Delphi consensus on gene therapy of spinal muscular atrophy with onasemnogene abeparvovec in Germany, Austria and Switzerland – part II – expert based recommendations for surveillance and management of side-effects

BACKGROUND: Systematic protocols for long-term surveillance of children with spinal muscular atrophy treated with onasemnogene abeparvovec are lacking. Together with best practice recommendations for safety monitoring and management, such recommendations should increase patient safety and confidence levels of healthcare providers. OBJECTIVE: Based on systematic literature review and evidence grading from part 1, this initiative aims to develop a structured treatment plan applicable across all treatment centers in Germany, Austria and Switzerland. Additionally, it seeks to establish consensus recommendations for the clinical management of safety alerts. METHODS: Part 2 describes the methodology used to formulate Delphi consensus statements, the development of a structured treatment plan for OA treatment, and the anonymous consensus voting process with standardized follow-up in case of disagreement. RESULTS: A total of 12 consensus statements were developed, addressing diagnostic work-up, safety evaluation, and best practice management of common adverse drug reactions associated with OA gene therapy. All statements achieved >95% consensus in the anonymous Delphi voting. Additionally, two consensus recommendations for handling of positive newborn screening result achieved consensus of 97% and 87%, respectively. A structured treatment plan for gene therapy was consented with 100% agreement, as were standardized recommendations for laboratory testing and a consensus-based algorithm for management of liver transaminase elevations. CONCLUSIONS: Delphi-based expert recommendations, developed in co-creation with patient representatives, provide a framework to minimize complications associated with gene therapy and establish the basis for standardized post-marketing data collections. The methodology used in this Delphi-consensus-group can serve as a blueprint for future gene therapy approvals.

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Journal
Journal of Neuromuscular Diseases
Published
2026-08-27
DOI
https://doi.org/10.1177/22143602261433270
Primary Topic
Neurogenetic and Muscular Disorders Research
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article
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article

Delphi consensus on gene therapy of spinal muscular atrophy with onasemnogene abeparvovec in Germany, Austria and Switzerland – part II – expert based recommendations for surveillance and management of side-effects

Ralf A. Husain, Maria‐Luisa Schubert, Heymut Omran, Hendrik Rosewich et al.
Journal of Neuromuscular Diseases
Neurogenetic and Muscular Disorders Research
article

Delphi consensus on gene therapy of spinal muscular atrophy with onasemnogene abeparvovec in Germany, Austria and Switzerland – part II – expert based recommendations for surveillance and management of side-effects

Ralf A. Husain, Maria‐Luisa Schubert, Heymut Omran, Hendrik Rosewich, Matthias Baumann, Samuel Gröschel, Marina Flotats‐Bastardas, Klaus Goldhahn, Janbernd Kirschner, Astrid Eisenkölbl, Hans Hartmann, Veronka Horber, Regina Trollmann, Andreas Ziegler, Arpad von Moers, Jonas Denecke, Georg M. Stettner, Johannes Friese, Marcel Teichert, Moritz Tacke, Thomas Opladen, Eva Stumpe, Gudrun Schreiber, Georg Lenz, Beate Hauptrock, Sabine Illsinger, Astrid Blaschek, Jessika Johannsen, Günther Bernert, Raphael Teipel, Katharina Vill, Silke Heidenreich, Simon Call, Oswald Hasselmann, Corinna Stoltenburg, Olaf Penack, Julia Riemann, David Jacquier, Heike Kölbel, Francis Ayuk, Dorothea Holzwarth, Cornelia Köhler, Gesine Bug, Claudia Weiß, Claudia Lengerke, Georg F. Hoffmann, Fabian Hauck, Oliver Schwartz, Christian Rauscher, Ulrike Schara, Michael von Bergwelt, Claudia Ganter, Martin Smitka, A. Klein, Joachim Sproß, Maja von der Hagen, Eva Wagner‐Drouet, Michael Kolodzig, Veit Bücklein, Bastian von Tresckow, Ekkehard Wilichowski, Antonia Busse, Angela M. Kaindl, Hubert Serve, Ania C. Muntau, Fabian Müller, Joanna Schneider, Dennis Bene, Lars Bullinger, Andreas Mackensen, Nina Rademacher, Bärbel Entrich, Lisa-Valerie Bitzan, Astrid Pechmann, Martin Bornhäuser, Wolfgang Bethge, Gerd Wiegand, Matthias Stelljes, Andreas Ziegler, Christian Reinhardt, Andreas A. Hahn, Carsten Müller-Tidow, Stefan Kölker, Wolfgang Müller-Felber, Peter Dreger, Christoph Klein, Josephine Schädlich, Hélène Guillemot, Malte von Bonin, Ulrike Schara-Schmidt, Marion Subklewe
article en

