families to what extent they can expect a benefit from the novel treatment. We report the results of a modified Delphi consensus process among child neurologists from Germany, Austria and Switzerland about the indication or continuation of nusinersen treatment in children with SMA type 1 based on different clinical case scenarios.

Details

Original languageEnglish
Pages (from-to)41-46
Number of pages6
JournalJournal of neuromuscular diseases
Volume7
Issue number1
Publication statusPublished - 2020
Peer-reviewedYes

External IDs

PubMed 31744015

Keywords

ASJC Scopus subject areas