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

Astrid Pechmann¹, Matthias Baumann², Günther Bernert³, Marina Flotats-Bastardas⁴, Ursula Gruber-Sedlmayr⁵, Maja von der Hagen⁶, Oswald Hasselmann⁷, Elke Hobbiebrunken⁸, Veronka Horber⁹, Jessika Johannsen¹⁰, Anna Kellersmann³, Cornelia Köhler¹¹, Arpad von Moers¹², Wolfgang Müller-Felber¹³, Barbara Plecko⁵, Christof Reihle¹⁴, Kurt Schlachter¹⁵, Gudrun Schreiber¹⁶, Oliver Schwartz¹⁷, Martin Smitka⁶, Elisabeth Steiner¹⁸, Corinna Stoltenburg¹⁹, Burkhard Stüve²⁰, Manuela Theophil¹², Claudia Weiß¹⁹, Gert Wiegand²¹, Ekkehard Wilichowski⁸, Benedikt Winter²², Wolfgang Wittmann²³, Ulrike Schara²⁴, Janbernd Kirschner^{1

25}

Affiliations

¹ Department of Neuropediatrics and Muscle Disorders, Medical Center - University of Freiburg, Faculty of Medicine, University of Freiburg, Germany.
² Department of Pediatrics I, Division of Pediatric Neurology, Medical University of Innsbruck, Innsbruck, Austria.
³ Department of Pediatrics, Neuropediatrics, Kaiser Franz Josef Krankenhaus mit Gottfried von Preyer Children Hospital, Vienna, Austria.
⁴ Department of Pediatric Neurology, Saarland University Medical Center, Homburg, Germany.
⁵ Department of Pediatrics and Adolescent Medicine, Division of General Pediatrics, Medical University of Graz, Graz, Austria.
⁶ Abteilung Neuropädiatrie, Medizinische Fakultät Carl Gustav Carus, Technische Universität Dresden, Germany.
⁷ Children's Hospital of Eastern Switzerland, St Gallen, Switzerland.
⁸ Department of Pediatrics and Adolescent Medicine, Division of Pediatric Neurology, University Medical Center Göttingen, Georg August University, Robert Koch Strasse 40, Göttingen, 37075, Germany.
⁹ Department of Paediatric Neurology, University Children's Hospital, Tübingen, Germany.
¹⁰ Department of Pediatrics, Neuropediatrics, University Medical Center Hamburg-Eppendorf, Hamburg, Germany.
¹¹ Department of Neuropaediatrics, University Children's Hospital, Ruhr-University Bochum, Bochum, Germany.
¹² Department of Pediatrics and Neuropediatrics, DRK Klinikum Westend, Berlin, Germany.
¹³ Department of Neuropediatrics, Dr. v. Haunersche Kinderklinik, University Children's Hospital, LMU, München, Germany.
¹⁴ Department for Pediatric Neurology, Center for Child and Adolescent Medicine Olgahospital, Psychosomatic and Pain Medicine, Child Pain Center Baden-Württemberg, Klinikum Stuttgart, Stuttgart, Germany.
¹⁵ Department of Pediatrics, State Hospital of Bregenz (LKH Bregenz), Bregenz, Austria.
¹⁶ Department of Neuropediatrics, Klinikum Kassel, Kassel, Germany.
¹⁷ Department of Neuropediatrics, University Children's Hospital Muenster, Muenster, Germany.
¹⁸ Department of Pediatrics, Klinikum Wels-Grieskirchen, Wels, Austria.
¹⁹ Department of Neuropediatrics, Charité - Universitätsmedizin Berlin, Berlin, Germany.
²⁰ Department of Neuropediatrics, Kliniken der Stadt Köln, Children's Hospital, Cologne, Germany.
²¹ Neuropediatrics Section of the Department of Pediatrics, Asklepios Clinic Hamburg, Nord-Heidberg, Hamburg, Germany.
²² Department of Pediatrics, Ulm University, Ulm, Germany.
²³ Kinderzentrum St. Martin - Social Paediatric Centre Regensburg, Regensburg, Germany.
²⁴ Department of Neuropediatrics, Children's Hospital 1, University of Duisburg-Essen, Essen, Germany.
²⁵ Department of Neuropediatrics, University Hospital Bonn, Bonn, Germany.

