Exploring synthetic controls in rare diseases with a proof of concept in spinal cord injury
We provide a comprehensive benchmark of data-driven prediction architectures for neurological recovery after SCI. Apart from offering individual patients a specific recovery prediction, these models constitute the basis for synthetic controls. Using real-world data from a completed trial in SCI, we show that synthetic controls could mitigate the challenges of small cohorts and patient recruitment in rare disorders, offering the opportunity to maximize the number of patients receiving an investigative treatment. GITHUB REPOSITORY: https://gitlab.ethz.ch/BMDSlab/publications/sci/sci-in-silico-trials .
Authors
- Norbert Weidner (ORCID: https://orcid.org/0000-0002-1447-8967)
- Rainer Abel (ORCID: https://orcid.org/0000-0002-8328-8438)
- Fred H. Geisler (ORCID: https://orcid.org/0000-0002-0899-9045)
- Frank Röhrich
- Thomas Liebscher (ORCID: https://orcid.org/0000-0002-6025-6564)
- Abel Torres‐Espín (ORCID: https://orcid.org/0000-0002-9787-8738)
- Martin Schubert (ORCID: https://orcid.org/0000-0003-4495-6533)
- John L. K. Kramer (ORCID: https://orcid.org/0000-0001-7722-131X)
- Sarah C. Brüningk (ORCID: https://orcid.org/0000-0003-3176-1032)
- Catherine R. Jutzeler (ORCID: https://orcid.org/0000-0001-7167-8271)
- Marc Bolliger (ORCID: https://orcid.org/0000-0001-9059-0304)
- Doris Maier (ORCID: https://orcid.org/0000-0002-3261-2381)
- Armin Curt
- Louis P. Lukas (ORCID: https://orcid.org/0000-0001-9307-0945)
- Yorck-Bernhard Kalke (ORCID: https://orcid.org/0000-0002-4548-258X)
- Samuel Håkansson (ORCID: https://orcid.org/0000-0002-8681-0113)
- Miklovana Tuci (ORCID: https://orcid.org/0009-0005-0507-2331)
- Harvinder Singh Chhabra (ORCID: https://orcid.org/0000-0001-9795-2443)
- Rüdiger Rupp
- Olga Taran
Institutions
- University of Bern (CH)
- SIB Swiss Institute of Bioinformatics (CH)
- University of British Columbia (CA)
- University of Waterloo (CA)
- University of Alberta (CA)
- University of California, San Francisco (US)
- Universität Ulm (DE)
- University of Zurich (CH)
- Heidelberg University (DE)
- University Hospital of Bern (CH)
- University Hospital Heidelberg (DE)
- Bayreuth Medical Center (DE)
- University of Saskatchewan (CA)
- ETH Zurich (CH)
- Berufsgenossenschaftliche Unfallklinik Murnau (DE)
- Heidelberg Engineering (Germany) (DE)
- International Collaboration On Repair Discoveries (CA)
- BG Klinikum Bergmannstrost Halle (DE)
- Diagnostic and Treatment Centre Berlin (DE)
- Neurological Surgery (US)
- Heidelberg University (US)
Publication Details
- Journal
- BMC Medicine
- Published
- 2025-10-24
- DOI
- https://doi.org/10.1186/s12916-025-04405-3
- Citations
- 1
- Primary Topic
- Spinal Cord Injury Research
- Type
- article
- Field-Weighted Citation Impact
- 1.47
Funders
- National Science Foundation
- Wings for Life
- International Foundation for Research in Paraplegia
- Schweizerischer Nationalfonds zur Förderung der Wissenschaftlichen Forschung