Abstract

BACKGROUND: Systematic protocols for long-term surveillance of children with spinal muscular atrophy treated with onasemnogene abeparvovec are lacking. Together with best practice recommendations for safety monitoring and management, such recommendations should increase patient safety and confidence levels of healthcare providers. OBJECTIVE: Based on systematic literature review and evidence grading from part 1, this initiative aims to develop a structured treatment plan applicable across all treatment centers in Germany, Austria and Switzerland. Additionally, it seeks to establish consensus recommendations for the clinical management of safety alerts. METHODS: Part 2 describes the methodology used to formulate Delphi consensus statements, the development of a structured treatment plan for OA treatment, and the anonymous consensus voting process with standardized follow-up in case of disagreement. RESULTS: A total of 12 consensus statements were developed, addressing diagnostic work-up, safety evaluation, and best practice management of common adverse drug reactions associated with OA gene therapy. All statements achieved >95% consensus in the anonymous Delphi voting. Additionally, two consensus recommendations for handling of positive newborn screening result achieved consensus of 97% and 87%, respectively. A structured treatment plan for gene therapy was consented with 100% agreement, as were standardized recommendations for laboratory testing and a consensus-based algorithm for management of liver transaminase elevations. CONCLUSIONS: Delphi-based expert recommendations, developed in co-creation with patient representatives, provide a framework to minimize complications associated with gene therapy and establish the basis for standardized post-marketing data collections. The methodology used in this Delphi-consensus-group can serve as a blueprint for future gene therapy approvals.

Journal of Neuromuscular Diseases
University of Bern (CH), Innsbruck Medical University (AT), Universität Hamburg (DE), University of Freiburg (DE), Friedrich-Alexander-Universität Erlangen-Nürnberg (DE), Universität Innsbruck (AT), Heidelberg University (DE), Paracelsus Medical University (AT), University Hospital Bonn (DE), University Hospitals of the Ruhr-University of Bochum (DE), University Hospital of Bern (CH), University Hospital Heidelberg (DE), Medizinische Hochschule Hannover (DE), Uniwersytecki Szpital Dziecięcy (PL), Rems-Murr-Klinikum (DE), University Hospital Münster (DE), University Medical Center Hamburg-Eppendorf (DE), Children's Clinical University Hospital (LV), Universitätsmedizin Göttingen (DE), Kepler Universitätsklinikum (AT), University of Education Freiburg (DE), German Cardiac Society (DE), Changchun Children's Hospital (CN), University Children's Hospital Zurich (CH), Jena University Hospital (DE), German Liver Foundation (DE), Klinikum Kassel (DE), University Hospital Carl Gustav Carus (DE), Asklepios Kliniken Hamburg (DE), Ostschweizer Kinderspital (CH), University of Duisburg-Essen (DE), Technische Universität Dresden (DE), Charité - Universitätsmedizin Berlin (DE), Ludwig-Maximilians-Universität München (DE), Ruhr University Bochum (DE), Saarland University (DE), University of Lausanne (CH)
Good health and well-being
Openalex Percentile: Top 11%
Neurogenetic and Muscular Disorders Research
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