PMID: 31744015
DOI: 10.3233/JND-190441

Free article

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

Astrid Pechmann et al. J Neuromuscul Dis. 2020.

Free article

. 2020;7(1):41-46.

doi: 10.3233/JND-190441.

Authors

Affiliations

¹ Department of Neuropediatrics and Muscle Disorders, Medical Center - University of Freiburg, Faculty of Medicine, University of Freiburg, Germany.
² Department of Pediatrics I, Division of Pediatric Neurology, Medical University of Innsbruck, Innsbruck, Austria.
³ Department of Pediatrics, Neuropediatrics, Kaiser Franz Josef Krankenhaus mit Gottfried von Preyer Children Hospital, Vienna, Austria.
⁴ Department of Pediatric Neurology, Saarland University Medical Center, Homburg, Germany.
⁵ Department of Pediatrics and Adolescent Medicine, Division of General Pediatrics, Medical University of Graz, Graz, Austria.
⁶ Abteilung Neuropädiatrie, Medizinische Fakultät Carl Gustav Carus, Technische Universität Dresden, Germany.
⁷ Children's Hospital of Eastern Switzerland, St Gallen, Switzerland.
⁸ Department of Pediatrics and Adolescent Medicine, Division of Pediatric Neurology, University Medical Center Göttingen, Georg August University, Robert Koch Strasse 40, Göttingen, 37075, Germany.
⁹ Department of Paediatric Neurology, University Children's Hospital, Tübingen, Germany.
¹⁰ Department of Pediatrics, Neuropediatrics, University Medical Center Hamburg-Eppendorf, Hamburg, Germany.
¹¹ Department of Neuropaediatrics, University Children's Hospital, Ruhr-University Bochum, Bochum, Germany.
¹² Department of Pediatrics and Neuropediatrics, DRK Klinikum Westend, Berlin, Germany.
¹³ Department of Neuropediatrics, Dr. v. Haunersche Kinderklinik, University Children's Hospital, LMU, München, Germany.
¹⁴ Department for Pediatric Neurology, Center for Child and Adolescent Medicine Olgahospital, Psychosomatic and Pain Medicine, Child Pain Center Baden-Württemberg, Klinikum Stuttgart, Stuttgart, Germany.
¹⁵ Department of Pediatrics, State Hospital of Bregenz (LKH Bregenz), Bregenz, Austria.
¹⁶ Department of Neuropediatrics, Klinikum Kassel, Kassel, Germany.
¹⁷ Department of Neuropediatrics, University Children's Hospital Muenster, Muenster, Germany.
¹⁸ Department of Pediatrics, Klinikum Wels-Grieskirchen, Wels, Austria.
¹⁹ Department of Neuropediatrics, Charité - Universitätsmedizin Berlin, Berlin, Germany.
²⁰ Department of Neuropediatrics, Kliniken der Stadt Köln, Children's Hospital, Cologne, Germany.
²¹ Neuropediatrics Section of the Department of Pediatrics, Asklepios Clinic Hamburg, Nord-Heidberg, Hamburg, Germany.
²² Department of Pediatrics, Ulm University, Ulm, Germany.
²³ Kinderzentrum St. Martin - Social Paediatric Centre Regensburg, Regensburg, Germany.
²⁴ Department of Neuropediatrics, Children's Hospital 1, University of Duisburg-Essen, Essen, Germany.
²⁵ Department of Neuropediatrics, University Hospital Bonn, Bonn, Germany.

PMID: 31744015
DOI: 10.3233/JND-190441

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.

PubMed Disclaimer

MeSH terms

Actions
Actions
Actions
Actions
Actions
Actions
Actions
Actions
Actions
Actions
Actions

Substances

Actions
Actions

LinkOut - more resources

Full Text Sources
- Atypon
- IOS Press

Save citation to file

Email citation

Add to Collections

Add to My Bibliography

Your saved search

Create a file for external citation management software

Your RSS Feed

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

Affiliations

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

Authors

Affiliations

Abstract

MeSH terms

Substances

LinkOut - more resources

Full Text Sources